NCT07159139

Brief Summary

The purpose of this study is to provide participants who successfully completed the study titled "An Open-label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial Disease" with liquid Vatiquinone through an intermediate patient population expanded access protocol.

Trial Health

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 18, 2025

Completed
3 months until next milestone

First Posted

Study publicly available on registry

September 8, 2025

Completed
Last Updated

April 9, 2026

Status Verified

March 1, 2026

First QC Date

June 18, 2025

Last Update Submit

April 3, 2026

Conditions

Mitochondrial Disease

Interventions

VatiquinoneDRUG

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

Eligibility Criteria

Age1 Year+
Sexall
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Subjects with molecular genetic mitochondrial disease including Leigh syndrome, and Alpers syndrome.
  • Completed participation in the study titled "An Open-label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial Disease"
  • In the opinion of the clinical investigator, the continuation to receive Vatiquinone will be of benefit to the participants.
  • Enrollment into a clinical trial to receive Vatiquinone is not possible

You may not qualify if:

  • Current participation in any other interventional study
  • Pregnant or currently breast feeding.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Mitochondrial Diseases

Interventions

alpha-tocotrienol quinone

Condition Hierarchy (Ancestors)

Metabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Thomas Koch, MD

    Medical University of South Carolina

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
expanded access
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor-Faculty

Study Record Dates

First Submitted

June 18, 2025

First Posted

September 8, 2025

Last Updated

April 9, 2026

Record last verified: 2026-03