A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
ConditionPhenylketonuria
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
AgeUp to 9
SponsorPTC Therapeutics
About this trial
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.
Eligibility criteria
Qualifiers
Women of childbearing potential must have a negative pregnancy test at Screening and agree to abstinence or the use of at least one highly effective form of contraception for the duration of the study, and for at least 90 days after the last dose of the study drug.
Willing to maintain prescribed daily protein/Phe during Screening and Part 1.
Established diagnosis of PKU with hyperphenylalaninemia (HPA) evidenced by at least 2 blood Phe measurement ≥600 micromoles (μmol)/liter (L) as documented in the medical history.
A minimum of 1 documented blood Phe measurement <480 μmol/L within 1 month prior to Screening.
Disqualifiers
History of allergies or adverse reactions to any of the ingredients or excipients of synthetic tetrahydrobiopterin (BH4) or sepiapterin.
Serious neuropsychiatric illness (for example, major depression) not currently under medical control or other concurrent disease or condition that, in the opinion of the investigator or sponsor, would interfere with the participant's ability to participate in the study or increase the risk of participation for that participant.
Treatment with BH4 supplementation (sapropterin, KUVAN®) within 3 months prior to Screening.
Current participation in another investigational drug study or use of any investigational agent within 30 days prior to Screening.
Trial design
Treatments tested in this trial
- Sepiapterin
Treatment groups
56 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov