A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
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Phase
Phase 3
Status
Completed
Study type
Interventional
Design
Na · None · Treatment
Enrollment
101actual
Sites
26
Countries
France, Italy, Japan +4
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2022-06-22 | actual | When the study began enrolling |
| Primary completion | 2025-04-15 | actual | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2025-04-15 | actual | The whole study's end, after follow-up |
| First posted | 2022-02-01 | actual | When this record first appeared on the registry |
| Results posted | 2025-12-22 | actual | When the sponsor posted results to the registry |
| Record updated | 2025-12-22 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Inherited Mitochondrial Disease
Intervention
- Drug: Vatiquinone — also filed as PTC743
Primary outcome
what the primary-completion date above is the date OFNumber of Participants With Treatment-emergent Adverse Events (TEAEs)
measured Baseline (Day 1) up to 30 days after last dose of study drug (956 days)
Posted documents
hosted by the registry — we link, never re-serveStudy Protocolposted 2023-01-25 · 1.2 MB · Prot_000.pdf
Statistical Analysis Planposted 2025-04-04 · 1.0 MB · SAP_001.pdf
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