A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
← catalyst calendarNCT06302348 · readout in 1,654 d
Sponsored by PTC Therapeutics (industry) · PTCT — their whole pipeline →.
Phase
Phase 3
Status
Recruiting
Study type
Interventional
Design
Na · None · Treatment
Enrollment
56estimated
Sites
10
Countries
Australia, France, Ireland +3
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2024-03-04 | actual | When the study began enrolling |
| Primary completion | 2031-02-28 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2031-02-28 | estimated | The whole study's end, after follow-up |
| First posted | 2024-03-08 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-07-23 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Phenylketonuria
Intervention
- Drug: Sepiapterin — also filed as PTC923, Sephience
Primary outcomes
what the primary-completion date above is the date OFMean Change From Baseline in Full-scale Intelligence Quotient (FSIQ) Wechsler Preschool and Primary Scale of Intelligence - Fourth Edition (WPPSI-IV) Score
measured Baseline, Year 2
Mean Change From Baseline in FSIQ Wechsler Intelligence Scale for Children - Fifth Edition (WISC-V) Score
measured Baseline, Year 2
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