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ClinicalTrials.gov public records Last synced May 21, 2026, 7:45 PM EDT

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Showing 1–2 of 2 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Pontocerebellar Hypoplasia Type 6 (PCH6) Clear all
Terminated Phase 2Phase 3 Interventional Results available
View directory record Official record
Conditions
Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome, Mitochondrial Encephalopathy (MELAS), Pontocerebellar Hypoplasia Type 6 (PCH6), Alpers Disease, Alpers Syndrome
Interventions
Vatiquinone, Placebo
Drug · Other
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Up to 20 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
12
States / cities
San Diego, California • Stanford, California • New Haven, Connecticut + 7 more
Source: ClinicalTrials.gov public record
Updated Mar 30, 2026 · Synced May 21, 2026, 7:45 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Inherited Mitochondrial Disease
Interventions
Vatiquinone
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Not listed
Enrollment
101 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
14
States / cities
La Jolla, California • Stanford, California • New Haven, Connecticut + 10 more
Source: ClinicalTrials.gov public record
Updated Dec 21, 2025 · Synced May 21, 2026, 7:45 PM EDT