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

Phase 3

Active, not recruiting

• Conditions: Inherited Mitochondrial Disease
• Interventions: Drug: Vatiquinone

• Registration Number: NCT05218655
• Lead Sponsor: PTC Therapeutics

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-01-19
• Study First Submitted QC: 2022-01-19
• Study First Posted: 2022-02-01
• Study Start: 2022-06-21
• Status Verified: 2024-08
• Last Update Submitted: 2025-03-02
• Last Update Posted: 2025-03-04
• Primary Completion: 2025-03-31
• Study Completion: 2025-03-31

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 102

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Vatiquinone	Vatiquinone	Participants will receive vatiquinone oral solution (100 milligrams \[mg\]/milliliter \[mL\]), up to 400 mg, administered orally or via feeding tube 3 times daily (TID).

Primary Outcome Measures

Name	Time	Method
Number of Participants With Adverse Events (AEs)	Baseline (Day 1) up to end of study (up to approximately 3 years)

Trial Locations

Locations (26)

University of California, San Diego Altman Clinical and Translational Research Institute; 🇺🇸 La Jolla, California, United States

Stanford University; 🇺🇸 Stanford, California, United States

Yale Medicine; 🇺🇸 New Haven, Connecticut, United States

Children's National; 🇺🇸 Washington, District of Columbia, United States

Child Neurology Center of Northwest Florida; 🇺🇸 Gulf Breeze, Florida, United States

The Johns Hopkins University; 🇺🇸 Baltimore, Maryland, United States

Columbia University Medical Center - CUMC; 🇺🇸 New York, New York, United States

Akron Children's Hospital; 🇺🇸 Akron, Ohio, United States

Cleveland Clinic; 🇺🇸 Cleveland, Ohio, United States

Children's Hospital of Philadelphia - CHOP; 🇺🇸 Philadelphia, Pennsylvania, United States

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