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

NCT05218655 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 101

Last updated 2025-12-22

Study results available
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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

DRUG

Vatiquinone

Vatiquinone will be administered per dose and schedule specified in the arm description.

Sponsors & Collaborators

Principal Investigators

  • Vinay Penematsa, MD · PTC Therapeutics

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-06-22
Primary Completion
2025-04-15
Completion
2025-04-15
FDA Drug
Yes

Countries

  • United States
  • France
  • Italy
  • Japan
  • Poland
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05218655 on ClinicalTrials.gov