Health Trials

Clinical trials

2

Search and review clinical trials. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Not yet recruiting

Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)

The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.

Participants needed: 120
Trial details
Phase: Phase 3Age: 7-21Biological sex: AllType: InterventionalSponsor: PTC TherapeuticsUpdated: Jul 2, 2026
Eligibility criteria

mFARS ≥20 to ≤70 at Screening (4 weeks prior to Day 1) and Baseline (Day 1). [+5]

Individuals with clinical diagnosis of FA who have point mutations, deletions, o... [+10]

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Status: Recruiting

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.

Participants needed: 56
Trial details
Phase: Phase 3Age: Up to 9Biological sex: AllType: InterventionalSponsor: PTC TherapeuticsUpdated: Jun 17, 2026Locations: 8
Eligibility criteria

Women of childbearing potential must have a negative pregnancy test at Screening... [+6]

History of allergies or adverse reactions to any of the ingredients or excipient... [+8]

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