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Phenylketonuria (PKU): full-pipeline trial registry

3 of 7 indexed Phenylketonuria (PKU) trials are currently recruiting.

Corpus placement: #2,497 of 28,707 conditions by registered trial count.

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7 US clinical trials · 3 currently recruiting · #2,497 of 28,707 conditions by registered trial count

Phenylketonuria (PKU): 7 US clinical trials tracked, 3 recruiting.

Phenylketonuria (PKU) is the subject of 7 registered US clinical trials on ClinicalTrials.gov, 3 of them currently open to new participants. 2 are in Phase 3-4 (later-stage) and 2 in Phase 1-2 (earlier-stage). The most active sponsor is BioMarin Pharmaceutical, running 6 of these trials.

Enrollment posture brief

Registry condition phenylketonuria-pku

OPEN 3 | RECR 43% | LATE 2 | EARLY 2 | RANK #2497 | SPON 6

Volume-matched condition peers

Nearest open-share peer: Adrenoleukodystrophy (43% recruiting · 7 trials)

ACL Tear71.4%ACL57.1%Phenylketonuria (this)43%Acute Myelogenous Leukemia42.9%Acne Scars28.6%
Open-enrollment share among volume-matched conditions near Phenylketonuria (PKU)

Phenylketonuria (PKU) lists 3 open slots (43% of 7 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 (PKU) 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.

  1. Phenylketonuria (PKU) is recruiting below its size-band peers.

    3 of 7 indexed trials (42.9%) 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.

  2. One sponsor concentrates Phenylketonuria (PKU) research.

    BioMarin Pharmaceutical accounts for 6 of 7 trials (85.7%). 2 distinct sponsors appear on at least one study for this condition label.

  3. Typical Phenylketonuria (PKU) enrolment sits around 100 participants.

    Median target enrolment is 100 among the 7 trials that report a genuine target (sentinel values excluded). 3 of those (42.9%) plan for fewer than 100 participants.

  4. Phenylketonuria (PKU) splits between interventional and observational designs.

    4 interventional and 3 observational studies make up this index (57.1% interventional). Study type is sponsor-reported on ClinicalTrials.gov.

Phase 11Phase 21Phase 32

Phase Distribution

PhaseTrial count
Phase 1 1
Phase 2 1
Phase 3 2

Top Sponsors

BioMarin Pharmaceutical 6 trials
Otsuka Pharmaceutical Development & Commercialization 1 trials

Named interventions

Most-linked intervention names on Phenylketonuria (PKU) records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.

Pegvaliase Drug
3 trials
BMN165 40mg/day Drug
1 trials
BMN165 20mg/day Drug
1 trials
Diet Only Other
1 trials
BMN 307 Drug
1 trials
JNT-517 Tablet Drug
1 trials
Placebo Other
1 trials
Placebo Drug
1 trials

US sites by state

Facility locations filed on Phenylketonuria (PKU) trials. A trial with sites in several states counts in each.

Illinois 5 trials
Texas 4 trials
Florida 4 trials
Colorado 4 trials
Pennsylvania 3 trials
Massachusetts 3 trials
Indiana 3 trials
Wisconsin 2 trials

Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.

Related

Read our methodology - how this data is sourced, computed, and verified.

What to do with this Phenylketonuria (PKU) page

7 registered trials, 3 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.

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 (PKU) condition card totals 7 registered trials; most-linked intervention Pegvaliase; top US site state Illinois. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.