A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
Recruiting · Phase 3 · NCT06302348
Conditions: Phenylketonuria
At a glance
- Research payments reported, 2025
- $7,116
- U.S. investigators named
- 1
- Sites paid
- 1
- Planned or actual enrollment
- 56
About the trial
- Sponsor
- PTC Therapeutics
- Tested
- Sepiapterin (drug)
- Dates
- Started 2024-03-04; completion 2031-02-28
- Results posted
- Not yet
The main purpose of this trial is to evaluate the long-term efficacy of sepiapterin on preserving neurocognitive functioning in children with PKU when treatment is initiated in early childhood.
From ClinicalTrials.gov, last updated 2026-10-23. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Ptc Therapeutics, Inc. reported $7,116 in research payments for this study in 2025 (3 payments).
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- The Trustees of Indiana University $3,928
- $3,188
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.