A Study of Sepiapterin in Participants With Phenylketonuria (PKU)

Part of paid clinical trials in Indianapolis, Indiana.

Sponsor
PTC Therapeutics
Study ID
NCT06302348
Phase
PHASE3
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
N/A - 9 Years
Healthy Volunteers
Not accepted

Interventions

  • Sepiapterin — DRUG
    Sepiapterin powder for oral use will be mixed in water or apple juice prior to administration.

Study Details

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.

Key Dates

Start date
Mar 4, 2024
Status verified
Apr 2026
Primary completion
Feb 28, 2031
Completion
Feb 28, 2031

Study Design

Enrollment
56 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Sepiapterin
    Participants will receive age- and weight-adjusted doses of sepiapterin orally once daily for up to 6 years.

Primary Outcome Measure

Mean Change From Baseline in Full-scale Intelligence Quotient (FSIQ) Wechsler Preschool and Primary Scale of Intelligence - Fourth Edition (WPPSI-IV) Score [ Time Frame: Baseline, Year 2 ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Indiana University School of MedicineIndianapolisIndiana46202
Susan Romie
Melissa Lah (PRINCIPAL_INVESTIGATOR)

Find similar trials in Indianapolis, IN

By research site
Indiana University School of Medicine· Indianapolis, IN

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