Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Phenylketonuria: later-stage trial registry
4 of 13 indexed Phenylketonuria trials are currently recruiting, with more weight in Phase 3-4.
Corpus placement: #1,315 of 28,707 conditions by registered trial count.
13 US clinical trials · 4 currently recruiting · #1,315 of 28,707 conditions by registered trial count
Phenylketonuria: 13 US clinical trials tracked, 4 recruiting.
Phenylketonuria is the subject of 13 registered US clinical trials on ClinicalTrials.gov, 4 of them currently open to new participants. 7 are in Phase 3-4 (later-stage) and 4 in Phase 1-2 (earlier-stage). The most active sponsor is BioMarin Pharmaceutical, running 5 of these trials.
Enrollment posture brief
Registry condition phenylketonuria
OPEN 4 | RECR 31% | LATE 7 | EARLY 4 | RANK #1315 | SPON 5
Volume-matched condition peers
Nearest open-share peer: Acute Decompensated Heart Failure (31% recruiting · 13 trials)
Phenylketonuria lists 4 open slots (31% of 13 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Phenylketonuria on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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Phenylketonuria is recruiting below its size-band peers.
4 of 13 indexed trials (30.8%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
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One sponsor concentrates Phenylketonuria research.
BioMarin Pharmaceutical accounts for 5 of 13 trials (38.5%). 7 distinct sponsors appear on at least one study for this condition label.
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Most Phenylketonuria trials plan to enrol fewer than 100 people.
Median target enrolment is 56 among the 13 trials that report a genuine target (sentinel values excluded). 8 of those (61.5%) plan for fewer than 100 participants.
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Phenylketonuria research is mostly interventional.
12 interventional and 1 observational studies make up this index (92.3% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
13 total, page 1 of 1
Educational, Social Support, and Nutritional Interventions and Their Cumulative Effect on Pregnancy Outcomes and Quality of Life in Teen and Adult Women With Phenylketonuria
Emory University
NCT01659749
A Study to Evaluate the Safety and Efficacy of JNT-517 in Participants With Phenylketonuria (PKU)
Otsuka Pharmaceutical Development & Commercialization
NCT06971731
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
PTC Therapeutics
NCT06302348
Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq
BioMarin Pharmaceutical
NCT06780332
A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
PTC Therapeutics
NCT05166161
First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria
Otsuka Pharmaceutical Development & Commercialization
NCT05781399
A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria
Sanofi
NCT05972629
Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients
BioMarin Pharmaceutical
NCT01114737
Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU
BioMarin Pharmaceutical
NCT00838435
A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects
BioMarin Pharmaceutical
NCT00789568
Sapropterin in Individuals With Phenylketonuria
Washington University School of Medicine
NCT00730080
A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks
BioMarin Pharmaceutical
NCT01560286
The Effectiveness of Kuvan in Amish PKU Patients
University Hospitals Cleveland Medical Center
NCT02677870
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 3 |
| Phase 2 | 1 |
| Phase 3 | 5 |
| Phase 4 | 2 |
Top Sponsors
Named interventions
Most-linked intervention names on Phenylketonuria records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Phenylketonuria trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Phenylketonuria page
13 registered trials, 4 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 4 Phenylketonuria trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- BioMarin Pharmaceutical sponsors the most Phenylketonuria trials on record, review their full trial history before evaluating a specific study. See BioMarin Pharmaceutical's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Phenylketonuria condition card totals 13 registered trials; most-linked intervention sapropterin dihydrochloride; top US site state Florida. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.