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Trackers Clinical trials Phenylketonuria
Clinical trial register · United States · industry-sponsored

Phenylketonuria Trials in the USA

Every industry-sponsored study on the ClinicalTrials.gov registry that lists a US site and is open, about to open, active-but-closed, or enrolling by invitation. This deliberately excludes academic and other non-industry sponsors โ€” the full US registry across all sponsors runs to over 22,000 recruiting studies alone.

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Source: ClinicalTrials.gov. Complete coverage of the industry-sponsored slice โ€” all 9094 matching records are in this view (non-industry sponsors are out of scope by design, not truncated). Synced 17 Sep 2026, 08:23 UTC
9094
Studies in scope
industry-sponsored, all four open statuses
4935
Recruiting now
open to new participants
2188
Lead sponsors
across recruiting studies
Top states
Florida 4 Indiana 3 California 3 Pennsylvania 3 Texas 3
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8 of 9094 studies
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Study
Lead sponsor
Phase
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US states
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Recruiting
A Trial to Examine if Repinatrabit is Processed Differently in Adults With Reduced Liver or Kidney Function Compared to Adults With Normal Liver and Kidney Function
NCT07713758Phenylketonuria
Otsuka Pharmaceutical Development & Commercialization, Inc.
Phase 1
32
Florida
2026-07-17
Recruiting
Immune Modulation During Palynziqยฎ Treatment in Adults (IMPALA)
NCT07477691Phenylketonuria
BioMarin Pharmaceutical
Phase 4
12
California, Colorado, Florida, Illinois, Indiana, Minnesota, New York, Oregon
2026-05-29
Recruiting
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria
NCT07241234Phenylketonuria
Agios Pharmaceuticals, Inc.
Phase 1
20
Indiana, Pennsylvania, Texas
2026-04-17
Recruiting
A Study of JNT-517 in Participants With Phenylketonuria (PKU)
NCT06971731Phenylketonuria
Otsuka Pharmaceutical Development & Commercialization, Inc.
Phase 3
120
California, Florida, Illinois, Oregon, Pennsylvania, Tennessee, Texas, Utah
2025-10-20
Active, closed
Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq
NCT06780332Phenylketonuria, PKU
BioMarin Pharmaceutical
Phase 4
9
Maryland
2025-02-07
Recruiting
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
NCT06302348Phenylketonuria
PTC Therapeutics
Phase 3
56
Indiana
2024-03-04
Active, closed
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
NCT05972629Phenylketonuria
Sanofi
Phase 1/2
32
California, Colorado, Florida, Ohio, Pennsylvania, South Carolina, Texas
2023-08-07
By invitation
Long-Term Follow Up Study of Subjects Previously Administered HMI 102
NCT04348708Phenylketonuria, PAH Deficiency
Homology Medicines, Inc
Observational
21
Massachusetts, New York
2020-08-19
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Cite this view

Priya Life Science (2026). Clinical trial register: industry-sponsored studies with a US investigative site, filtered. Derived from ClinicalTrials.gov. Retrieved 18 September 2026. https://priyalifescience.com/clinical-trials-usa/condition/phenylketonuria

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Listing here is informational and not medical advice; eligibility is determined by each study team. Compare pay for the teams running these studies in the US Pharma Salary Guide, see who's behind them in the US Company Directory, or track approvals on the FDA Approvals Tracker. For Irish, UK and EU sites, see the Ireland, UK and EU registers.