Clinical Trials Directory

Trials / No Longer Available

No Longer AvailableNCT07159139

Vatiquinone Expanded Access Protocol

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

Status
No Longer Available
Phase
Study type
Expanded Access
Enrollment
Sponsor
Medical University of South Carolina · Academic / Other
Sex
All
Age
1 Year
Healthy volunteers

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.

Conditions

Interventions

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

Timeline

First posted
2025-09-08
Last updated
2026-04-09

Source: ClinicalTrials.gov record NCT07159139. Inclusion in this directory is not an endorsement.