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NCT06302348 · ClinicalTrials.gov registry record · Phase 3
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
A Phase 3 study of Phenylketonuria, sponsored by PTC Therapeutics.
- Recruiting
- Registry status
- Phase 3
- Development phase
- 56
- Enrollment target
- 1
- Study location
NCT06302348: Recruiting Phase 3 study of Phenylketonuria, sponsored by PTC Therapeutics.
NCT06302348 is a Phase 3 study of Phenylketonuria that is actively recruiting participants, run by PTC Therapeutics. The registered enrollment target is 56 participants, below the 94-participant average among 12 other Phenylketonuria trials with a reported enrollment target (40% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06302348, a Phase 3 study of Phenylketonuria, is actively recruiting participants, sponsored by PTC Therapeutics.
- RECRUITING
- Registry status
- Phase 3
- Development phase
- 56 participants
- Enrollment target
- 1
- Study location
Study 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.
Primary Outcome
Analysis for participants ≥30 months to \<6 years of age.
Conditions Studied
Interventions
- DRUG Sepiapterin
Study Locations (1)
Indiana
- Indiana University - Indianapolis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 56 participants |
| Start Date | 2024-03-04 |
| Est. Completion | 2031-02-28 |
| Phase | Phase 3 |
What NCT06302348 shows while recruiting
NCT06302348 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 56 participants, a relatively small participant target, below the 94-participant average among 12 other Phenylketonuria trials with a reported enrollment target (40% lower).
The record links to 1 condition, with Phenylketonuria appearing as the primary indexed condition, and to 1 intervention - of which Sepiapterin is the first listed.
NCT06302348 reports a single indexed study location in Indiana.
Frequently Asked Questions
What is clinical trial NCT06302348 about?
NCT06302348 is a clinical study titled "A Study of Sepiapterin in Participants With Phenylketonuria (PKU)". 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.
What is the current status of trial NCT06302348?
This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 56 participants. The study started on 2024-03-04. Estimated completion is 2031-02-28.
What conditions does trial NCT06302348 study?
This clinical trial studies the following conditions: Phenylketonuria.
What interventions are being tested in trial NCT06302348?
The interventions under investigation include: Sepiapterin (DRUG).
Who is sponsoring clinical trial NCT06302348?
This trial is sponsored by PTC Therapeutics, which has 52 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06302348 being conducted?
This trial has 1 study location across Indiana. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Phenylketonuria
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT06302348's enrollment target sits among peer trials
56 7th of 12 higher than 5 of 12 other Phenylketonuria trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phenylketonuria trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
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