A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
NCT ID: NCT05515536
Last Updated: 2025-10-30
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
130 participants
INTERVENTIONAL
2022-12-08
2027-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Vatiquinone
Participants with FA who were previously treated with vatiquinone at an investigational site and completed participation in a prior PTC Therapeutics (PTC)-sponsored clinical study will continue to receive the same dose/formulation of vatiquinone (unless there has been a change in age and/or weight that meets the criteria for a different dose/formulation as described below; dose/formulation will also be changed if participants meet the criteria for a different dose/formulation during study treatment): If \<7 years of age, participants will receive an oral solution (100 milligrams \[mg\]/milliliter \[mL\]) 3 times a day (TID) at one of the following doses: 15 mg/kilogram (kg) if body weight \<13 kg, or 200 mg if body weight ≥13 kg. If ≥7 years of age, participants will receive a capsule formulation (200 mg) orally TID at one of the following doses: 200 mg if body weight ˂25 kg, or 400 mg if body weight ≥25 kg.
Vatiquinone
Vatiquinone will be administered per dose and schedule specified in the arm.
Interventions
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Vatiquinone
Vatiquinone will be administered per dose and schedule specified in the arm.
Eligibility Criteria
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Inclusion Criteria
* Males and females of childbearing potential must be willing to use an effective method of contraception (for example, implants, injectables, transdermal patches, combined oral contraceptives, barrier methods, and intrauterine devices) from the time consent is signed until 30 days after the last dose of study drug or Early Termination Visit.
Exclusion Criteria
* Pregnancy or breastfeeding
ALL
No
Sponsors
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PTC Therapeutics
INDUSTRY
Responsible Party
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Locations
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UCLA
Los Angeles, California, United States
University of Florida
Gainesville, Florida, United States
University of South Florida
Tampa, Florida, United States
University of Iowa
Iowa City, Iowa, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Murdoch Children's Research Institute
Parkville, Victoria, Australia
University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology
São Paulo, , Brazil
Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)
Montreal, Quebec, Canada
CHU Sainte-Justine
Montreal, Quebec, Canada
Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute)
Paris, , France
Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE)
Tübingen, , Germany
Ospedale Pediatrico Bambino Gesu' IRCCS
Roma, , Italy
CBR Neurogenetic Research Clinic, University of Auckland
Auckland, , New Zealand
Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares
Barcelona, , Spain
Countries
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Other Identifiers
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2022-002668-65
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
2024-516505-23-00
Identifier Type: OTHER
Identifier Source: secondary_id
PTC743-NEU-003e-FA
Identifier Type: -
Identifier Source: org_study_id
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