What changed for Phenylketonuria
A plain English summary of what recently moved in the public data Voxsanity tracks for Phenylketonuria. Updated from government registries. Not medical advice.
How to read these changes: every figure on this page is the difference between two snapshots of what Voxsanity had tracked on each date. Voxsanity only began taking daily snapshots in July 2026 and is still ingesting the public registries, so a rise here is mostly our own coverage expanding rather than new research appearing. Read it as our tracked coverage growing, not as a claim about real-world research changing this fast: it overstates how much actually changed.
Comparing the two most recent snapshots, 27 July 2026 and 28 July 2026.
- Trials recruiting worldwide 29 to 30 (+1)
- Trials on record 166 to 167 (+1)
Newly subsidised on the PBS
No medicines were newly subsidised on the PBS for Phenylketonuria in July 2026. Voxsanity tracks 16 PBS-subsidised medicines for this condition; none of them was listed for the first time this month.
Source: Pharmaceutical Benefits Scheme (PBS), © Commonwealth of Australia. Data used and redistributed under permission; not modified from its original wording where displayed verbatim.
Past updates
Tracking began in July 2026. Month on month changes will appear here once a second month of data has been recorded. Until then, the comparison above shows the most recent movement.
Recent trend
Weekly, most recent weeks (fills out to a monthly trend as history grows).
Recent trial results
Trials tracked for Phenylketonuria that have reported results to a public registry.
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 un...
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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) —...
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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 releva...
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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 comparis...
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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 d...
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...
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 s...
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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, 4...
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...
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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 k...
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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' b...
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 Ku...
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...
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 "no...
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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 perio...
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...
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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....
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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 atte...
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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...
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. Th...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
These are the results as reported to the registry, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
What is coming
AI generated pipeline 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
According to NIH funding data, public research investment in Phenylketonuria is currently at a moderate level, and encouragingly, that funding has been rising year on year. This upward trend suggests that researchers and funding bodies see value in continuing to explore this condition, which is a positive signal for patients hoping that scientific understanding will keep improving. Rising public i...
See the full Phenylketonuria page
Not medical advice. Voxsanity republishes public government health data in plain English. These figures describe research and registry activity, not the suitability of any treatment for any person. Trial status can change; always verify current status directly with the trial site and confirm Australian availability with your doctor.