Cure My Disease

Don't miss your cure!

 Log in / Sign up

Last chance access: vatiquinone for mitochondrial disease patients

NCT ID NCT07159139

Disease control Sponsor: Medical University of South Carolina Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended) This study
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This program offered vatiquinone, an experimental liquid medication, to patients with inherited mitochondrial diseases like Leigh syndrome who had already completed a previous safety study. The goal was to continue treatment for those who might benefit, but enrollment is now closed. It was not a cure, but aimed to help manage the disease.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

1 year and older

Sex

Anyone

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Alpers syndrome are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

inborn mitochondrial metabolism disorder Leigh syndrome mitochondrial disease mitochondrial DNA depletion syndrome 4a

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Mitochondrial disease

More trials for these conditions

Other studies related to the condition(s) this trial covers.