Reported trial results for Motor Neurone Disease

50 trials have reported results.

AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

• NCT04220021 · results posted 2 December 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT04220021) enrolled 41 people with a specific genetic form of ALS (a motor neurone disease) caused by a change in a gene called C9orf72, who were already taking the medication metformin. The trial was looking at two main things: whether any unexpected side effects arose during the study period, and whether levels of a particular protein in the fluid around the brain and spine (called a RAN protein, specifically a type known as polyglycine-proline or "GP") changed over time. A total of 23 participants completed the study, while 18 did not finish. The reported data shows that, for the primary safety measure, zero participants — both among those who completed the study and among all 41 who started — experienced unexpected treatment-related side effects (called "treatment-emergent adverse events"). For the protein level measure, the reported data shows an average decrease of about 28% in GP protein levels in the spinal fluid among those completers who had reliable test results — though it is important to note this figure comes from a smaller subset of participants, and no comparison group without metformin was included. The reported data also shows scores on a disability rating scale (the ALSFRS-R, which runs from 0 to 48, where 48 means full function) across four time points: at the start of the study, completers averaged around 38.6 out of 48, and by the final visit at roughly 24 weeks, this had dropped to around 34.1–34.5, reflecting a gradual change in functional scores over time. It is worth noting that 18 of the 41 participants did not complete the study, which the reported data does not fully explain, and there was no separate control group (a group not receiving the treatment) to compare results against. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04866771 · results posted 7 August 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT04866771) involved 14 people with ALS (a serious neurological condition) who were split into two groups of 7. One group received a brain stimulation technique called transcranial direct current stimulation (tDCS) — which uses a mild electrical current applied to the scalp — while the other group was a delayed-start control group, meaning they were scheduled to receive the same treatment later. The trial was primarily measuring how participants' day-to-day functioning changed over time using a standard ALS questionnaire, and also looked at walking speed and ankle movement control, among other things. Not everyone completed the study — all 7 participants in the tDCS group finished, while only 3 of the 7 in the control group did. The reported data shows that on the main questionnaire (called the ALSFRS-R, which scores daily functioning from 0 to 48, with higher scores meaning more ability retained), the tDCS group's scores changed by −1 at one point and −3.2 at another, while the control group's scores changed by −7.1 at both time points. A negative number here means a decline in functioning. For walking speed, the reported data shows the tDCS group's average speed changed by −0.11 metres per second, compared to −0.30 metres per second in the control group. For the remaining secondary measures — ankle motor control, quality of life questionnaires, and a fatigue scale — the data was not reported in the results submitted to ClinicalTrials.gov. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT05349721 · results posted 1 August 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT05349721) looked at a drug called utreloxastat in people living with ALS (also known as motor neurone disease). The trial had three parts. In the first 24-week part, 222 people were assigned to utreloxastat and 114 to a placebo (a dummy treatment with no active ingredient). The main thing researchers were measuring was a combined score that took into account both survival and day-to-day functioning — using a standard ALS questionnaire called the ALSFRS-R, which rates 12 everyday activities on a scale where higher scores mean better function (maximum score of 48). The reported data shows that for the main measure — a ranking system where a higher number meant a better combined outcome of survival and functioning — the utreloxastat group scored an average rank of 172.22 and the placebo group scored 165.36, out of a possible range of 1 to 306. For the secondary measure of actual ALSFRS-R questionnaire scores at 24 weeks, the reported data shows the utreloxastat group averaged 29.4 and the placebo group averaged 31.4 (out of 48). For breathing capacity (measured as a percentage of what would be expected for a healthy person of similar age), the utreloxastat group showed a change of −14.62% and the placebo group −15.92% from the start of the trial. The reported data also shows that 174 out of 220 people in the utreloxastat group and 90 out of 114 in the placebo group experienced at least one adverse event (an untoward medical occurrence) during the trial. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03705390 · results posted 26 June 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT03705390) involved a single group of participants who received a treatment called ILB®. Eleven people started the initial 10-week treatment period, and nine completed it (two did not finish). Those nine then moved into an extended treatment phase, with eight completing that stage. The trial's primary focus was on tracking and recording adverse events — that is, any unwanted or unexpected health occurrences that happened during the study — and serious adverse events (SAEs), which are a more severe category involving hospitalisation or other significant medical concerns. The reported data shows that across the study, a total of 270 adverse events were recorded among the participants, along with 1 serious adverse event. When the 270 adverse events were broken down by how severe they were (using a standard grading scale from 1 to 5, where 1 is mild and 5 is death), the reported data shows 265 were Grade 1 (mild), 4 were Grade 2 (moderate), 1 was Grade 3 (severe but not immediately life-threatening), and none were Grade 4 or Grade 5. The single serious adverse event was graded as Grade 3 and was reported as unrelated to the study treatment. When the 270 adverse events were assessed by how likely they were to be connected to the treatment, 127 were reported as unrelated, 45 as unlikely to be related, 93 as definitely related, 4 as possibly related, and 1 as probably related. It is worth noting that this was a very small, single-arm trial — meaning there was no comparison group — and the results reflect what was observed and recorded in this specific group of participants only. No secondary outcome data was included in the submitted results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT05053035 · results posted 18 June 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT05053035) was a small study involving just 5 participants in total — 3 received the investigational treatment AL001 and 2 received a placebo (an inactive substitute). The trial was measuring several things: whether participants experienced any unwanted health events (called adverse events), whether the body produced any immune responses to AL001 (known as antibodies against the drug), how much of the drug appeared in the blood and in cerebrospinal fluid (the fluid surrounding the brain and spine) at 24 weeks, and how levels of a protein called progranulin changed over the same period. Progranulin is a naturally occurring protein in the body that this drug is designed to influence. The reported data shows that 2 out of 3 participants in the AL001 group and both participants in the placebo group experienced at least one adverse event during the study. Regarding immune responses to the drug, 1 out of 3 participants in the AL001 group tested positive for antibodies against AL001 at week 24, while neither placebo participant did. For the drug concentration measurements, AL001 was detected in the blood at an average level of approximately 705,793 ng/mL and in the cerebrospinal fluid at approximately 548 ng/mL at week 24; no corresponding figures were reported for the placebo group for these measures. The reported data also shows that progranulin levels in the blood appeared to change by an average of +175 ng/mL in the AL001 group compared with −24.4 ng/mL in the placebo group, and progranulin in the cerebrospinal fluid changed by approximately +2.9 ng/mL in the AL001 group compared with −0.4 ng/mL in the placebo group. It is worth noting that with only 5 participants overall, these numbers reflect a very small group of people. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03645031 · results posted 15 May 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT03645031) looked at whether a breathing treatment called "acute intermittent hypoxia" (AIH) — which involves briefly breathing air with lower oxygen levels than normal — had any measurable effect on breathing muscle strength and hand grip strength in people with ALS (also known as motor neurone disease) compared to healthy volunteers. A total of 29 people started the study across four groups: people with ALS and healthy controls, each receiving either the real AIH treatment first or a sham (fake) version first, then swapping over. Between 4 and 8 people completed each group. The reported data shows the following measured changes, expressed as percentage changes from each person's starting (baseline) levels, taken 60 minutes after the breathing session. For breathing muscle strength measured at the mouth (called Maximal Inspiratory Pressure), the ALS group showed a reported change of +0.3% after AIH and −4.8% after sham, while the healthy control group showed −3.1% after AIH and −5.0% after sham. For a separate breathing strength test measured through the nose (Sniff Nasal Inspiratory Pressure), the ALS group showed +2.5% after AIH and +4.9% after sham; the healthy control group showed +7.4% after AIH and +5.8% after sham. For hand grip strength, the ALS group showed −8.2% after AIH and +14.5% after sham, while healthy controls showed −11.2% after AIH and +18.2% after sham. Secondary measures — including how much air was breathed per minute, a measure of breathing drive (called occlusion pressure, measured in units of cm H₂O), and electrical activity across eight breathing muscles — were also recorded and reported across the groups, with varying positive and negative percentage changes as detailed in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04579666 · results posted 24 April 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT04579666) looked at a drug called pegcetacoplan in people with ALS (also known as motor neurone disease). The first part of the trial (called the Randomised Treatment Period, or RTP) ran for up to 52 weeks and included 169 people who received pegcetacoplan and 80 people who received a placebo (a dummy treatment with no active ingredient). A second open-label period then ran from week 52 to week 104, where 97 and 50 participants respectively continued on or switched to pegcetacoplan. The trial measured a combined score that ranked participants based on how their physical functioning and survival compared to everyone else in the trial, as well as tracking unwanted medical events and a number of other outcomes. The reported data shows that for the main combined ranking score at week 52 — where a higher number meant a relatively better outcome compared to other participants — the pegcetacoplan group had an average rank of 123.0 and the placebo group had an average rank of 126.0, out of a possible range of 1 to 247. For the secondary measure of physical functioning (the ALSFRS-R scale, scored 0–48 where higher is better), both groups showed a decline from their starting scores by week 52: the pegcetacoplan group declined by an average of 16.5 points and the placebo group by 15.8 points. Regarding unwanted medical events during the first 52 weeks, 137 of 169 participants in the pegcetacoplan group and 61 of 80 in the placebo group reported at least one such event; serious unwanted medical events were reported in 57 and 27 participants respectively. Suicidal thoughts were recorded in 16 pegcetacoplan participants and 6 placebo participants; suicidal behaviour was reported in 1 pegcetacoplan participant and none in the placebo group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03580616 · results posted 20 April 2025

  According to the results reported on ClinicalTrials.gov, this trial enrolled 43 people, all of whom received L-Serine, a naturally occurring amino acid. The trial was looking at how well participants tolerated the supplement (based on self-reported stomach and digestive symptoms) and also tracked two other measures: a standard ALS function score (called the ALSFRS-R, which rates a person's ability to carry out 12 everyday activities on a scale from 0 to 48, where higher means better function), and a breathing test called Forced Vital Capacity. It is worth noting that while 43 people started the trial, none were recorded as having formally "completed" it in the reported data, and 28 were included in an interim (partway-through) analysis. The reported data shows that when it came to tolerability, 7 participants reported gastrointestinal (digestive) symptoms at some point, 1 participant reported symptoms at another recorded time point, and 8 participants were captured in a third category — though the data as submitted does not provide labels clearly distinguishing these three groups, so the exact breakdown cannot be described with full certainty. For the ALS function score, the reported data shows an average score of approximately 28.9 out of 48 across participants. For the breathing test (Forced Vital Capacity), the reported data shows no numbers were submitted to ClinicalTrials.gov, so those results were not reported. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT05178810 · results posted 18 March 2025

  According to the results reported on ClinicalTrials.gov, this trial looked at a drug called FAB122 compared to a placebo (an inactive treatment) in people living with ALS (Amyotrophic Lateral Sclerosis, also known as Motor Neurone Disease). A total of 205 people were assigned to receive FAB122 and 97 received the placebo. The trial ran for up to 72 weeks and used a standard ALS questionnaire called the ALSFRS-R — a 48-point scale where higher scores mean better day-to-day functioning — as its main way of measuring change over time. The reported data shows that after 48 weeks, participants in the FAB122 group had an average decline of 11.2 points on the ALSFRS-R scale, while those in the placebo group had an average decline of 10.8 points. For the secondary measures, the reported data shows that at 72 weeks, the FAB122 group had an average decline of 15.2 points compared to 12.6 points in the placebo group. A combined ranking score that takes into account both survival and functioning (called CAFS, where a higher rank is a better result) was also reported: at 48 weeks the FAB122 group scored 153.31 versus 147.68 for placebo, but at 72 weeks the FAB122 group scored 48.7 versus 54.15 for placebo. For survival probability over 72 weeks — which in this trial also included needing a breathing machine for extended periods — the reported figure was 0.728 (roughly 73%) for the FAB122 group and 0.870 (roughly 87%) for the placebo group. Scores measuring specific functions like speech and swallowing were also reported, and the data was not reported in a way that showed a clear consistent pattern favouring either group across all time points. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04607044 · results posted 9 January 2025

  According to the results reported on ClinicalTrials.gov, this trial enrolled 45 participants, all of whom had a nerve-related condition (neuropathy) and received one or more lower-limb surgical implant devices made by Wright Medical. The trial was measuring things like how many participants kept their limb (i.e., did not need an amputation) over time, as well as how participants felt about their own health, daily activities, and physical function. The reported data shows that none of the 45 participants were recorded as having completed the study — all 45 are listed under "not completed," which may reflect the ongoing or early-closure nature of the trial, though no further explanation was provided in the submitted data. The reported data shows the following numbers at the one-year mark: only 1 participant was counted in the primary survival analysis looking at amputation rates. For the self-reported quality-of-life questionnaire (called EQ-5D-5L), participants reported a health score of 0.88 out of a possible 1 — where a higher number means a person rated their overall health state more favourably. On a separate part of the same questionnaire where people rated their health on a scale from 0 to 100, the reported average was 86.3. For daily physical activities (measured using a tool called the FAAM-ADL, scored as a percentage where 100% means no difficulty at all), the reported average was 96.9%. The five-year amputation survival analysis and the x-ray bone-healing assessment did not have any numbers reported in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04944784 · results posted 5 December 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT04944784) looked at a drug called reldesemtiv in people living with ALS (amyotrophic lateral sclerosis, also known as motor neurone disease). During the main double-blind period — where neither participants nor researchers knew who was receiving the drug or a dummy treatment (placebo) — 325 people were assigned to reldesemtiv and 161 to placebo. Of those, 180 and 96 respectively completed that phase. The trial's main goal was to measure changes in everyday physical functioning over 24 weeks using a standard ALS rating scale (scored 0–48, where higher means better function). The reported data shows that, on the main measure, the reldesemtiv group's average score declined by 5.57 points over 24 weeks, while the placebo group's average score declined by 4.76 points — both groups showed a drop in function. On a combined measure that also factored in survival and the need for breathing support, the reldesemtiv group received an average rank of 232.5 compared to 264.0 for the placebo group (higher rank indicating a relatively better combined outcome). For breathing capacity, the reldesemtiv group showed an average decline of about 10.6 percentage points versus 9.7 for placebo. A quality-of-life questionnaire (scored 0–100, lower being better) showed an average increase — meaning worsening — of 11.4 points in the reldesemtiv group and 9.8 points in the placebo group. Handgrip strength declined by an average of about 10.1 pounds in the reldesemtiv group and 7.4 pounds in the placebo group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT05442775 · results posted 13 August 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT05442775) enrolled 71 people who received a treatment called reldesemtiv. The trial was an open-label, long-term study looking at what happened to participants over time when taking this medicine. Notably, the reported data shows that none of the 71 participants were recorded as having formally "completed" the study — all 71 were listed under "not completed," though no further explanation for this is provided in the submitted data. The primary thing the trial was measuring was safety and tolerability over the long term — specifically, how many participants experienced what are called "treatment-emergent adverse events" (meaning any unwanted health events that occurred after starting the treatment). The reported data shows that 39 out of 71 participants experienced at least one such event. The trial also tracked participants' physical functioning using a standard ALS rating scale called the ALSFRS-R, which runs from 0 to 48, where 48 represents fully normal function. The reported data shows average scores of 29.5, 27.4, and 26.6 at different points during the study — suggesting the scores were measured at three separate time points, though the exact timing of each measurement was not specified in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04098406 · results posted 3 July 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT04098406) looked at a drug called CNM-Au8 (a suspension of gold nanocrystals) in people with ALS (also known as motor neurone disease). A total of 45 people took part — 22 were assigned to a placebo (an inactive treatment) and 23 received a 30 mg daily dose of CNM-Au8. The trial ran for 36 weeks and was primarily measuring changes in nerve and muscle function over time using a specialised electrical test called MUNIX, which gives a score reflecting how many working nerve cells are still supplying certain muscles. A higher score is better; a falling score suggests disease progression. The reported data shows that, over the 36-week period, both groups experienced a decline in their MUNIX scores — meaning both groups showed a drop in this measure of nerve function. The placebo group's combined MUNIX score (across four muscles) fell by approximately 39.6%, while the CNM-Au8 group's score fell by approximately 31.8%. In absolute terms, the placebo group's score dropped by about 141 points on the index, compared to about 124 points in the CNM-Au8 group. For the secondary measure of breathing capacity (Forced Vital Capacity, or FVC — a standard test of how much air someone can breathe out), the placebo group showed a reported decline of about 20.3%, while the CNM-Au8 group showed a reported decline of about 16.7%. Several other pre-specified outcome measures were listed in the trial registration but no numerical results were reported for those. It is worth noting this was a relatively small trial with 45 participants, and the data was submitted to ClinicalTrials.gov as described above. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04490148 · results posted 18 June 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT04490148) enrolled 29 people in total — 12 in a remote lung function testing-only group and 17 in a remote lung function testing plus nurse coaching group. The study was looking at whether testing lung function from home (rather than attending a clinic) could pick up earlier when someone might need breathing support (a machine called non-invasive ventilation, or NIV). It also tracked participants' confidence in managing their own health — including their medications, social life, and symptoms — over 12 months, as well as how much breathlessness they reported over time. The reported data shows that, across all participants combined, remote home-based lung function testing flagged the point at which someone may need breathing support at an average of 176 days after joining the study. In comparison, the standard in-clinic lung function test reached that same point at an average of 259 days after enrolment — meaning the remote test appeared to reach this threshold earlier. For the self-confidence (self-efficacy) scores — measured on a scale where 50 is the average — the reported data shows that in the remote-testing-only group, scores for managing medications and treatments changed by about −0.24 points per month, while the nurse coaching group showed a change of about +0.47 points per month. Changes in scores for managing social interactions and managing symptoms were small in both groups, ranging from roughly −0.04 to −0.28 points per month depending on the group and category. The reported data also shows that breathlessness scores — on a scale of 0 to 44, where higher means more breathlessness — changed slowly over time in both groups: by about 0.03 points per month in the remote-testing-only group and 0.27 points per month in the nurse coaching group. It is worth noting that 1 person in the remote-testing group and 5 in the nurse coaching group did not complete the study, and the reasons for this were not reported in the data provided. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04254913 · results posted 24 January 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT04254913) enrolled 6 participants, all of whom received the study treatment MT-1186, a form of edaravone. Five participants completed the trial and one did not. The trial was measuring how the drug moves through the body after it is taken — specifically, how quickly it is absorbed into the bloodstream, how high the concentration in the blood gets, and how long it takes for the body to clear it. This type of measurement is known as pharmacokinetics, which simply means tracking how a drug travels through the body over time. The reported data shows the following numbers for the active ingredient edaravone in the blood: the total exposure to the drug over time (a measure called AUC, or area under the curve) was reported as 2,300 ng·h/mL. The highest level of the drug detected in the blood (called the peak concentration) was reported as 2,163 ng/mL, and this peak was reached very quickly — at around 0.29 hours (roughly 17 minutes) after dosing. The time it took for the amount of drug in the body to reduce by half (called the half-life) was reported as approximately 4.47 hours, and the average time the drug spent in the body overall was reported as 1.89 hours. These figures describe how MT-1186 behaved in the bloodstream of the small group of people who took part in this particular study. No data on other outcomes beyond these blood-level measurements was included in the reported results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04470050 · results posted 26 October 2023

  According to the results reported on ClinicalTrials.gov, this trial enrolled 225 people in total across seven groups. All participants were first stabilised on morphine (a strong painkiller used here to represent opioid dependence) during an initial maintenance phase. They were then moved into a double-blind treatment phase — meaning neither the participants nor the researchers knew who was receiving which treatment — where different groups received one of six doses of an investigational treatment (30, 60, 90, 120, 180, or 240 micrograms) or a placebo (a dummy treatment with no active ingredient). The trial was measuring opioid withdrawal symptoms using two questionnaires: the SOWS (scored 0–30, where higher means more severe symptoms) and the COWS (scored 0–48, with similar meaning). It also tracked how long participants stayed in the study and how many dropped out after the morphine maintenance phase ended. The reported data shows that at the start of the double-blind phase (baseline), peak SOWS scores across the active-dose groups ranged from about 3.6 to 6.5 out of 30, and the placebo group scored about 6.2. Over time, peak SOWS scores in some groups where data was reported ranged from around 4.0 to 9.1. For the COWS scale, baseline peak scores ranged from approximately 2.4 to 5.2 across dose groups, with the placebo group at 2.7; later scores ranged from about 4.1 to 6.7 where data was available. Not all time-point measurements were reported for every group in the data submitted. The reported data also shows that the average number of days participants stayed in the study before dropping out ranged from 1.5 days (60 microgram group) to 6.1 days (180 microgram group), with the placebo group at 2.0 days. Drop-out numbers during days 6–14 were also recorded, though complete figures for all groups across all time points were not reported in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT05039268 · results posted 11 October 2023

  According to the results reported on ClinicalTrials.gov, this trial enrolled 16 people in total, split into two groups of 8 — one group received a 15mg dose and the other received a 30mg dose of an investigational treatment called 3K3A-APC, which was being studied in the context of ALS (also known as motor neurone disease). All 16 participants completed the study. The trial was primarily measuring two things: whether participants experienced any serious unwanted health events (called adverse events) during the study, and whether there were any changes in a brain scan measure called the PERSI score, which tracks a type of brain cell activity (microglial activation) in the motor cortex — the part of the brain involved in movement. The reported data shows that zero participants in either the 15mg or the 30mg group experienced any serious adverse events, or any adverse events rated as more severe than "moderate." Regarding the brain scan measure, the reported data shows the PERSI score changed by −0.14% in the 15mg group and +0.53% in the 30mg group, meaning a very small decrease was recorded in one group and a very small increase in the other. It is important to note that these numbers alone do not tell us what a meaningful change would look like. For the secondary outcome measures — which included MRI brain scans, blood cell activity, and levels of certain proteins in blood and spinal fluid — the reported data shows that no numerical results were submitted to ClinicalTrials.gov for any of these measures. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04615923 · results posted 23 August 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT04615923) looked at a medicine called pridopidine in people living with ALS (also known as motor neurone disease). A total of 162 people took part — 120 received pridopidine and 42 received a matching placebo (a dummy treatment with no active ingredient). Of those, 96 people in the pridopidine group and 38 in the placebo group completed the study. The trial was mainly measuring two things: how quickly participants' physical function declined over time (using a standardised ALS rating scale scored out of 48, where higher scores mean more function), and how often death or the need for full-time breathing machine support occurred. The reported data shows that, for the main physical function measure, participants in the pridopidine group lost an average of 0.99 points per month on the rating scale, compared with 1.00 points per month in the placebo group — figures that are virtually identical. For the mortality measure, both groups reported the same rate of 0.012 events per month. The reported data also shows results for several secondary measures. Breathing capacity (measured as a percentage change) declined by 8.81% in the pridopidine group and 8.35% in the placebo group. For swallowing and speech function (the "bulbar" subdomain, scored out of 12), the overall change across all participants was −1.16 points in the pridopidine group and −1.25 in the placebo group. Among participants who already had some swallowing or speech difficulties at the start, the reported changes were −1.78 (pridopidine) and −1.69 (placebo). Among those whose disease had been progressing quickly before the trial began, both groups reported an identical change of −1.54 points in that same function area. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02623699 · results posted 28 July 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT02623699) tested a drug called BIIB067 (also known as tofersen) and was conducted in three parts. Parts A and B were early-phase dosing studies involving healthy or at-risk volunteers who received either BIIB067 at various doses (10 mg, 20 mg, 40 mg, 60 mg, or 100 mg) or a placebo (an inactive substance used for comparison). Part C was a larger pivotal stage involving 108 participants — 72 received BIIB067 at 100 mg and 36 received placebo. In total, across all three parts, around 178 people took part. The main things being measured in Parts A and B were how participants responded to the drug in terms of adverse events (unexpected or unwanted medical occurrences), as well as changes in laboratory tests, vital signs, physical and neurological examinations, and heart tracings (ECGs). The reported data shows that in Parts A and B, adverse events (any unwanted medical occurrence during the trial) were recorded across all groups. In Part A, 2 out of 5 placebo participants experienced an adverse event, while the numbers in the BIIB067 groups ranged from 2 out of 3 participants (10 mg group) up to all 6 participants in the 60 mg group. In Part B, all participants across most groups — including the 12 placebo participants — recorded at least one adverse event. No serious adverse events were reported for the placebo groups in Parts A or B based on the data provided. The reported data shows that no participants had clinically significant changes in vital signs across any group. Clinically significant laboratory abnormalities were recorded in 2 participants in the 40 mg group and 1 participant in the 60 mg group in Part B, and none in other groups. A small number of clinically significant ECG changes were recorded in Part B across several groups, including 3 placebo participants and between 1 and 3 participants in the various BIIB067 dose groups. Outcome measure data for Part C was not reported in the submitted results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04414345 · results posted 25 July 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT04414345) looked at a treatment called CNM-Au8 in people living with ALS (also known as motor neurone disease). A total of 161 people took part — 120 received CNM-Au8 and 41 received a matching placebo (a dummy treatment with no active ingredient). The trial was measuring two main things: how quickly participants' physical function declined over time (using a standard ALS rating scale called the ALSFRS-R, where higher scores mean better physical ability out of a maximum of 48), and the rate of death or the need for round-the-clock breathing machine support. The reported data shows that, on the ALSFRS-R scale, participants in the CNM-Au8 group declined by an average of 1.01 points per month, compared with 1.03 points per month in the placebo group. For the mortality measure, the CNM-Au8 group had a reported rate of 0.006 events per month, while the placebo group had a rate of 0.007 events per month. Looking at the secondary outcomes, the reported data shows that respiratory function (measured by lung capacity) changed by −9.32% in the CNM-Au8 group and −8.53% in the placebo group. Muscle strength declined by −27.54% in the CNM-Au8 group and −24.44% in the placebo group. By week 24, 3 participants in the CNM-Au8 group and 5 participants in the placebo group had died or met the criteria for a "death equivalent" (needing a breathing machine for more than 22 hours a day for more than 7 days in a row). These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04322149 · results posted 28 June 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT04322149) tested a treatment called AT-1501 across four different dose levels: 1.0 mg/kg, 2.0 mg/kg, 4.0 mg/kg, and 8.0 mg/kg. A total of 54 people took part — 9 in each of the two lower-dose groups and 18 in each of the two higher-dose groups. Most participants completed the study, with 49 out of 54 finishing. The trial's main focus was on tracking adverse events — that is, any unwanted or unexpected health changes that occurred while participants were receiving the treatment (called Treatment Emergent Adverse Events, or TEAEs). The reported data shows that across all four dose groups, a number of participants experienced at least one TEAE: 8 out of 9 in the 1.0 mg/kg group, 8 out of 9 in the 2.0 mg/kg group, 10 out of 18 in the 4.0 mg/kg group, and 16 out of 18 in the 8.0 mg/kg group. In terms of serious adverse events (SAEs — meaning health changes considered more significant in nature), the reported data shows that 0 participants in the 1.0 mg/kg group, 1 in the 2.0 mg/kg group, 0 in the 4.0 mg/kg group, and 0 in the 8.0 mg/kg group experienced at least one SAE. The total number of individual TEAEs recorded was 30, 66, 54, and 67 across the four dose groups respectively. No secondary outcome data was reported in the submitted results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT05003167 · results posted 8 June 2023

  According to the results reported on ClinicalTrials.gov, this trial involved 13 participants who all received a treatment called Expiratory Muscle Strength Training (EMST) — a type of breathing exercise where a person blows forcefully into a special device to build up the muscles used when breathing out. Twelve of the 13 participants completed the study. The trial was measuring whether this training changed how strongly participants could breathe out, how forcefully they could cough, and certain aspects of their speech — such as how many words they could say in one breath and where they paused to breathe while reading aloud. The training was delivered via telehealth (online video appointments), and the trial also asked participants how satisfied they were with that format. The reported data shows the following before and after training figures. For breathing-out muscle strength (measured in units called cmH2O — essentially a measure of air pressure), the reported average went from 66.10 before training to 83.75 afterwards. For cough strength (measured by how fast air moves during a cough, in litres per minute), the reported average went from 265.97 to 273.47. For speech, the average number of syllables said in one breath went from 10.76 to 10.39, and the average number of breathing pauses while reading went from 15.89 to 16.75. Regarding where participants paused to breathe during reading, the reported data shows figures around 47% and 46% for pauses at natural sentence boundaries, and around 9% and 12% for pauses at less natural points in a sentence, before and after training respectively. For telehealth satisfaction, the reported data shows an average score of 60.58 out of a possible 75, suggesting participants generally rated their telehealth experience positively on this scale. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03456882 · results posted 21 April 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT03456882) enrolled 147 people with ALS (motor neurone disease) — 74 received an investigational treatment called RNS60 and 73 received normal saline (a saltwater solution used as a comparison). All participants were also taking riluzole, a standard ALS medication. The trial set out to measure how RNS60 affected certain biological markers in the blood — substances that researchers use to track what is happening inside the body at a cellular level. Of those who started, 60 in the RNS60 group and 50 in the saline group completed the study. The reported data shows changes over the treatment period in six biological markers, measured in units called pg/ml (picograms per millilitre — a very small unit of concentration). For MCP-1, the reported change was 4.6 in the RNS60 group versus 2.2 in the saline group. For Cyp-A, the figures were 6.6 versus 6.9. For Actin-NT, the reported values were 9.4 versus 2.9. For 3-NT, the figures were 14.3 in the RNS60 group and −2.4 in the saline group (a negative number meaning the marker went down in that group). For IL-17, the reported change was 0.12 versus 0.39. Finally, for Nfl (a marker linked to nerve cell activity), the reported change was 2.7 in the RNS60 group versus 5.6 in the saline group. No secondary outcome data was included in the submitted results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03948178 · results posted 9 March 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT03948178) enrolled 227 people, all of whom received a medicine called levosimendan. The trial was measuring a number of things related to safety and body function over time, including adverse events (unwanted health events that occurred during the study), heart rate, heart electrical activity (via ECG recordings), lung breathing capacity, a standard ALS function score, and whether participants withdrew from the study due to their disease getting worse. The reported data shows that none of the 227 participants were recorded as having "completed" the trial under the study's own definitions, though this does not necessarily mean anything unexpected — how studies define completion can vary. The reported data shows that 161 out of 227 participants experienced at least one adverse event during the study. For heart rate, a range of values were recorded at different time points, with the average (mean) resting heart rate reported as 75.8 beats per minute at one measured point, and changes from the starting point ranging from around 3.5 to 15.0 beats per minute across the various time points. For the ECG heart recordings, the number of participants with certain findings ranged from 11 to 69 depending on the specific measurement being reported. Regarding the disease progression secondary outcome, 164 participants withdrew from the study due to disease progression, and 30 did not. For breathing capacity (slow vital capacity), changes from the starting point ranged from about +2.1% to -7.3% of predicted normal across different time points and positions. The ALS respiratory function score (on a 0–12 scale where 12 is normal) showed a reported change of -1.2 points, with a reported average score of 9.4 at one recorded point. It is important to note that the data as submitted provides a series of numbers across multiple time points, but full context — such as which numbers correspond to which time points — was not always clearly labelled in the submitted data, so some figures cannot be described in further detail here. Any figures not fully described above were not reported in a way that allowed plain attribution. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04248465 · results posted 10 January 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT04248465) looked at a medicine called ravulizumab as a potential treatment for ALS (also known as motor neurone disease), a condition that progressively affects the nerves controlling movement and breathing. A total of 382 people took part — 255 received ravulizumab from the start, and 127 received a dummy treatment (placebo) before later switching to ravulizumab in an open-label extension phase. The main thing the trial was measuring was how participants' ability to carry out everyday functions changed over time, using a standard ALS rating scale (scored from 0 to 48, where a higher score means better function). The reported data shows that, on the main measure, both groups saw a decline in their functional scores from the start of the trial. The ravulizumab group's score fell by an average of 11.9 points, while the placebo group's score fell by an average of 10.6 points. On a secondary measure looking at how long people survived without needing permanent breathing assistance, the reported figures were 6.05 months for the ravulizumab group and 7.69 months for the placebo group. Lung capacity (the ability to breathe out slowly) declined by a similar amount in both groups — around 20–21 percentage points. A measure of muscle strength also declined in both groups, with the ravulizumab group recording a drop of 46.5% and the placebo group a drop of 53.4%. A blood marker linked to nerve damage (called neurofilament light chain) increased in both groups — by 91.5 units in the ravulizumab group and 73.1 units in the placebo group. Regarding unwanted health events during the trial, 204 of 255 people in the ravulizumab group and 108 of 127 in the placebo group experienced some kind of adverse event; serious adverse events were recorded in 41 and 24 people respectively; and 2 people in the ravulizumab group (and none in the placebo group) stopped taking the study drug due to an adverse event. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03411863 · results posted 25 October 2022

  According to the results reported on ClinicalTrials.gov, this trial (NCT03411863) enrolled two groups of participants: 8 people without any neurological disease and 11 people with Amyotrophic Lateral Sclerosis (ALS, also known as motor neurone disease). Of those who started, 5 people in the non-neurological group and 9 people in the ALS group completed the study. The trial was measuring electrical activity in a hand muscle (the muscle at the base of the thumb) in response to a brain stimulation technique, both at rest and during movement. The researchers were looking at whether adding electrical stimulation to the neck at different time points changed the muscle's response compared to brain stimulation alone. The reported data shows results from the "at rest" measurements across several different timing intervals (measured in milliseconds) between the two types of stimulation. The muscle responses are reported as a percentage change compared to using brain stimulation on its own. For the group without neurological disease, the reported percentage changes ranged from approximately −5.9% to +14.1% across the different timing intervals. For the ALS group, the reported percentage changes ranged from approximately −6.1% to +48.3% across those same intervals. Positive numbers indicate the muscle response was larger when both stimulations were combined, while negative numbers indicate the response was smaller compared to brain stimulation alone. The reported data shows that no results were recorded or submitted for the second primary outcome measure, which looked at muscle activity during active hand or wrist movements — that data was not reported. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01622088 · results posted 7 April 2022

  According to the results reported on ClinicalTrials.gov, this trial enrolled 616 people who all received dexpramipexole at a dose of 300 mg per day (taken as 150 mg twice daily). The trial was set up as a long-term monitoring study, meaning its primary focus was on tracking and recording any unwanted health events — sometimes called adverse events — that participants experienced while taking the study drug, rather than measuring whether the drug treated a particular condition. None of the 616 participants were recorded as having completed the study in the standard sense, as the study design meant all participants fell into the "not completed" category. The reported data shows that out of 616 participants, 454 reported at least one adverse event of any kind during the study. Of those, 152 experienced what was classified as a "serious adverse event" — meaning a health event considered more significant in nature. Thirty participants stopped taking the study treatment because of an adverse event. The reported data also shows that small numbers of participants had test results flagged as potentially noteworthy across routine health checks: for example, varying numbers of participants (ranging from 0 to 65 depending on the specific measurement) had blood, blood chemistry, or vital signs results — such as heart rate or blood pressure — recorded as potentially outside normal ranges. The labels describing exactly which specific measurements these individual numbers correspond to were not fully detailed in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04947436 · results posted 4 November 2021

  According to the results reported on ClinicalTrials.gov, this trial looked at three different airway clearance approaches for people with breathing difficulties: a vest-like device that vibrates the chest wall (High Frequency Chest Wall Oscillation), a machine that helps push air in and pull it out of the lungs (Mechanical Insufflation/Exsufflation), or a combination of both. A total of 28 people started the trial — 7 in the chest wall oscillation group, 10 in the insufflation/exsufflation group, and 11 in the combination group. The trial measured changes in respiratory (breathing-related) complications over six months, as well as how participants felt their overall condition had changed. Notably, only 10 of the 28 participants completed the study — 3, 2, and 5 in each group respectively — so a large proportion did not finish. The reported data shows that the main outcome — respiratory complication severity — was measured on a scale from 0 to 9, where a score of 9 means no complications and lower scores indicate more serious complications. The chest wall oscillation group averaged a score of 8.33, the insufflation/exsufflation group averaged 9.0, and the combination group averaged 7.8. For the secondary outcome, participants were asked how they felt their condition had changed since starting the study, rated on a scale of 1 (much worse) to 5 (much better). The reported data shows average scores of 3.33 for the chest wall oscillation group, 3.00 for the insufflation/exsufflation group, and 4.40 for the combination group — with a score of 3 roughly representing "no change." It is worth noting that the number of people who completed the trial was very small, and no additional context about why so many participants did not finish was reported in the data. The reported figures are averages across a small number of people, and no further statistical detail was provided in the submitted results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00326625 · results posted 20 October 2021

  According to the results reported on ClinicalTrials.gov, this trial looked at a drug called glatiramer acetate (at a 40mg dose) compared to a placebo (an inactive treatment) in people with ALS (also known as motor neurone disease). A total of 184 people were assigned to the glatiramer acetate group and 182 to the placebo group. The main thing the trial was measuring was how quickly participants' ability to carry out daily activities declined over time, using a standardised questionnaire called the ALSFRS-R, where a higher score means less disability. The trial also tracked how long it took before participants died, needed a breathing tube inserted into their throat (tracheostomy), or required permanent assistance with breathing. The reported data shows that, on average, participants in the glatiramer acetate group declined at a rate of about 1.05 points per month on the ALSFRS-R scale, while those in the placebo group declined at about 1.00 points per month. For the second outcome — tracking how long before death, tracheostomy, or permanent assisted ventilation occurred — the reported data shows that a median time to event (the point at which half the participants had experienced one of these outcomes) could not be calculated for either group, because fewer than half of all participants reached one of those endpoints during the study period. As a result, those figures are listed as not available. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03505021 · results posted 31 August 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT03505021) enrolled 496 people with ALS (also known as motor neurone disease) — 329 received levosimendan and 167 received a placebo (a dummy treatment with no active ingredient). Of those who started, 217 in the levosimendan group and 112 in the placebo group completed the study. The trial was primarily measuring whether levosimendan made a difference to breathing capacity (specifically a lung test called "slow vital capacity," measured lying down) over 12 weeks, compared to placebo. Several secondary measures were also tracked over 48 weeks, including survival, overall function, and breathing-related symptoms. The reported data shows that for the main outcome — change in breathing capacity expressed as a percentage of what would be normal for that person — the levosimendan group showed a change of approximately −6.7 percentage points from their starting level, while the placebo group showed a change of approximately −7.0 percentage points. For the combined measure of survival and overall function (ranked on a scale of 1 to 496, where higher is better), the levosimendan group scored an average rank of about 240 and the placebo group about 229. For the time until a respiratory (breathing-related) event occurred, the reported median was 90 days in the levosimendan group and 126 days in the placebo group. On a self-rated scale of how participants felt overall (0 = completely well, 100 = worst possible), the reported change from the start of the study was approximately +17 points for levosimendan and +13 points for placebo. The monthly rate of decline in the breathing-related portion of the function scale was reported as −0.191 for levosimendan and −0.205 for placebo. On a separate breathlessness rating (0–10, where negative means improvement), the levosimendan group reported −0.15 and the placebo group reported +0.12. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00931944 · results posted 16 August 2021

  According to the results reported on ClinicalTrials.gov, this trial enrolled 74 people, all of whom received a daily dose of 300 mg of the study drug KNS-760704. The trial was looking at a condition called ALS (also known as motor neurone disease). Of the 74 who started, 19 completed the study and 55 did not finish. The main things the trial was set up to measure were changes in blood test results, liver enzyme levels, heart tracing (ECG) readings, and vital signs (such as blood pressure and pulse) — essentially, the trial was tracking certain body measurements over time to see how they changed while participants were taking the study drug. The reported data shows that, among participants who had at least one measurable result after starting the drug, small numbers showed readings that were flagged as potentially noteworthy. For blood (haematology) tests, the numbers flagged across different measures ranged from 2 to 5 participants. For liver enzyme readings, the numbers ranged from 0 to 5 participants across the different tests measured. For heart tracings (ECGs), numbers flagged ranged from 1 to 5 participants across the various measurements taken. For vital signs, the reported figures varied more widely — for example, 26 participants had a flagged reading for one particular vital sign measure, while others had as few as 0. The trial also tracked participants' ability to function day-to-day using a standard ALS questionnaire (scored 0–48, where higher means better function). The reported data shows an average change of −2.48 points from the start to week 12, and −4.29 points from the start to week 24, meaning scores on average moved downward over time. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01281189 · results posted 7 June 2021

  According to the results reported on ClinicalTrials.gov, this trial enrolled 468 people in the placebo group and 474 people in the dexpramipexole group — 942 participants in total. The trial was studying a drug called dexpramipexole in people with ALS (also known as motor neurone disease). The main thing researchers were measuring was a combined score called the "Composite Assessment of Function and Survival" (CAFS), which ranked participants based on how long they survived and how much their ability to function day-to-day changed over 12 months — a higher rank meant a better outcome. The reported data shows that, on the CAFS ranking scale (which ran from 1 to 941, where higher numbers are better), the placebo group scored an average rank of 438.84 and the dexpramipexole group scored 441.76. For the individual parts of this score: around 17.2% of the placebo group and 16.0% of the dexpramipexole group had died by 12 months; and both groups showed a similar decline in their day-to-day function score over that time (a drop of about 13.4 points out of 48 in both groups). The reported data shows that for secondary measures tracked out to 18 months, 23.1% of the placebo group and 20.5% of the dexpramipexole group had died; 27.2% versus 22.3% had died or reached a significant breathing milestone; and 41.9% versus 36.5% had died or reached a reduced lung capacity threshold. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00537446 · results posted 27 November 2020

  According to the results reported on ClinicalTrials.gov, this trial involved 8 participants, and all 8 completed the study. The trial was looking at two different levels of ventilation support (described as "high-level" and "low-level") and comparing how each affected participants' breathing. Specifically, the study was measuring lung function, the strength of the breathing muscles, how well the body was exchanging gases (such as oxygen and carbon dioxide), and how breathless participants felt during each mode of ventilation. The reported data shows that, despite all 8 participants completing the trial, no numerical results were submitted to ClinicalTrials.gov for any of the three primary outcome measures — lung and breathing muscle function, gas exchange, or self-reported breathlessness. In other words, while the trial ran to completion, the actual measurement figures for each of these outcomes were not included in the data made available on ClinicalTrials.gov. Because no outcome numbers were reported, it is not possible to describe what the measurements showed for either the high-level or low-level ventilation groups. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03160898 · results posted 11 September 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT03160898) enrolled 458 people with ALS (amyotrophic lateral sclerosis, also known as motor neurone disease). Participants were divided into four groups: one group received a placebo (a dummy treatment with no active ingredient), while the other three groups received different doses of a drug called reldesemtiv — either 150 mg, 300 mg, or 450 mg, each taken twice a day. The trial ran for 12 weeks and was primarily measuring changes in lung breathing capacity, specifically a test called "slow vital capacity" (how much air a person can breathe out after taking the deepest breath possible), expressed as a percentage of what would be expected for someone of similar age, height, and sex. The reported data shows that for the main measurement — change in breathing capacity over 12 weeks — all groups showed a decline from their starting point. The placebo group declined by an average of 6.46 percentage points, while the reldesemtiv groups declined by 4.97 (150 mg), 4.62 (300 mg), and 4.58 (450 mg) percentage points respectively. For the secondary measurements, the reported data shows changes in an ALS disability rating scale (scored 0–48, where higher means better function): the placebo group's average score fell by 3.53 points, compared to falls of 2.40, 2.62, and 2.94 points in the three reldesemtiv groups. A third measure tracked the rate of muscle strength change over time — again, all groups showed a decline, with the placebo group declining at a rate of −0.1444 units per day, and the reldesemtiv groups at −0.1198, −0.1299, and −0.0956 units per day respectively. It is worth noting that across all three measures, every group — including those taking reldesemtiv — showed a decline from their starting point over the 12 weeks; the reported numbers simply reflect different amounts of decline between groups. No safety or side-effect data was included in the structured results submitted to ClinicalTrials.gov, so that information is not available here. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03068754 · results posted 3 September 2020

  According to the results reported on ClinicalTrials.gov, this trial enrolled 143 people living with ALS (amyotrophic lateral sclerosis, also known as motor neurone disease). During the main treatment period, 95 participants received Acthar Gel and 48 received a placebo (an inactive dummy treatment). The trial was primarily measuring changes in participants' physical functioning using a standardised questionnaire called the ALSFRS-R — a 12-item scale scored from 0 to 48, where higher scores indicate better physical function across areas such as movement, speech, swallowing, and breathing. A smaller follow-on phase (the Open Label Extension, or OLE) then gave Acthar Gel to 33 of those participants who chose to continue. The reported data shows that at the start of the main treatment period, average ALSFRS-R scores (assessed by phone) were 34.7 in the Acthar Gel group and 34.3 in the placebo group. By the end of that period, the reported average scores were 26.4 for the Acthar Gel group and 30.8 for the placebo group — meaning both groups recorded lower scores over time. For a secondary measure using in-person assessments of the same scale, starting scores were 35.8 (Acthar Gel) and 35.4 (placebo), ending at 28.0 and 30.9 respectively. Regarding adverse events (unwanted health events recorded during the study) in the main period, 74 participants in the Acthar Gel group and 40 in the placebo group experienced at least one adverse event; serious adverse events were recorded in 13 participants on Acthar Gel and 6 on placebo. Lung function measurements and OLE period scores were also reported, but because the numbers of participants who completed the full trial were small, the data should be interpreted with that context in mind. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03506425 · results posted 24 December 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT03506425) looked at a treatment called Triheptanoin in people with ALS (also known as motor neurone disease). A total of 15 people took part, split equally into three groups of five. Of those, 9 people completed the study (3 from Group 1, 2 from Group 2, and 4 from Group 3). The trial was mainly measuring whether the rate of decline on a standard ALS function scale changed during treatment compared to before treatment. It also looked at two other things: a brain chemical ratio measured by a specialised scan, and a marker of cellular stress measured in urine. The reported data shows that for the primary measure — the ALS Functional Rating Scale (a 0–48 score where higher means better day-to-day function) — the rate of decline before treatment was reported as approximately 0.39 points lost per month, and during treatment it was approximately 0.57 points lost per month, across the completed participants in Groups 1 and 2 combined. For the brain chemical ratio (NAA/Cr from the motor cortex, measured by brain scan), the reported change was 0.42 in Group 1 and 0.09 in Group 2. For the urine stress marker, the reported change was 0.33 ng/mg in Group 1, 0.32 ng/mg in Group 2, and 0.26 ng/mg in Group 3. No further context or comparison figures for these secondary measures were included in the submitted data. It is worth noting that with only 15 participants in total, this was a very small study, and the submitted results do not include any explanation of what these numbers mean in terms of the treatment's impact. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03293394 · results posted 22 November 2019

  According to the results reported on ClinicalTrials.gov, this trial enrolled 30 people with ALS (also known as motor neurone disease) — 20 received real transcranial direct current stimulation (tDCS, a technique that passes a very mild electrical current through the scalp) and 10 received a sham (inactive/pretend) version of the same procedure. All 30 completed the initial two-week treatment phase, and 24 (16 in the real tDCS group, 8 in the sham group) completed the six-month follow-up. The trial was measuring things like muscle strength, brain activity, everyday functioning, and self-rated quality of life, comparing the two groups over time. The reported data shows that for the main outcome — a muscle strength score (ranging from 0 to 100, where 100 means no impairment) — both groups started at 74.7. By the end of the two-week treatment, the real tDCS group scored 76.4 and the sham group 74.5; at three months these were 76.4 and 72.9 respectively; and at six months, 75.9 and 72.0. For everyday functioning (a scale from 0 to 40), both groups began at 31.5, with the real tDCS group sitting at 30.4 and the sham group at 29.7 at six months. For a self-rated quality-of-life scale (0 to 100, higher being better), the real tDCS group went from 51.6 at the start to 54.7 at six months, while the sham group went from 51.6 to 40.2. The reported data also shows results for a measure of brain activity (recorded in millivolts), where the real tDCS group's readings decreased from 1.0 at baseline to 0.6 at six months, while the sham group remained at 1.0 throughout. Additional quality-of-life questionnaire scores were also reported for both groups across all time points, with both groups showing relatively small changes from their shared starting points. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00868166 · results posted 15 February 2019

  According to the results reported on ClinicalTrials.gov, this trial enrolled 259 people who received olesoxime and 253 people who received a placebo (an inactive treatment used for comparison), giving a total of 512 participants. All participants had ALS (also known as motor neurone disease). The trial ran for 18 months and was measuring whether olesoxime had any effect on survival, physical functioning, and breathing capacity compared to the placebo. Around 147 people in the olesoxime group and 139 in the placebo group completed the full study period. The reported data shows that the 18-month survival rate — the primary thing the trial was designed to measure — was 67.5% in the olesoxime group and 69.4% in the placebo group. For the secondary measures, the reported data shows that 67.1% of the olesoxime group and 65.5% of the placebo group reached a "failure" event, meaning they needed a breathing device (invasive or non-invasive ventilation) or a tracheostomy. Participants' ability to carry out daily activities was tracked using a standard ALS questionnaire scored from 0 (most impaired) to 48 (normal); scores in both groups declined over the 18 months in a broadly similar pattern. Breathing capacity and the proportion of participants whose scores fell below certain thresholds were also measured, and the reported figures were likewise similar between the two groups across the study period. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02447952 · results posted 26 December 2018

  According to the results reported on ClinicalTrials.gov, this trial enrolled 25 participants, all of whom used a monitoring device called the Mega Faros combined with a skin attachment called Fast Fix. The trial was measuring how long people actually wore the device during the day and night, and how they spent their time while wearing it — specifically how many minutes they were active, sitting still (but not lying down), or lying down. Of the 25 people who started, 18 completed the trial and 7 did not finish. The reported data shows that daytime wear time — the total minutes the device was worn during daylight hours — varied across different measurement points in the study, with average figures ranging from roughly 207 minutes up to around 821 minutes depending on the time point. Night-time wear time figures ranged from approximately 108 minutes to around 625 minutes across the different time points. Not all participants had data recorded at every time point, so the number of people contributing to each figure varied. For movement patterns, the reported data shows that average time spent actively moving per recording period ranged from about 12 to 34 minutes, time spent sitting still (but not lying) ranged from roughly 376 to 562 minutes, and time spent lying down ranged from approximately 50 to 136 minutes across the various time points measured. It is worth noting that the data as submitted does not include labels specifying which time point each individual figure corresponds to, so a full picture of how wear time changed across the study cannot be drawn from the available information. The trial appears to have been primarily focused on understanding how the device was worn in practice, rather than measuring a health outcome. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02709330 · results posted 13 December 2018

  According to the results reported on ClinicalTrials.gov, this trial looked at a supplement regimen containing lunasin in people living with ALS (also known as motor neurone disease). Sixty people took part in total — 50 in the lunasin group and 10 in a control group (people with ALS who did not take lunasin). Of the 50 people in the lunasin group, 37 completed the full study, while all 10 in the control group finished. The trial was primarily measuring how quickly participants' everyday physical functioning changed over time, using a standard ALS questionnaire called the ALSFRS-R, which scores a person's ability to carry out 13 daily activities on a scale from 0 (unable to do) to 52 (full ability). The reported data shows that, in the lunasin group, the average change in the functioning score was 0.44 points per month. The data does not include a comparison figure from the control group for this primary measure, so a direct comparison cannot be drawn from what was reported. For one of the secondary measures — looking at a biological marker in the blood called H3 histone acetylation (a chemical tag on the DNA packaging inside cells) — the reported figures showed the lunasin group's levels were at about 99% of their starting level after one month, compared to about 125% in the ALS control group and about 180% in healthy people not taking lunasin. The reported data also shows that none of the participants in the lunasin group (0%) experienced a meaningful and lasting improvement in their functioning score (defined as a gain of at least 4 points lasting at least 12 months). The reported data also shows some practical details about how the trial ran: participants were enrolled at a rate of about 9 people per month, and 84% of those still alive at the end of the study completed the final 12-month visit. Self-reported weights by participants matched those recorded by study staff very closely, at around 99% agreement at both check-in points. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00424463 · results posted 4 June 2018

  According to the results reported on ClinicalTrials.gov, this trial involved 181 people with ALS (also known as motor neurone disease) across three groups. The trial was testing a drug called edaravone (also referred to as MCI-186) against a placebo (a dummy treatment with no active ingredient). Participants were assigned to one of three sequences: placebo first then edaravone, edaravone first then placebo, or edaravone for the full duration. The main thing being measured was how participants' everyday physical functioning changed over 24 weeks, using a standard ALS rating scale where 0 is the worst possible score and 48 is the best. The reported data shows that all three groups saw a decline in their functioning scores from their starting point over the 24-week period. The group that received edaravone first and then switched to placebo declined by an average of 5.5 points on the scale; the group that received edaravone throughout declined by an average of 4.2 points; and the group that received placebo first and then switched to edaravone declined by an average of 5.4 points. For breathing capacity (measured as a percentage of normal lung function), all three groups also showed declines, ranging from around 10.5 to 12.9 percentage points. Regarding adverse events (any unwanted health occurrences noted during the trial), the reported data shows these were recorded in approximately 91.7% to 97.8% of participants across the three groups, though the data does not allow conclusions to be drawn about cause. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01232738 · results posted 18 May 2018

  According to the results reported on ClinicalTrials.gov, this trial enrolled 36 people with ALS (a serious disease affecting the nerves that control movement) who were given a drug called rasagiline. Of the 36 who started, 23 completed the trial and 13 did not finish. The trial was measuring two main things: how quickly participants' day-to-day physical functioning declined over time (using a standardised questionnaire called the ALSFRS-R, where a score of 48 means fully normal function and 0 means no function at all), and how long it took before a serious health event occurred — such as death, needing a breathing tube, or requiring almost constant ventilator support. The rasagiline group's results were compared against a historical group — meaning data from patients in past clinical trials, rather than a separate group running alongside this one. The reported data shows that on the functioning scale, participants taking rasagiline declined at a rate of approximately −1.20 points per month, while the historical comparison group declined at approximately −0.94 points per month. For the secondary outcome, the reported median time until a serious health event occurred was approximately 0.94 years in the rasagiline group, compared with approximately 0.90 years in the historical comparison group. The trial also tracked several biological measurements in the blood related to how cells' energy-producing parts (mitochondria) were functioning; changes in these markers over 12 months were reported, though the clinical meaning of those specific numbers was not explained further in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00330681 · results posted 17 May 2017

  According to the results reported on ClinicalTrials.gov, this trial involved 206 people with ALS (also known as motor neurone disease) — 102 received a treatment called MCI-186 (also known as edaravone) and 104 received a placebo (a dummy treatment with no active ingredient). The trial ran for 24 weeks and was primarily measuring changes in everyday functioning using a standard ALS rating scale called the ALSFRS-R, where a score of 48 means the best possible function and 0 means the worst. The reported data shows that on the main measure — the ALSFRS-R functional rating scale — both groups declined over the 24 weeks. The MCI-186 group's score dropped by an average of 5.7 points from their starting score, while the placebo group's score dropped by an average of 6.35 points. For the secondary measures, the reported data shows the MCI-186 group had an average decline of 14.57 percentage points in a breathing capacity test (called forced vital capacity, or FVC), compared to 17.49 points in the placebo group. On a movement disorder scale (Modified Norris Scale, where higher is better), the MCI-186 group dropped by an average of 14.12 points versus 16.15 points in the placebo group. A quality-of-life questionnaire showed a similar worsening in both groups (19.6 points in the MCI-186 group versus 19.13 in the placebo group). Regarding disease milestones such as death or major loss of function, small numbers of participants in both groups reached these points, with the reported data showing broadly similar counts across most categories. The reported data also shows that 89.2% of participants in the MCI-186 group and 88.5% in the placebo group experienced at least one adverse event (an unwanted or unexpected health occurrence noted during the trial). These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01753076 · results posted 19 April 2017

  According to the results reported on ClinicalTrials.gov, this trial enrolled 303 people with ALS (also known as motor neurone disease) — 151 received a placebo (an inactive infusion) and 152 received a drug called ozanezumab at a dose of 15 mg/kg. The trial ran for 48 weeks and was designed to measure two main things: how well participants could carry out everyday activities (using a questionnaire called the ALSFRS-R, which scores physical function from 0 to 48, where higher is better), and whether participants were still alive at 48 weeks. These two measures were combined into a single "joint rank score" — a way of comparing each participant against every other participant to produce an overall ranking. The reported data shows that for the primary combined measure, the placebo group received an average joint rank score of 15.0, while the ozanezumab group received a score of −14.9. For the secondary measures, the reported data shows that ALSFRS-R scores declined by an average of 9.1 points in the placebo group and 10.4 points in the ozanezumab group over 48 weeks. The reported monthly rate of decline in that score was −0.84 points per month for placebo and −0.96 for ozanezumab. Lung capacity (slow vital capacity) declined by an average of 0.899 litres in the placebo group and 1.026 litres in the ozanezumab group. Muscle strength (measured by a handheld device) declined by an average of 34.7% in the placebo group and 42.9% in the ozanezumab group. On a global improvement rating scale, 23 participants in the placebo group and 18 in the ozanezumab group were classed as "responders" at week 48. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01906658 · results posted 6 January 2017

  According to the results reported on ClinicalTrials.gov, this trial (NCT01906658) tested four different doses and schedules of a medicine called Acthar, given by injection under the skin. A total of 43 people took part across the four groups: 11 received the highest dose (80 units) twice a week, 11 received 24 units daily, 10 received 56 units twice a week, and 11 received the lowest dose (16 units) daily. The trial's main focus was on tracking certain side effects — specifically, how many participants experienced unwanted effects serious enough to require them to stop taking the study drug, or that could not be brought under control with other medicines. The reported data shows that for the primary measure, 2 out of 11 participants in the 80-unit twice-weekly group and 2 out of 10 in the 56-unit twice-weekly group had side effects meeting this threshold, while no participants in either of the daily-dose groups did. When these were broken down further as secondary measures, the reported data shows those same 2 participants in each of the two twice-weekly groups needed to stop the study drug, and no participants in any group had side effects that could not be controlled with other medicines. A separate secondary measure tracked the number of participants who showed signs of suicidal thoughts or behaviour that emerged during the study: 1 out of 11 in the 80-unit twice-weekly group and 3 out of 11 in the 24-unit daily group were reported to have experienced this, while none were reported in the other two groups. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01154283 · results posted 2 January 2017

  According to the results reported on ClinicalTrials.gov, this trial enrolled 28 participants in total — 14 in each group. It used a "crossover" design, meaning everyone tried both types of breathing support machines (called NIPPV): one called "bi-level, standard NIPPV" and one called "IPAP-only NIPPV." Each person used one type for six weeks, then switched to the other type for another six weeks. The trial was measuring how many hours per week participants used the machines, which type they preferred, any changes in their breathing difficulty, and their self-rated quality of life. Not everyone completed both periods — by the end, 16 of the 28 participants had finished the full 12 weeks. The reported data shows that, on average, participants used the bi-level machine for about 49.9 hours per week, compared with about 45.8 hours per week for the IPAP-only machine. When asked which type they preferred, 12 out of the participants who answered said they definitely or probably preferred the IPAP-only machine, while 2 said they definitely or probably preferred the bi-level machine. For breathing difficulty, the trial used a scoring scale running from −9 (major worsening) to +9 (major improvement); the reported average scores were −3.14 for the bi-level period and −1.21 for the IPAP-only period, both indicating some worsening was reported during each period. For self-rated quality of life (on a scale of 0 to 100, where 100 is the best imaginable health), the reported average score was about 52.9 during the bi-level period and about 62.8 during the IPAP-only period. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01363401 · results posted 20 July 2016

  According to the results reported on ClinicalTrials.gov, this trial looked at a treatment called HYNR-CS Inj. for people living with ALS (also known as Lou Gehrig's disease), a serious condition that progressively affects movement and muscle control. A total of 72 people started the trial — 41 in the treatment group and 31 in the no-treatment (control) group. The trial tracked participants over 16 weeks after the first injection, measuring changes in their ability to perform everyday functions, breathing capacity, and self-reported quality of life. The reported data shows that the main thing being measured was a functional rating score (called ALSFRS-R), where a higher score means better ability to carry out daily activities, with 48 being the best possible score. Over the 16-week period, the treatment group's score changed by an average of −1.36 points (a small decline), while the no-treatment group's score changed by an average of −4.67 points (a larger decline). For a secondary measure called the Appel Scale — where a higher number means greater disability — the treatment group's score increased by an average of 8.97 points, compared to 17.96 points in the no-treatment group. For breathing capacity (measured as a percentage of what would be expected for a healthy person of similar age), both groups showed a similar decline: −10.51% in the treatment group and −10.75% in the no-treatment group. A quality-of-life questionnaire (SF-36, scored 0–100 where higher means better) showed a change of −8.59 points in the treatment group and −11.83 points in the no-treatment group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00860951 · results posted 11 June 2015

  According to the results reported on ClinicalTrials.gov, this trial looked at a Brain Computer Interface (BCI) keyboard — a device that allows a person to type using brain signals rather than physical movement. The trial enrolled 29 participants, of whom 22 completed the study. The remaining 7 did not finish. The trial was measuring how accurately people could type using this BCI keyboard, compared to typing in two other settings: a standard computer environment and an assistive technology environment (meaning other existing tools designed to help people with physical limitations). The reported data shows that accuracy was measured as the percentage of typed characters that matched the intended characters in a sentence-copying task. Each participant completed typing sessions on separate days, with three sessions in total across three different environments. According to the results reported on ClinicalTrials.gov, the BCI keyboard environment recorded an average accuracy of 85%, the standard computer environment recorded 83%, and the assistive technology environment also recorded 83%. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00877604 · results posted 26 November 2014

  According to the results reported on ClinicalTrials.gov, this trial looked at a substance called TUDCA compared to a placebo (a dummy treatment with no active ingredient) in people with Amyotrophic Lateral Sclerosis (ALS), sometimes known as Motor Neurone Disease. A total of 34 people took part — 17 in the TUDCA group and 17 in the placebo group. The main thing the trial was measuring was how many people in each group showed a meaningful improvement (at least 15%) in their rate of decline on a standard ALS function rating scale (called the ALSFRS-R), which tracks things like movement, speech, and breathing ability. The reported data shows that in the TUDCA group, 13 out of 17 participants met the definition of a "responder" — meaning their rate of decline on the rating scale improved by at least 15% compared to before the treatment period. In the placebo group, 6 out of 17 participants were counted as responders by the same measure. Fifteen people in the TUDCA group and 14 in the placebo group completed the trial, with a small number not finishing in each group. For the secondary outcomes — which included breathing capacity, quality of life, time to needing a breathing tube, survival time, and overall function score at the end of the study — the reported data shows no figures were submitted to ClinicalTrials.gov, so those results are not available here. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00349622 · results posted 1 April 2014

  According to the results reported on ClinicalTrials.gov, this trial looked at a drug called ceftriaxone (an antibiotic) in people with ALS (also known as Motor Neurone Disease). A total of 513 people took part — 340 in the ceftriaxone group and 173 in a placebo (dummy treatment) group. The trial measured two main things: how long participants survived without needing a permanent breathing machine or tracheostomy, and how much their everyday functioning changed over one year using a standard ALS rating scale (scored 0–48, where 48 means full function). The reported data shows that, for survival, the median time — meaning the point by which half the participants in each group had reached the defined endpoint — was 664 days for the ceftriaxone group and 581 days for the placebo group. For the functioning scale, both groups declined over the year at a similar rate: the ceftriaxone group dropped by about 1.13 points every 8 weeks, while the placebo group dropped by about 1.22 points every 8 weeks. The reported data also shows results for several secondary measures — breathing capacity, muscle strength in the arms and legs, and quality of life — and in each case both groups showed gradual declines over the year at broadly similar rates, with no large numerical differences between the two groups reported. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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Not medical advice. These are results as reported by the trial sponsor to a public registry, summarised in plain English. They describe what a trial recorded, not whether a treatment is suitable for any person. Verify anything important against the original registry record and discuss what it means for you with your doctor.