NCT05218655

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

An Open-Label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial Disease

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 101 (actual)
• Sponsor: PTC Therapeutics · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Conditions

• Inherited Mitochondrial Disease

Interventions

Type	Name	Description
DRUG	Vatiquinone	Vatiquinone will be administered per dose and schedule specified in the arm description.

Timeline

• Start date: 2022-06-22
• Primary completion: 2025-04-15
• Completion: 2025-04-15
• First posted: 2022-02-01
• Last updated: 2025-12-22
• Results posted: 2025-12-22

Locations

26 sites across 7 countries: United States, France, Italy, Japan, Poland, Spain, United Kingdom

Regulatory

• FDA-regulated drug study