- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05218655
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
March 15, 2024 updated by: PTC Therapeutics
An Open-Label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial Disease
The primary objective of this study is to assess the safety of vatiquinone in participants with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron sponsored (previously Edison) clinical study or treatment plan.
The study will continue until vatiquinone becomes commercially available or the program is terminated.
Study Overview
Status
Enrolling by invitation
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Estimated)
100
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Angers Cedex 9, France, 49933
- Chu Angers
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Montpellier Cedex 5, France, 34295
- CHU Montpellier - Hopital Saint-Eloi
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Paris, France, 75015
- Hôpital Necker-Enfants Malades
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Strasbourg, France, 67000
- Hôpital de Hautepierre - Hôpitaux Universitaires de Strasbourg
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Roma, Italy, 00165
- Ospedale Pediatrico Bambino Gesù
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Japanese City, Japan
- PTC Clinical Site
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Warszawa, Poland, 04-730
- Instytut Pomnik - Centrum Zdrowia Dziecka
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Barcelona, Spain, 08950
- Hospital Sant Joan de Déu
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Madrid, Spain, 28034
- Hospital Ruber Internacional
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Madrid, Spain, 28003
- Hospital Universitario 12 de Octubre
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London, United Kingdom, WC1N 3JH
- Great Ormond Street Hospital For Children NHS Foundation Trust
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Newcastle Upon Tyne, United Kingdom, NE1 4LP
- The Newcastle Upon Tyne Hospitals NHS Foundation Trust
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California
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La Jolla, California, United States, 92037
- University of California, San Diego Altman Clinical and Translational Research Institute
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Stanford, California, United States, 94305
- Stanford University
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Connecticut
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New Haven, Connecticut, United States, 06511
- Yale Medicine
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District of Columbia
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Washington, District of Columbia, United States, 20010
- Children's National
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Florida
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Gulf Breeze, Florida, United States, 32561
- Child Neurology Center of Northwest Florida
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Maryland
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Baltimore, Maryland, United States, 21205
- The Johns Hopkins University
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New York
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New York, New York, United States, 10032
- Columbia University Medical Center - CUMC
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Ohio
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Akron, Ohio, United States, 44308
- Akron Children's Hospital
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Cleveland, Ohio, United States, 44195
- Cleveland Clinic
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104
- Children's Hospital of Philadelphia - CHOP
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South Carolina
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Charleston, South Carolina, United States, 29425
- Medical University of South Carolina - MUSC
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Texas
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Houston, Texas, United States, 77030
- Baylor College of Medicine
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Houston, Texas, United States, 77030
- UT Health The University of Texas
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Washington
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Seattle, Washington, United States, 98105
- Seattle Children Hospital
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- Participants with inherited mitochondrial disease including Leigh syndrome, Alpers syndrome, mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS), myoclonic epilepsy with ragged-red fibers (MERRF), pontocerebellar hypoplasia type 6 (PCH6), or other mitochondrial disease who participated in a previous vatiquinone clinical study or treatment plan.
- Women of childbearing potential must have a negative pregnancy test at screening/baseline and agree to abstinence or the use of at least 1 of the highly effective forms of contraception as specified in the protocol (with a failure rate of <1% per year when used consistently and correctly). Highly effective contraception or abstinence must be continued for the duration of the study, and for up to 30 days after the last dose of study drug.
- Fertile men who are sexually active with women of childbearing potential and who have not had a vasectomy, must agree to use a barrier method of birth control during the study and for up to 30 days after the last dose of study drug.
Exclusion Criteria:
- Current participation in any other interventional study.
- Pregnancy or breast feeding.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Vatiquinone
Participants will receive vatiquinone oral solution (100 milligrams [mg]/milliliter [mL]), up to 400 mg, administered orally or via feeding tube 3 times daily (TID).
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Vatiquinone will be administered per dose and schedule specified in the arm description.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
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Number of Participants With Adverse Events (AEs)
Time Frame: Baseline (Day 1) up to end of study (up to approximately 3 years)
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Baseline (Day 1) up to end of study (up to approximately 3 years)
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Vinay Penematsa, MD, PTC Therapeutics
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
June 21, 2022
Primary Completion (Estimated)
March 31, 2025
Study Completion (Estimated)
March 31, 2025
Study Registration Dates
First Submitted
January 19, 2022
First Submitted That Met QC Criteria
January 19, 2022
First Posted (Actual)
February 1, 2022
Study Record Updates
Last Update Posted (Actual)
March 18, 2024
Last Update Submitted That Met QC Criteria
March 15, 2024
Last Verified
March 1, 2024
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- PTC743-CNS-005-LSEP
- 2022-000375-39 (EudraCT Number)
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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