A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
NCT ID: NCT05218655
Last Updated: 2025-12-22
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
PHASE3
101 participants
INTERVENTIONAL
2022-06-22
2025-04-15
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
The study will continue until vatiquinone becomes commercially available or the program is terminated.
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Keywords
Explore important study keywords that can help with search, categorization, and topic discovery.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
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).
Vatiquinone
Vatiquinone will be administered per dose and schedule specified in the arm description.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Vatiquinone
Vatiquinone will be administered per dose and schedule specified in the arm description.
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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
* Pregnancy or breast feeding.
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
PTC Therapeutics
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Vinay Penematsa, MD
Role: STUDY_DIRECTOR
PTC Therapeutics
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
University of California, San Diego Altman Clinical and Translational Research Institute
La Jolla, California, United States
Stanford University
Stanford, California, United States
Yale Medicine
New Haven, Connecticut, United States
Children's National
Washington D.C., District of Columbia, United States
Child Neurology Center of Northwest Florida
Gulf Breeze, Florida, United States
The Johns Hopkins University
Baltimore, Maryland, United States
Columbia University Medical Center - CUMC
New York, New York, United States
Akron Children's Hospital
Akron, Ohio, United States
Cleveland Clinic
Cleveland, Ohio, United States
Children's Hospital of Philadelphia - CHOP
Philadelphia, Pennsylvania, United States
Medical University of South Carolina - MUSC
Charleston, South Carolina, United States
Baylor College of Medicine
Houston, Texas, United States
UT Health The University of Texas
Houston, Texas, United States
Seattle Children Hospital
Seattle, Washington, United States
Chu Angers
Angers, , France
CHU Montpellier - Hopital Saint-Eloi
Montpellier, , France
Hopital Necker-Enfants Malades
Paris, , France
Hôpital de Hautepierre - Hôpitaux Universitaires de Strasbourg
Strasbourg, , France
Ospedale Pediatrico Bambino Gesù
Roma, , Italy
PTC Clinical Site
Japanese City, , Japan
Instytut Pomnik - Centrum Zdrowia Dziecka
Warsaw, , Poland
Hospital Sant Joan de Déu
Barcelona, , Spain
Hospital Universitario 12 de Octubre
Madrid, , Spain
Hospital Ruber Internacional
Madrid, , Spain
Great Ormond Street Hospital for Children NHS Foundation Trust
London, , United Kingdom
The Newcastle Upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
Provided Documents
Download supplemental materials such as informed consent forms, study protocols, or participant manuals.
Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2022-000375-39
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
PTC743-CNS-005-LSEP
Identifier Type: -
Identifier Source: org_study_id