Mitochondrial DiseaseEAPNCT07159139
Vatiquinone Expanded Access Protocol
A study testing Vatiquinone in people with Mitochondrial Disease, run by Medical University of South Carolina.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Mitochondrial Disease
- What is being tested?
- Vatiquinone
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- Current participation in any other interventional study
- Pregnant or currently breast feeding.
About this study
Official title: Expanded Access Protocol for Vatiquinone in Previously Treated Patients With Inherited Mitochondrial Disease
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.
Read the 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
- Mitochondrial Disease
- Being tested
- Vatiquinone
- Medical subject headings
- Mitochondrial Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VatiquinoneDrug
Vatiquinone will be administered per dose and schedule specified in the arm description.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Sep 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mitochondrial DiseaseStudies of the same condition, open and under way.
- More trials run by Medical University of South CarolinaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Medical University of South Carolina
- Sponsor type
- University, hospital or other
- Collaborators
- PTC Therapeutics
- Responsible party
- Principal investigator, Thomas Koch, Medical University of South Carolina
- Investigators
- Thomas Koch, MD, Principal investigator, Medical University of South Carolina
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07159139
- Sponsor's protocol ID
- Pro00144275
- Acronym
- EAP
- Status last verified
- Mar 2026
- First submitted
- 18 Jun 2025
- Last updated
- 9 Apr 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Protocol for Vatiquinone in Previously Treated Patients With Inherited Mitochondrial Disease
- ClinicalTrials.gov ID
- NCT07159139
- Acronym / sponsor ID
- EAP / Pro00144275
- Conditions
- Mitochondrial Disease
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Medical University of South CarolinaUniversity, hospital or other
- Collaborators
- PTC Therapeutics
- First posted
- 8 Sep 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (2 listed)
- Current participation in any other interventional study
- Pregnant or currently breast feeding.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VatiquinoneVatiquinone will be administered per dose and schedule specified in the arm description. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Apr 2026.
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Mitochondrial Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Thomas Koch, MD principal investigator
Medical University of South Carolina
Questions about this trial
What is this clinical trial studying?
A study testing Vatiquinone in people with Mitochondrial Disease, run by Medical University of South Carolina.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 9 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Apr 2026.