Trials / Recruiting
RecruitingNCT06302348
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
A Phase 3b Open-Label Study of Long-Term Neurocognitive Outcomes in Children With Phenylketonuria Treated With Sepiapterin
- Status
- Recruiting
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 56 (estimated)
- Sponsor
- PTC Therapeutics · Industry
- Sex
- All
- Age
- 10 Years
- Healthy volunteers
- Not accepted
Summary
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.
Detailed description
The study includes 2 parts: Part 1 and 2. Part 1 is an open-label sepiapterin-responsiveness test and Part 2 is an open-label treatment period.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Sepiapterin | Sepiapterin powder for oral use will be mixed in water or apple juice prior to administration. |
Timeline
- Start date
- 2024-03-04
- Primary completion
- 2031-02-28
- Completion
- 2031-02-28
- First posted
- 2024-03-08
- Last updated
- 2026-03-27
Locations
5 sites across 4 countries: United States, Australia, Poland, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT06302348. Inclusion in this directory is not an endorsement.