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Vatiquinone Expanded Access Protocol

Expanded Access Protocol for Vatiquinone in Previously Treated Patients With Inherited Mitochondrial Disease

Status
NO_LONGER_AVAILABLE
Phases
Unknown
Study type
Expanded Access
Source
ClinicalTrials.gov
Registry ID
NCT07159139
Acronym
EAP
Enrollment
Unknown
Registered
2025-09-08
Start date
Unknown
Completion date
Unknown
Last updated
2026-04-09

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Mitochondrial Disease

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.

Detailed description

This expanded access protocol serves to make Vatiquinone accessible to subjects with inherited mitochondrial disease who previously participated in a Vatiquinone clinical trial at the Medical University of South Carolina (MUSC) who in the opinion of the investigator would benefit from continued access to liquid Vatiquinone.

Interventions

DRUGVatiquinone

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

Sponsors

Medical University of South Carolina
Lead SponsorOTHER
PTC Therapeutics
CollaboratorINDUSTRY

Eligibility

Sex/Gender
ALL
Age
1 Years to No maximum

Inclusion criteria

* 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

Exclusion criteria

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

Contacts

PRINCIPAL_INVESTIGATORThomas Koch, MD

Medical University of South Carolina

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Apr 10, 2026