Reported trial results for Gaucher Disease

19 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

• NCT05529992 · results posted 26 February 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT05529992) enrolled 20 people who received a treatment called VPRIV. Nineteen of the 20 participants completed the study, and one did not. The trial was primarily measuring how many participants experienced a serious unexpected medical event (called a serious treatment-emergent adverse event, or serious TEAE) — that is, an unwanted medical occurrence that happened after starting the treatment and was serious enough to cause death, be life-threatening, require hospitalisation, or result in lasting disability, among other criteria. The reported data shows that 20% of participants (roughly 4 out of 20 people) had at least one serious event of this kind — this was the primary thing the trial was tracking. Looking at broader medical events of any kind (not just serious ones), the reported data shows that 95% of participants experienced at least one treatment-emergent adverse event of any type. A smaller number — 5% of participants — had a reaction during or shortly after their infusion that was considered possibly or probably related to the treatment. Regarding antibodies (proteins the body can produce in response to a treatment), the reported data shows that approximately 15.8% of participants developed antibodies against VPRIV by week 53, and approximately 10.5% developed a specific type called neutralising antibodies. Some participants also showed clinically significant changes in blood and urine tests at week 53, though the detailed breakdown of those figures was not reported with full category labels in the data 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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• NCT03625882 · results posted 10 January 2025

  According to the results reported on ClinicalTrials.gov, this trial enrolled 63 participants who received intravenous infusions of a medicine called velaglucerase alfa. Sixty participants completed the study, while three did not. The trial was measuring how many participants experienced unwanted medical events (called adverse events) after starting treatment, and also tracked several body measurements — including red blood cell levels (haemoglobin), platelet counts (tiny blood cells that help with clotting), liver and spleen size, and bone density — to see how these changed over the course of the study. The reported data shows that, out of 63 participants, 41 experienced at least one adverse event (an unwanted sign, symptom, or test result that occurred during the study period), and 27 experienced a serious adverse event (one involving hospitalisation, a life-threatening situation, significant disability, or another major medical concern). For the secondary measurements, participants were grouped into three response categories — "good," "moderate," or "ineffective" — based on how much their results changed. For haemoglobin, 11 participants fell into the "good" category, 4 into "moderate," and 20 into "ineffective." For platelet counts, 9 were "good," 6 "moderate," and 4 "ineffective." For liver size, 0 were "good," 1 "moderate," and 6 "ineffective." For spleen size, 3 were "good," 2 "moderate," and 3 "ineffective." It is worth noting that not all 63 participants appear in every category, which may reflect missing data that was not reported. The reported data also shows bone density scores (a measure comparing a person's bone density to a young adult average, where zero is average and negative numbers mean lower than average). For the lower spine, the average score was -4.30 at the start of the study and -0.30 at the last recorded measurement. For the hip (femur neck), it was -2.90 at the start and -0.60 at the last measurement. The reported data does not include the number of participants measured for bone density at each time point, so the full picture of this result cannot be described 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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• NCT04145037 · results posted 18 January 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT04145037) looked at a gene therapy called AVR-RD-02, which was being studied in people with Gaucher disease — a condition where the body cannot properly break down certain fatty substances. The trial enrolled two groups: people who had been stable on existing enzyme replacement therapy ("Switch Stable") and people who had never received treatment ("Treatment-naïve"). Eight people started in the Switch Stable group and none started in the Treatment-naïve group. Of those eight, six went through the cell collection process, five received the gene therapy infusion, and four completed the study. The reported data shows several things were measured. For safety-related events (called adverse events and serious adverse events — meaning any unwanted medical occurrences), the data reports zero clinically significant adverse events and zero serious adverse events directly linked to the gene therapy infusion, but 2 events in another category and 189 events overall across all categories (including those related to the preparatory medicines, procedures, and the underlying disease). On a measure called "vector copy number" in blood — essentially an estimate of how many copies of the inserted gene were present per cell on average — the reported figure was 0.55 copies per cell (a value of 1 would mean roughly one copy per cell on average). The corresponding bone marrow measurement was not reported in the submitted data. The reported data also shows that spleen volume (measured by MRI scan) decreased by about 17% from the starting point, and liver volume decreased by about 5%. A measure of haemoglobin concentration (the protein in red blood cells that carries oxygen) showed a ratio of 1.00, meaning it remained essentially unchanged from the starting level at the 52-week point. It is worth noting that this was a very small study with only a handful of participants completing it, and no participants were enrolled in the Treatment-naïve group, so the numbers reflect a limited dataset. 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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• NCT04718779 · results posted 21 December 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT04718779) enrolled a very small number of participants — just 2 people in a prospective group (followed forward in time) and 2 people in a retrospective group (reviewed using past medical records), giving a total of 4 participants. All 4 completed the study. The trial was measuring whether certain key blood and organ markers stayed stable over 12 months in people with Gaucher disease who were receiving treatment. Specifically, it tracked red blood cell levels (haemoglobin), platelet counts, and the size of the liver and spleen. The reported data shows that, for the two measures where results were provided, all participants in both groups met the stability thresholds set by the researchers. All 2 participants in the prospective group and all 2 in the retrospective group had haemoglobin levels that did not fall by 1.5 grams per decilitre or more from their starting point over 12 months. Similarly, all 4 participants had platelet counts that did not drop by 25% or more from their starting point. For liver volume and spleen volume stability — the other two primary measures — no results data was reported on ClinicalTrials.gov, so those figures are not available. Regarding unwanted medical events (adverse events and serious adverse events), the reported data shows that zero participants in either group experienced any such events during the study. It is worth noting that with only 4 participants in total, this was an extremely small study, and the numbers above reflect just those 4 individuals. 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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• NCT00813865 · results posted 15 August 2018

  According to the results reported on ClinicalTrials.gov, this trial (NCT00813865) enrolled 8 participants, all of whom received a medicine called afegostat tartrate. The trial was measuring the number of participants who experienced severe side effects (called severe treatment-emergent adverse events — meaning serious unwanted health events that appeared or got worse after starting the study drug), as well as changes in the size of the spleen and liver, measured using MRI scans. Only 1 of the 8 participants completed the study, while 7 did not complete it; the reasons for this are not detailed in the reported data. The reported data shows that none of the 8 participants (0 out of 8) experienced a severe treatment-emergent adverse event during the study period. Regarding organ size, the reported data shows an average change in spleen volume of minus 138 cubic centimetres from the start of the study to the end of treatment — a negative number meaning the spleen was smaller on average at the end than at the beginning. For the liver, the reported data shows an average change of plus 57 cubic centimetres, meaning liver volume was on average slightly larger at the end of treatment compared to the start. It is important to note that with only 8 participants and just 1 completing the study, these figures are based on 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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• NCT02770625 · results posted 12 July 2017

  According to the results reported on ClinicalTrials.gov, this trial (NCT02770625) involved 8 participants, all of whom completed the study. All participants received the treatment being studied, called ISU302. The trial was measuring a range of things in the blood, liver, and spleen — including levels of haemoglobin (a protein in red blood cells that carries oxygen), platelet counts (platelets are tiny blood cells that help with clotting), spleen and liver size, and two substances in the blood called angiotensin-converting enzyme and chitotriosidase, which can act as markers of certain conditions. The reported data shows two sets of measurements for each outcome — these appear to represent readings taken at two different points in time (for example, before and after treatment), though the exact timing of each measurement was not specified in the submitted data. For haemoglobin, the reported figures were 9.49 g/dL and 11.44 g/dL. For platelet counts, the figures were 132,600 and 180,300 platelets per microlitre of blood. Spleen volume was reported as 29,047 millilitres and 15,207 millilitres across the two time points. Liver volume was reported as 1.530 and 1.537 (in relative units). The angiotensin-converting enzyme level went from 195.71 to 159.31 U/L (units per litre), and the chitotriosidase level went from 11,093.67 to 3,409.63 nmol/mL/hr. It is worth noting that because only 8 people took part, these numbers represent a very small group, and no comparison group (such as a placebo group) appears to have been included in the reported data. The reported data shows only what was measured in this single group of participants, and no conclusions about why these numbers changed can be drawn from the figures alone. 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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• NCT01074944 · results posted 2 December 2016

  According to the results reported on ClinicalTrials.gov, this trial tested a medicine called eliglustat in people with Gaucher disease (a rare condition where a fatty substance builds up in organs and bones). The trial ran in several stages. First, 170 participants went through a lead-in period of up to 78 weeks where they all received eliglustat. Then 131 of those participants moved into a main comparison period of up to 52 weeks, where they were randomly assigned to receive eliglustat either once a day (65 people) or twice a day (66 people). After that, 121 participants continued into a longer treatment phase, and 25 went on to an extended phase of up to 42 months. The main thing being measured in the comparison period was whether participants stayed "stable" — meaning their bone health, blood counts, and organ sizes did not worsen beyond set limits over 52 weeks. The reported data shows that in the main 52-week comparison period, 80.4% of participants in the once-daily group and 83.1% in the twice-daily group met the criteria for remaining stable. For the secondary measurements, haemoglobin levels (a measure of red blood cells) stayed broadly similar across both groups from the start to weeks 26 and 52, ranging between roughly 13.6 and 13.9 g/dL. Platelet counts (cells that help blood clot) also remained in a similar range throughout. Spleen volume, measured as a multiple of what is considered a normal spleen size, appeared to show small reductions from the starting point in both groups over the 52 weeks. Liver volume figures also remained relatively steady. A blood marker called chitotriosidase — which can be elevated in Gaucher disease — showed lower reported values at weeks 26 and 52 compared to the starting values in both groups. The reported data shows the numbers across both dosing groups were broadly comparable on these measures, though exact differences between groups were not broken down further in the submitted results. Some participants did not complete each phase of the trial, and the reasons were not detailed in the data provided 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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• NCT01842841 · results posted 14 December 2015

  According to the results reported on ClinicalTrials.gov, this trial (NCT01842841) enrolled 5 participants who were already receiving velaglucerase alfa (VPRIV®), a treatment for Gaucher disease, in an earlier related study. All 5 participants completed the trial — none dropped out. The trial was measuring a range of things, including any unwanted health events (called adverse events), whether participants developed antibodies against the study drug, laboratory test results, and changes in two blood measurements: haemoglobin (a protein in red blood cells) and platelet count (tiny blood cells that help with clotting). The reported data shows that, of the 5 participants, 2 experienced adverse events that were considered related to the drug, and 2 experienced serious adverse events (meaning events involving things like hospitalisation or other significant outcomes). No participants experienced adverse events specifically linked to the infusion process itself. One participant had laboratory test results that were considered abnormal and clinically significant by the study investigators. Notably, none of the 5 participants developed antibodies against the study drug. All 5 participants used other medications alongside the study treatment during the trial. The reported data also shows changes in blood measurements from the start of the earlier linked study to week 101 of this follow-on study. On average, haemoglobin levels changed by +0.22 grams per decilitre, and platelet counts changed by +9.8 (×10⁹ per litre). These are the average changes across the group — no individual figures were 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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• NCT01132690 · results posted 11 November 2014

  According to the results reported on ClinicalTrials.gov, this trial enrolled 11 people in total — 6 received a lower dose of the study treatment (30 Units per kilogram of body weight) and 5 received a higher dose (60 Units per kilogram). All 11 participants completed the trial, with no drop-outs recorded. The trial was measuring several things in the blood and organs, including haemoglobin levels (a measure of red blood cells), platelet counts (tiny blood cells involved in clotting), the size of the spleen and liver (organs often affected in the condition being studied), and levels of two biological markers in the blood — chitotriosidase and CCL18 — which can reflect disease activity. The reported data shows that for the primary measure, haemoglobin, the change from starting levels varied across time points: in the lower-dose group the reported median changes were 1.2, 1.1, 0.9, and 1.4 g/dL across the measurement periods, while in the higher-dose group they were 0.4, 1.5, 1.5, and 1.6 g/dL. For spleen volume, the lower-dose group started at a reported average of 1,218 mL and was later measured at 811.6 mL; the higher-dose group started at 1,023 mL and was later measured at 524.0 mL. Liver volume in the lower-dose group went from 1,214 mL to 1,116 mL, and in the higher-dose group from 991.7 mL to 849.1 mL. Platelet counts appeared to rise in both groups over time. The two biological markers — chitotriosidase and CCL18 — both showed percentage decreases from starting levels in both groups across all time points, with reported reductions ranging roughly from around 18% to over 66% depending on the marker, group, and time point. 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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• NCT00943111 · results posted 4 September 2014

  According to the results reported on ClinicalTrials.gov, this trial involved people with Gaucher disease who were already being treated with a medicine called imiglucerase. The trial had two main phases. In the first phase (the Primary Analysis Period, lasting 52 weeks), 106 participants were switched to a tablet medicine called eliglustat and 54 continued on imiglucerase, for comparison. In the second, longer phase (up to 5 years), 152 participants received eliglustat. The trial was primarily measuring how many participants stayed "stable" — meaning their blood measures (haemoglobin levels and platelet counts) and organ sizes (spleen and liver) did not worsen beyond set thresholds. The reported data shows that during the 52-week period, 84.8% of participants taking eliglustat and 93.6% of participants taking imiglucerase met the criteria for remaining stable. During the longer 5-year phase, the percentage of eliglustat participants reported as remaining stable each year was 83.6% at year one, 75.65% at year two, 60.53% at year three, and 26.97% at year four. The trial also measured bone density using a scoring system that compares a person's bone strength to that of a healthy young adult (called a T-score and a Z-score). The reported data shows that changes in these bone scores from the start of the trial to week 52 were very small in both groups — generally less than a tenth of a point in either direction — across measurements of the spine and femur (thigh bone). 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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• NCT00891202 · results posted 3 September 2014

  According to the results reported on ClinicalTrials.gov, this trial involved 40 adults who took part in the main comparison phase (called the Primary Analysis Period, or PAP), with 20 receiving the study drug eliglustat and 20 receiving a placebo (a dummy treatment with no active ingredient). The trial was looking at whether eliglustat had any effect on several body measurements in people with Gaucher disease, including the size of the spleen and liver, haemoglobin levels (a measure of red blood cells in the blood), and platelet counts (tiny blood cells involved in clotting). After this phase ended at around nine months (Week 39), most participants continued into a longer follow-up period of several more years, where everyone received eliglustat. The reported data shows that in the main phase, the spleen volume in the eliglustat group decreased by about 27.8% on average, while in the placebo group it increased by about 2.3%. For liver volume, the eliglustat group showed an average decrease of about 5.2%, compared to an average increase of about 1.4% in the placebo group. Platelet counts in the eliglustat group increased by an average of about 32%, while the placebo group saw an average decrease of about 9%. For haemoglobin levels, the eliglustat group showed an average increase of 0.69 grams per decilitre (g/dL) from their starting point, while the placebo group showed an average decrease of 0.54 g/dL. The reported data also shows results from the longer follow-up period. At around Week 234 (roughly four and a half years into the study), participants who had been on eliglustat from the very beginning showed an average spleen volume reduction of about 66.9% from their original starting point. Those who had started on placebo and then switched to eliglustat showed an average spleen volume reduction of about 64.0% from when they began taking eliglustat. 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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• NCT00705939 · results posted 15 July 2014

  According to the results reported on ClinicalTrials.gov, this trial enrolled 44 people across three groups. Twelve participants received a lower dose of the study treatment (30 units per kilogram of body weight) and had not previously been treated for their condition — this group is called "Naive 30 Units/kg." Fourteen participants received a higher dose (60 units per kilogram) and were also treatment-naïve — the "Naive 60 Units/kg" group. The remaining 18 participants had previously been on a different treatment and switched to the study treatment — the "Switchover" group. The trial tracked spleen size and liver size (measured by MRI scan), as well as haemoglobin levels (a measure of red blood cells) and platelet counts (cells that help blood clot), across multiple time points. The reported data shows that spleen volume in the Naive 30 Units/kg group started at an average of 2,324 mL and was recorded at 1,708 mL, then 1,420 mL, and then 1,237 mL at later time points. For the Naive 60 Units/kg group, starting spleen volume was 2,120 mL, moving to 1,268 mL, 947 mL, and 761 mL over time. The Switchover group began at a lower average of 778 mL, with later readings of 884 mL, 609 mL, and 548 mL. For liver volume, the Naive 30 Units/kg group went from 3,000 mL to around 2,341 mL, the Naive 60 Units/kg group from 2,471 mL to 1,972 mL, and the Switchover group remained relatively stable, moving from 1,776 mL to 1,821 mL across time points. The reported data also shows that haemoglobin levels — which started at 12.5, 11.4, and 13.6 mg/dL respectively across the three groups — were recorded at 14.3, 14.1, and 13.3 mg/dL at the final time point. Platelet counts in the Naive 30 Units/kg group started at around 64,900 per cubic millimetre and were recorded at approximately 94,700 by the last time point; the Naive 60 Units/kg group went from about 69,000 to 144,400; and the Switchover group went from around 163,800 to 172,500. 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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• NCT00391625 · results posted 23 June 2014

  According to the results reported on ClinicalTrials.gov, this trial enrolled 10 participants, all of whom received a treatment referred to as GA-GCB. The trial ran over a long period — up to 84 months (seven years) — and was designed to track two things: any unwanted health events (called adverse events) that occurred during that time, and changes in certain body measurements such as blood counts, liver size, and spleen size. The reported data shows that all 10 participants experienced at least one adverse event over the course of the trial. One participant experienced an adverse event considered related to the drug, and one experienced an infusion-related adverse event (a reaction around the time of receiving the treatment). Two participants experienced at least one severe adverse event, and four experienced at least one serious adverse event (a more significant health concern requiring attention). No adverse events were reported as drug-related and severe, life-threatening, or drug-related and serious. No participants died or stopped the trial because of an adverse event. Regarding the body measurement results, the reported data shows that haemoglobin levels (a measure of red blood cells) were roughly 17–21% higher than baseline at each yearly check-in across the seven years. Platelet counts (tiny blood cells that help clotting) were reported as 78–140% higher than baseline, with the increase growing over the first few years before levelling off. Liver volume was reported as approximately 28–42% smaller than baseline across the measurement points, and spleen size was reported as approximately 67–78% smaller than baseline over the same 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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• NCT00712348 · results posted 5 June 2014

  According to the results reported on ClinicalTrials.gov, this trial enrolled 31 people who received a treatment called taliglucerase alfa. Thirty of the 31 participants completed the study, with one person not finishing. The trial was measuring several things in people's blood and organs, with the main focus being haemoglobin levels — haemoglobin is the protein in red blood cells that carries oxygen around the body. The study also tracked platelet counts (tiny blood cells that help with clotting), as well as the size of the spleen and liver, both of which can be affected in the condition being studied. The reported data shows that haemoglobin levels across the measurement points were 13.5, 13.3, 13.3, and 13.4 grams per decilitre (g/dL), a common unit for measuring haemoglobin in blood. For platelet counts, the reported figures across measurement points were approximately 161,137, 150,800, 157,586, and 161,167 platelets per cubic millimetre of blood. The reported data shows spleen volume, measured by MRI scan, was 720.5 millilitres at one time point and 654.7 millilitres at another. Liver volume was reported as 1,766 millilitres and 1,716 millilitres across two time points. The data does not include labels specifying exactly when each of these measurements was taken, so precise timing details were not reported in the structured 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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• NCT00635427 · results posted 28 January 2014

  According to the results reported on ClinicalTrials.gov, this trial (NCT00635427) was a long-term follow-on study looking at a medicine called velaglucerase alfa (VPRIV), which is given by drip infusion to people with Gaucher disease — a condition where a fatty substance builds up in the body. A total of 95 participants were enrolled across five groups, each coming from a different earlier ("parent") study. The groups differed by the dose of VPRIV they had previously received, or whether some had previously been on a different medicine called imiglucerase. The trial's main focus was on tracking safety-related events over time, and it also measured changes in four body markers: haemoglobin (a measure of red blood cells), platelet counts (cells that help blood clot), and the sizes of the liver and spleen. The reported data shows that, looking at unwanted health events that arose during treatment, the numbers varied across the three reporting groups. In terms of the body marker measurements at 24 months, the group that had previously been on VPRIV at 45 or 60 units per kilogram showed an average increase in haemoglobin of 2.75 g/dL, the group that had previously been on imiglucerase showed an average increase of 2.00 g/dL, and the group on a flexible VPRIV dose showed a very small average change of −0.05 g/dL. For platelet counts, the reported average changes were an increase of 87.85, 160.94, and 9.03 (in units of 10⁹/L) for those three groups respectively. Liver size (measured relative to body weight) showed average reductions of 1.206%, 1.688%, and 0.026% across the three groups. Spleen size showed average percentage reductions of 64.49%, 63.82%, and 8.04% respectively. It is worth noting that the trial itself states no formal comparisons or statistical tests were applied to these results, meaning the numbers are descriptive only and were not tested against each other in a standard scientific way. Completion rates also varied considerably — for example, only 1 of 13 participants in one group completed the study, while 30 of 38 completed in another, and the reasons for not completing were not detailed in the data provided 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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• NCT01427517 · results posted 2 August 2013

  According to the results reported on ClinicalTrials.gov, this trial enrolled nine people in total — three with Parkinson's disease (referred to as PD), three with Gaucher disease (referred to as GD), and three people without either condition who acted as a comparison group. All nine participants completed the study. The trial was measuring whether levels of a substance called glutathione (GSH) — a naturally occurring compound found in the brain — changed after participants received N-acetyl cysteine (NAC), a supplement. GSH levels were measured in the brain before and after NAC was given, roughly 90 to 110 minutes later. The reported data shows that brain GSH levels appeared to increase from the starting point in all three groups after NAC was administered. In the Parkinson's disease group, a 55% increase from baseline was reported. In the Gaucher disease group, the reported increase was 41%, and in the comparison (control) group, the reported increase was 34%. No other outcome measures were listed in the submitted results data. It is worth noting that this was a very small study — just three people per group — and the results as submitted to ClinicalTrials.gov reflect only what was measured at that single time point. 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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• NCT00376168 · results posted 26 July 2012

  According to the results reported on ClinicalTrials.gov, this trial looked at a medicine called ELELYSO given at two different doses — 30 units per kilogram of body weight, and 60 units per kilogram of body weight. A total of 32 adults took part, 16 in each dose group. The trial was measuring changes in spleen size (the spleen is an organ in the abdomen that can become enlarged in certain conditions), liver size, red blood cell levels, and platelet counts (platelets are tiny blood cells that help with clotting) over a period of 9 months. The reported data shows that for the main measurement — spleen size as seen on an MRI scan — the group receiving the lower dose (30 units/kg) had an average reduction of about 27%, while the group on the higher dose (60 units/kg) had an average reduction of about 38%. For the secondary measurements, the reported data shows liver size reduced by around 10–11% in both groups. Haemoglobin levels (a measure of red blood cells) increased on average by 1.6 g/dL in the lower-dose group and 2.2 g/dL in the higher-dose group. Platelet counts increased on average by approximately 11,400 per cubic millimetre in the lower-dose group and approximately 41,500 per cubic millimetre in the higher-dose group. The reported data also includes a measurement of a substance in the blood called chitotriosidase, which can be used as a marker of disease activity. The lower-dose group showed an average decrease of about 14,500 nmol/ml/hr and the higher-dose group showed an average decrease of about 12,500 nmol/ml/hr from the start of the trial to 9 months. 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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• NCT00430625 · results posted 10 September 2010

  According to the results reported on ClinicalTrials.gov, this trial involved 25 adults or children with Gaucher disease (a rare condition where a fatty substance builds up in the body's organs). Participants were split into two groups: 13 people received a lower dose of the study medicine, VPRIV® (velaglucerase alfa), at 45 units per kilogram of body weight, and 12 people received a higher dose at 60 units per kilogram. Both groups received the medicine through a drip into a vein every two weeks for 12 months. The trial was measuring changes in several body markers over that period, including the amount of haemoglobin (a protein in red blood cells that carries oxygen) in the blood, platelet counts (tiny blood cells that help with clotting), liver and spleen size, and levels of a body chemical called chitotriosidase. The reported data shows that, after 12 months, haemoglobin levels rose in both groups — by approximately 2.4 g/dL (grams per decilitre, a standard unit of measurement for blood) in both the lower-dose group (2.438 g/dL) and the higher-dose group (2.429 g/dL). Platelet counts also increased in both groups, with the lower-dose group rising by about 41 units and the higher-dose group by about 51 units. For liver size (measured as a proportion of body weight using MRI scans), the reported data shows a reduction of 0.30% in the lower-dose group and 0.84% in the higher-dose group. Spleen size similarly decreased, by 1.87% in the lower-dose group and 1.92% in the higher-dose group. The reported data also shows changes in chitotriosidase, a chemical in the blood sometimes used as a marker in Gaucher disease — but this was only measured in the subset of participants whose bodies naturally produce this chemical. In that subset, levels fell by around 61% in the lower-dose group and around 70% in the higher-dose group. It is worth noting that all 25 participants who started the trial completed it, and no dropout figures were 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.

  View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗

• NCT00478647 · results posted 2 September 2010

  According to the results reported on ClinicalTrials.gov, this trial enrolled 40 people, all of whom received a medicine called velaglucerase alfa. Thirty-eight participants completed the study, while two did not finish. The trial's main focus was on tracking unwanted or unexpected health events (called adverse events) experienced by participants over the course of the study, as well as monitoring things like heart tracings, blood tests, and whether participants developed antibodies to the medicine. The reported data shows that, out of 40 participants, 34 experienced at least one adverse event during the study — the results do not break down what those events were in this section, but further detail is noted to be available in the adverse events section of the trial record. For the secondary measurements, the reported data shows a very small average decrease in haemoglobin (a measure of red blood cells) of about 0.1 grams per decilitre by week 53. Platelet counts (tiny blood cells involved in clotting) showed an average increase of about 7% by week 53. Liver size, measured relative to body weight, showed almost no change (a decrease of 0.03%) by week 51. Spleen size, also measured relative to body weight, showed an average decrease of about 5.6% by week 51. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

  View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗

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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.