Reported trial results for Phenylketonuria
Every Phenylketonuria trial Voxsanity tracks that has reported results to a public registry, newest first. These trials have finished, so they are not recruiting. Not medical advice.
20 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
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NCT05270837 · results posted 21 July 2026
According to the results reported on ClinicalTrials.gov, this trial (NCT05270837) involved 55 people in total across two groups. Thirty-six participants received a medicine called pegvaliase, while 19 followed a diet-only plan as a comparison group. The trial was structured in two parts: the first ran to around 72 weeks, and a second part continued beyond that. The study was measuring changes in the level of a substance called phenylalanine (Phe) in the blood — Phe is an amino acid that builds up in people with the metabolic condition this trial was designed to study. The trial also tracked unwanted health events that occurred during the study, as well as how the body's immune system responded to the medicine by producing antibodies (proteins the body makes in response to a foreign substance). The reported data shows that, after 72 weeks, the pegvaliase group had an average reduction in blood Phe levels of approximately 450 units (µmol/L) from where they started, while the diet-only group had an average reduction of approximately 34 units. Regarding unwanted health events during Part 1, all 36 participants in the pegvaliase group experienced at least one treatment-emergent adverse event (a health event that appeared or got worse after starting the study), compared with all 17 participants in the diet-only group. Serious adverse events were reported in 9 out of 36 participants in the pegvaliase group; the reported data shows zero serious adverse events in the diet-only group during this period. For immune system responses, 100% of pegvaliase participants tested positive for antibodies against the medicine by the end of the study, compared with 22.2% at the start. Some other types of antibody responses also changed over the course of the study, with figures varying depending on the antibody type measured. 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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NCT05827536 · results posted 11 March 2026
According to the results reported on ClinicalTrials.gov, this trial (NCT05827536) involved 21 people with PKU (phenylketonuria — a condition where the body cannot properly break down a substance called phenylalanine, or "Phe", which can build up to harmful levels in the blood). It was a crossover trial, meaning each participant tried both treatments at different times: their usual standard of care (SoC) and a specialist dietary product called PKU GOLIKE PLUS. The trial's main focus was measuring Phe levels in the blood — checked using dried blood spot tests (a small drop of blood on a card) — at specific time points on the second day of each treatment period. Of the 21 people who started, between 12 and 13 completed both periods of the trial. The reported data shows that blood Phe levels were measured at several time points before eating and before taking the next dose of the product. For participants on their usual standard of care, the reported Phe readings across the different time points ranged from approximately 523 to 620 μmol/L (micromoles per litre — the unit used to measure how much Phe is in the blood). For participants using PKU GOLIKE PLUS, the reported Phe readings across the same types of time points ranged from approximately 505 to 553 μmol/L. These figures varied depending on which measurement point was used and which group the participants were in at the time of measurement. 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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NCT05487378 · results posted 8 June 2025
According to the results reported on ClinicalTrials.gov, this trial (NCT05487378) involved 16 people in total — 9 in one group and 7 in another. It was a crossover study, meaning each participant tried both products at different times: one group used a product called PKU GOLIKE first, then switched to a standard amino acid protein substitute, while the other group did it in the opposite order. The two treatment periods each lasted one week, with a two-week break in between. The trial was measuring levels of two substances in the blood — phenylalanine (Phe) and tyrosine (Tyr) — which are relevant to a condition called phenylketonuria (PKU), a rare inherited disorder affecting how the body processes certain proteins. The reported data shows that blood phenylalanine levels (measured in µmol/L — a standard unit for substance concentration in the blood) across the study were reported as four separate values: 357.5, 294.0, 346.8, and 442.4 µmol/L. For the secondary outcome, blood tyrosine levels were reported as 49.6, 62.1, 49.5, and 51.6 µmol/L. It is worth noting that the data as submitted does not clearly label which of these values corresponds to which treatment or time point, so a direct comparison between the two products cannot be drawn from the numbers as presented 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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NCT05764239 · results posted 20 June 2024
According to the results reported on ClinicalTrials.gov, this trial involved 35 participants in its first stage (called the "Dose Escalation Period" or DEP), where the investigational treatment SYNB1934v1 was given at increasing dose levels to find an appropriate dose. The trial was primarily measuring changes in blood levels of phenylalanine (phe) — an amino acid that builds up in people with a condition called phenylketonuria (PKU). Later stages involved smaller groups: 9 participants received the investigational treatment and 8 received a placebo (a dummy treatment) in a controlled comparison phase, and 10 participants entered a further open-label extension stage. The trial was tracking whether blood phenylalanine levels changed at different doses of the treatment. The reported data shows that in the first stage, at the lowest dose tested, blood phenylalanine levels fell by an average of about 3.85% (or around 55 µmol/L, a unit used to measure concentration in blood) after three weeks at that dose. At the middle dose, levels rose on average by about 5.33% (or 55 µmol/L), and at the highest dose, levels rose on average by about 3.35% (or 31 µmol/L). For a secondary measure — the number of participants who achieved at least a 20% reduction in blood phenylalanine at any point during the first stage — the reported data shows that 6 out of the participants in the highest dose group reached this threshold, while no participants in the two lower dose groups did. An overall figure for the entire first stage was 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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NCT05099640 · results posted 10 January 2024
According to the results reported on ClinicalTrials.gov, this trial (NCT05099640) tested a medicine called sepiapterin in people with phenylketonuria (PKU) — a condition where the body cannot properly break down a substance called phenylalanine (Phe), which can build up in the blood. The trial ran in two parts. In Part 1, 157 participants received sepiapterin for 14 days; 111 completed this stage. Those who showed a blood Phe reduction of at least 30% during Part 1 were then eligible for Part 2, a 6-week randomised stage where 56 participants received sepiapterin and 54 received a placebo (a dummy treatment with no active ingredient). The trial's main goal was to measure changes in blood Phe levels. The reported data shows that, for the main outcome measured at weeks 5 and 6 of Part 2, participants in the sepiapterin group had an average blood Phe level that was about 416 units (μmol/L) lower than their starting level, representing a decrease of around 63%. In the placebo group over the same period, blood Phe levels were largely unchanged, with the reported data showing an average decrease of about 20 units and a change of less than 1%. For the secondary outcomes, the reported data shows that among participants whose starting Phe levels were above 600 μmol/L, about 93% of the sepiapterin group reached levels below that threshold by weeks 5–6, compared with 30% in the placebo group. Among those starting above 360 μmol/L, about 84% of the sepiapterin group reached levels below that point, compared with around 9% 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.
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT02677870 · results posted 27 April 2022
According to the results reported on ClinicalTrials.gov, this trial involved 7 people in total — 4 in one group and 3 in another. It was a crossover study, meaning all participants went through multiple stages: a diet-only treatment period, two different doses of a medication called sapropterin dihydrochloride (a tablet used in the management of a condition affecting how the body processes a substance called phenylalanine, or Phe), and a "washout" break in between. The trial was looking at whether sapropterin or diet treatment could lower participants' blood phenylalanine levels by at least 20% — which the researchers defined as a positive response. The reported data shows that across all three treatment groups measured — diet treatment, standard dose sapropterin, and high dose sapropterin — the number of participants who achieved that 20% reduction in phenylalanine levels was zero in each case. In other words, none of the 7 participants were recorded as having a positive response under any of the three conditions, based on the primary outcome measure as defined by the trial. No secondary outcome data appears to have been 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.
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT03694353 · results posted 17 February 2022
According to the results reported on ClinicalTrials.gov, this trial (NCT03694353) enrolled 37 people in a single group, all of whom were receiving the study drug pegvaliase. The trial was measuring two main things: how often participants experienced unwanted health events (called adverse events) after starting the drug, and how levels of a substance called phenylalanine (Phe) in the blood changed over time compared to before treatment began. Phenylalanine is an amino acid that builds up in people with the metabolic condition this drug targets. Thirty-five of the 37 participants completed the study, and two did not. The reported data shows that when it came to unwanted health events, 36 out of 37 participants experienced at least one treatment-emergent adverse event (meaning a health issue that appeared or got worse after starting the drug). Of those, 17 experienced what were classified as more serious events, 7 experienced a particular category of serious events, and 1 experienced another specified category — though the data provided does not give further detail on the nature of these events. Regarding blood phenylalanine levels, the reported data shows that the average starting level was 1,376.9 µmol/L (micromoles per litre, a standard unit for measuring substances in blood). Across multiple follow-up time points during the study, the reported average change from that starting level ranged from a reduction of around 761 µmol/L to a reduction of around 1,002 µmol/L. The data does not specify exactly which time points these measurements correspond to. 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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NCT00924703 · results posted 12 October 2021
According to the results reported on ClinicalTrials.gov, this trial (NCT00924703) enrolled 68 adults who received injections of a study drug called rAvPAL-PEG (also known as pegvaliase). The trial was investigating a condition called phenylketonuria (PKU), where the body cannot properly break down a substance called phenylalanine (Phe) — an amino acid found in many foods. When Phe builds up in the blood, it can cause serious health problems. The trial's main goal was to measure whether blood Phe levels changed over time while participants were on the study drug. Of the 68 people who started, 46 completed the study and 22 did not finish. The reported data shows that, at the start of the study, the average blood Phe level was approximately 1,022 µmol/L (micromoles per litre — a standard way of measuring how much of a substance is in the blood). Across several follow-up points during the study, the reported changes from that starting level ranged from reductions of around 536 to 672 µmol/L. The reported data also shows that levels of the study drug measured in the blood at steady state (meaning when the drug had reached a relatively stable level in the body) ranged from approximately 962 to 7,765 ng/mL across different time points. Regarding side effects, the reported data shows that 68 out of 68 participants experienced at least one treatment-emergent adverse event (an unwanted health event that appeared or worsened after starting the drug), and 65 out of 68 experienced a serious adverse event. The data also shows that over time, varying numbers of participants developed antibodies (proteins the immune system produces in response to a foreign substance) against components of the drug. 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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NCT00532844 · results posted 17 May 2021
According to the results reported on ClinicalTrials.gov, this trial enrolled 48 adults in total — 24 in each group — and used a "crossover" design, meaning everyone tried both treatments at different times. The trial was looking at a medicine called sapropterin dihydrochloride, given either on its own or combined with Vitamin C, in people whose blood vessels were not functioning normally (described as "endothelial dysfunction"). The main thing being measured was how much of an active substance called BH4 — a molecule the body needs for healthy blood vessel function — built up in the blood over 12 hours after taking the medicine. The reported data shows that when participants took sapropterin alone, the BH4 blood level (measured as an area under the curve, which is a way of adding up the total amount in the blood over time) was recorded as 633 nM\*hr. When they took sapropterin combined with Vitamin C, the figure was 993 nM\*hr. For the secondary measurements, the reported data shows that levels of related substances (BH2 and B, which are breakdown products of BH4) were 875 and 772 nM\*hr respectively for BH2, and 231 and 185 nM\*hr for B. A measure of blood vessel function (called PAT, which tracks pulse wave changes in the finger) showed small changes from the starting point in both groups, and average daytime systolic blood pressure (the top number in a blood pressure reading) changed by −3.24 mmHg with sapropterin alone and −0.10 mmHg with the combination; however, the data does not include information about whether these differences were considered meaningful by the researchers. 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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NCT02468570 · results posted 4 June 2020
According to the results reported on ClinicalTrials.gov, this trial (NCT02468570) enrolled a total of 9 participants — 6 in the active treatment group and 3 in the placebo group. All 9 participants completed the study with no dropouts. The trial was measuring changes in three areas of thinking and mental processing: sustained attention (how well someone stays focused on a repetitive task), spatial working memory (how well someone remembers locations to avoid repeating mistakes), and the ability to stop or hold back a response quickly. These were all assessed using a set of computerised tasks known as CANTAB. The reported data shows the following changes from the start of the study to the end. For the sustained attention task, the active group's average response time changed by −24.23 milliseconds (a decrease, meaning faster responses) while the placebo group's changed by +18.03 milliseconds (slower). For the spatial working memory task, where a lower score means fewer repeated mistakes, the active group's average score changed by −4.2 and the placebo group's by +3.0. For the response-stopping task, where a lower time is considered better, the active group's average changed by +8.42 milliseconds and the placebo group's by +58.93 milliseconds. The reported data for the secondary measures showed broadly similar patterns across the groups, though individual scores varied considerably within each group. It is important to note that this was a very small study with only 9 people in total, which means these numbers should be interpreted with great caution and cannot be used to draw broad conclusions. 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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NCT01428258 · results posted 3 March 2017
According to the results reported on ClinicalTrials.gov, this trial involved 32 people in total (17 in one group and 15 in the other), all of whom had a condition called PKU (phenylketonuria). PKU is a metabolic disorder that requires people to carefully manage their intake of an amino acid called phenylalanine (Phe), which is found in protein. The trial was testing two different specially formulated diets — one based on a protein ingredient called glycomacropeptide (GMP) and one based on individual amino acids (AA) — to compare how each diet affected levels of phenylalanine in participants' blood. It was a crossover design, meaning participants tried both diets in sequence. The reported data shows that the primary measurement was the change in blood phenylalanine levels after three weeks on each diet. For the GMP diet group, the reported change was an increase of 62 micromoles per litre of blood plasma, while for the amino acid diet group, the reported change was a decrease of 85 micromoles per litre. For the secondary measures, dietary compliance was reported as similar between the two diets (0.74 versus 0.76 grams of protein from medical food per kilogram of body weight per day). The reported scores on an executive function (thinking and behaviour) test were also similar (49.0 versus 48.8 on a standard scale where scores below 50 are considered within the normal range). Vitamin D levels at the end of the study were nearly identical between groups (33.8 versus 33.6 ng/ml), as were measures of bone turnover (17.0 versus 17.0 micrograms per litre). The trial also compared two different methods of measuring phenylalanine — one from a blood draw and one from a dried blood spot — and reported average readings of 731 versus 514 micromoles per litre respectively, suggesting the two methods produced different numbers from the same samples. 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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NCT01617070 · results posted 18 November 2016
According to the results reported on ClinicalTrials.gov, this trial enrolled 10 participants, all grouped together as a single study group. The trial was looking at how different treatments affected levels of certain chemicals in the body — specifically melatonin (a hormone linked to sleep) measured in blood and urine, and dopamine (a brain chemical) measured in urine. Each participant went through four separate four-week phases: taking a supplement called LNAA (large neutral amino acids), a washout period (no treatment, to clear the body between phases), a medication called BH4 (brand name Kuvan), and a combination of both BH4 and LNAA. All 10 participants completed three of the four phases; one person did not complete the final combined-treatment phase. The reported data shows the following average levels at the end of each four-week phase. For melatonin measured in blood, the reported figures were 266.9 pg/ml during the LNAA phase, 205.7 pg/ml during the washout phase, 220.4 pg/ml during the BH4-only phase, and 301.2 pg/ml during the combined BH4 and LNAA phase. For a melatonin breakdown product measured in urine, the reported figures were 14.5 units during LNAA, 8.2 during washout, 8.6 during BH4 only, and 13.2 during the combined phase. For dopamine measured in urine, the reported figures were 63.7 units during LNAA, 42.6 during washout, 46.0 during BH4 only, and 67.6 during the combined phase. 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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NCT01376908 · results posted 12 May 2016
According to the results reported on ClinicalTrials.gov, this trial enrolled 56 children in total — 27 in a group receiving Kuvan® (sapropterin) combined with a phenylalanine (Phe)-restricted diet, and 29 receiving a Phe-restricted diet alone. Phenylalanine is an amino acid found in protein-containing foods, and people with the condition being studied (phenylketonuria, or PKU) need to limit how much of it they consume. The trial ran for 26 weeks and its main goal was to measure how much dietary phenylalanine each child could tolerate — that is, how much Phe they could eat each day while still keeping their blood Phe levels within a target range considered acceptable for their age and condition. The reported data shows that at week 26, the Kuvan® plus diet group was prescribed an average of 80.6 milligrams of Phe per kilogram of body weight per day (mg/kg/day), compared with 50.1 mg/kg/day in the diet-only group. When looking at the change from the start of the trial to week 26, the reported figures suggest an increase in Phe tolerance in both groups, though the specific before-and-after breakdowns across subgroups were also reported. Mean blood Phe levels (measured in micromoles per litre) were tracked at multiple two-week intervals throughout the trial; across those time points, the Kuvan® plus diet group's reported values ranged from approximately 202 to 287, while the diet-only group's values ranged from approximately 303 to 353. On the developmental assessments (Bayley III scales, scored from 40 to 160), composite scores for both groups generally fell within what the scale describes as the "within normal limits" range (85–114), with most individual scores sitting between roughly 93 and 107 across the different areas measured. Regarding adverse events (unexpected or unwanted medical occurrences during the study), the reported data shows that all 27 participants in the Kuvan® group and 27 out of 29 in the diet-only group experienced at least one such event; 8 participants in the Kuvan® group had events considered related to Kuvan®, compared with none in the diet-only group. Three participants in the Kuvan® group and one in the diet-only group experienced a serious adverse event. No deaths or discontinuations due to adverse events were reported in either 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.
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT01806051 · results posted 20 April 2016
According to the results reported on ClinicalTrials.gov, this trial enrolled 6 participants in total. The study was looking at changes in levels of two substances in the blood — phenylalanine (Phe, an amino acid that builds up in people with a condition called PKU) and tyrosine (another amino acid) — comparing measurements taken at the start of the study with those taken four weeks later. Participants were divided into two groups: one group made up of people with PKU, and one control group. The reported data shows that none of the 6 participants completed the study — all 6 are recorded as "not completed." Because no participants finished the trial, the primary outcome measure (the blood level changes that the study set out to record) has no measurement data reported on ClinicalTrials.gov. In other words, the numbers that would have shown any changes in phenylalanine and tyrosine levels were not collected or submitted. As a result, there are no outcome figures available to describe from this trial. The data was not reported, most likely because the study did not reach completion. 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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NCT01114737 · results posted 1 February 2016
According to the results reported on ClinicalTrials.gov, this trial enrolled 206 people with PKU (phenylketonuria) — 108 in the placebo group and 98 in the group receiving a medicine called 6R-BH4 (also known as sapropterin) at a dose of 20 mg/kg/day. The trial was measuring whether 6R-BH4 had any effect on a range of neurological and psychological symptoms in people with PKU, including attention difficulties, anxiety, depression, overall mental wellbeing, and executive function (things like planning, organisation, and self-control). These measurements were taken over a 13-week treatment period, followed by an open-label period where participants could continue on the active treatment. The reported data shows results for participants whose blood levels of phenylalanine (a marker relevant to PKU) actually dropped in response to treatment — these participants are referred to as "responders." For the primary outcome — a rating scale for attention and hyperactivity symptoms (scored 0–54, where higher means more severe) — the placebo responders showed an average decrease of 4.9 points from their starting score, while the 6R-BH4 responders showed an average decrease of 9.1 points. For the secondary outcomes, the reported data shows: anxiety scores (0–56 scale) decreased by 3.6 points in the placebo group and 3.2 points in the 6R-BH4 group; depression scores (0–48 scale) decreased by 2.5 and 2.1 points respectively; overall mental illness severity (1–7 scale) decreased by 0.5 and 0.6 points respectively; a self-reported executive function score decreased by 8.1 points (placebo) and 9.1 points (6R-BH4); and a parent-reported executive function score decreased by 0.7 points (placebo) and 4.8 points (6R-BH4). In all cases, a decrease in score represents a move toward fewer or less severe symptoms on that particular 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.
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT01917344 · results posted 19 June 2015
According to the results reported on ClinicalTrials.gov, this trial (NCT01917344) enrolled 10 adults with a condition called PKU (phenylketonuria — an inherited disorder affecting how the body processes a substance called phenylalanine, or "Phe"). Nine of the 10 participants completed the study, and one did not. The trial was measuring levels of phenylalanine — a building block of protein that can build up in the body and brain in people with PKU — both in the brain (using a specialised type of brain scan called MR spectroscopy) and in the blood, taken on the same day. The reported data shows three phenylalanine measurements listed for the group, in units of micromoles per litre (µmol/L — a standard way of measuring how much of a substance is in a fluid). The three figures reported were 130 µmol/L, 137 µmol/L, and 961 µmol/L. The context for these three numbers — for example, whether they represent brain levels, blood levels, or measurements taken at different time points — was not clearly described in the submitted data, so it is not possible to say with certainty what each figure refers to. The trial also intended to measure a number of other things, including IQ scores, brain scan findings, and tremor assessed by a neurologist, but no numerical results for any of those secondary outcomes were reported in the data 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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NCT00986973 · results posted 12 May 2015
According to the results reported on ClinicalTrials.gov, this trial enrolled 6 people, all of whom received the drug sapropterin (also known as KUVAN) at a set daily dose for four months. Five of the six participants completed the trial, while one did not finish. The trial was looking at whether sapropterin had any relationship with levels of a substance called phenylalanine (Phe) in the blood — a naturally occurring compound that can build up in people with certain metabolic conditions — as well as how participants performed on a range of thinking and memory tests before and after treatment. The reported data shows that the median blood phenylalanine level was 18.0 mg/dl before treatment and 17.2 mg/dl afterwards. On the verbal memory test (Hopkins Verbal Learning Test), the reported median scores went from 51 to 53 for total recall, and from 47 to 49 for delayed recall, on a scale where higher numbers indicate better verbal memory. On a test of auditory processing speed (PASAT), scores went from 0.47 to 0.94 on a scale comparing participants to a reference group (where zero equals the average of that group). On a test of attention and processing (Symbol-Digit Modalities Test), the reported score went from 63 to 79 out of a possible 110. On a digit-coding test drawn from an intelligence scale (WAIS-IV), the reported score went from 33 to 37 correct symbols. The reported data shows only the numbers before and after the four-month period; the trial did not report any further breakdown of these figures. Given that only six people took part, these numbers represent a very small group, and no comparison group (such as people who did not receive the treatment) 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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NCT01395394 · results posted 28 August 2014
According to the results reported on ClinicalTrials.gov, this trial involved 12 people in total across three groups: 4 people who did not respond to a treatment called BH4 (also known as Kuvan), 3 healthy volunteers used as a comparison group, and 5 people who did respond to BH4. The trial was measuring two markers in the blood — one called lipid peroxidation (a marker related to a type of cell damage) and another called C-Reactive Protein, or CRP (a marker that can rise when the body is under stress). Blood samples were taken every two hours over a six-hour visit. The non-responder group attended two such visits — one at the start and one after two weeks on BH4 — while the other groups attended only one visit. The reported data shows the following numbers for CRP levels (measured in mg/dL), taken at four time points across the visit: in the BH4 non-responder group, readings went from 1.46 to 1.50, 1.55, and 1.59; in the healthy control group, readings went from 0.61 to 0.58, 0.55, and 0.55; and in the BH4 responder group, readings went from 1.04 to 0.99, 0.96, and 0.90. For lipid peroxidation (measured in umole/L), the reported data shows four readings — 0.57, 0.62, 0.73, and 0.78 — however, the data as submitted does not clearly separate these figures across all three groups, so a full group-by-group breakdown cannot be provided here. It is worth noting that one participant in the non-responder group did not complete the first study visit, and the numbers across all groups are quite small. 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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NCT00688844 · results posted 20 August 2014
According to the results reported on ClinicalTrials.gov, this trial enrolled 58 people who had been diagnosed with phenylketonuria (PKU — a condition where the body cannot properly process a building block of protein called phenylalanine) and were starting treatment with sapropterin (also known as Kuvan). Of the 58 who started, 48 completed the study and 10 did not finish. The trial tracked a range of body composition and dietary measurements over 12 months to see how these figures changed from the start of treatment. The reported data shows the following average changes from the beginning of the study to the 12-month mark across the group. Body mass index (BMI — a number based on height and weight) went down by an average of about 0.66 kg/m². Bone mineral density (a measure of how dense or strong bones are) went up by a very small average of about 0.005 grams per centimetre squared. The proportion of the body made up of lean tissue (such as muscle) went up by an average of about 2.4 percentage points, while the proportion made up of fat also went up slightly, by an average of about 1.2 percentage points. The level of phenylalanine measured in the blood went down by an average of about 67.5 micromoles per litre (micromoles per litre is simply a way of measuring how much of a substance is in the blood). Finally, the total amount of protein eaten each day went down by an average of about 11 grams per day. 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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NCT00332189 · results posted 16 November 2012
According to the results reported on ClinicalTrials.gov, this trial enrolled 111 participants, all grouped together as a single patient population. Ninety of them completed the study, while 21 did not finish. The trial was looking at two things: first, how often participants experienced adverse events (unexpected health problems or side effects) and serious adverse events during the study; and second, how levels of a substance called phenylalanine (Phe) in the blood changed over time. Phenylalanine is an amino acid that can build up in the blood in people with certain metabolic conditions. The reported data shows that 83.8% of participants reported at least one adverse event of any kind during the study, while 33.3% reported a serious adverse event. The figures of 6.3% and 0.9% were also reported in relation to adverse events, though the data as submitted does not provide labels clearly distinguishing what each of these two figures specifically refers to, so their exact meaning cannot be confirmed here. For the blood Phe measurements, the reported data shows a starting average level of 607.4 micromoles per litre (a unit used to measure concentration in the blood). At various follow-up points during the study, average levels were recorded as 504.6, 494.4, 526.9, and 482.0 micromoles per litre, with the changes from the starting point ranging from approximately −68 to −122 micromoles per litre. A figure of 808.0 was also reported, though its context within the data was not clearly labelled. The trial notes there were no pre-specified analyses planned to test whether the treatment was effective. 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 ↗
See the full Phenylketonuria page · What changed recently
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.