Status and phase
Conditions
Treatments
Study type
Funder types
Identifiers
About
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.
Full 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.
Enrollment
Sex
Ages
Volunteers
Inclusion and exclusion criteria
Key Inclusion Criteria:
For all participants:
For participants <1 month of age at the time of informed consent/assent only:
For participants ≥30 months to <12 years of age:
Key Exclusion Criteria:
Note: Other protocol-defined inclusion and exclusion criteria may apply.
Primary purpose
Allocation
Interventional model
Masking
56 participants in 1 patient group
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Patient Advocacy
Data sourced from clinicaltrials.gov
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