Phase 3 N=101 Treatment

A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

Inherited Mitochondrial Disease

Bottom Line

View on ClinicalTrials.gov: NCT05218655 ↗

Enrolled (actual)

101

Serious AEs

44.6%

Results posted

Dec 2025

Primary outcome: Primary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) — 91 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Vatiquinone (Drug)
Age: Pediatric, Adult, Older Adult
Sex: All
Sponsor: PTC Therapeutics
Primary completion: Apr 2025

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Treatment-emergent Adverse Events (TEAEs)	91	—

Summary

The primary objective of this study is to assess the safety of vatiquinone in participants with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron sponsored (previously Edison) clinical study or treatment plan. The study will continue until vatiquinone becomes commercially available or the program is terminated.

Eligibility Criteria

Inclusion Criteria

Participants with inherited mitochondrial disease including Leigh syndrome, Alpers syndrome, mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS), myoclonic epilepsy with ragged-red fibers (MERRF), pontocerebellar hypoplasia type 6 (PCH6), or other mitochondrial disease who participated in a previous vatiquinone clinical study or treatment plan.
Women of childbearing potential must have a negative pregnancy test at screening/baseline and agree to abstinence or the use of at least 1 of the highly effective forms of contraception as specified in the protocol (with a failure rate of <1% per year when used consistently and correctly). Highly effective contraception or abstinence must be continued for the duration of the study, and for up to 30 days after the last dose of study drug.
Fertile men who are sexually active with women of childbearing potential and who have not had a vasectomy, must agree to use a barrier method of birth control during the study and for up to 30 days after the last dose of study drug.

Exclusion Criteria

Current participation in any other interventional study.
Pregnancy or breast feeding.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT05218655). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.