A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia

NCT05515536 | Active Not Recruiting Phase 3 FDA Drug

• 3 other identifiers: PTC743-NEU-003e-FA2022-002668-652024-516505-23-00
• Study Type: interventional
• Target: 130
• Locations: 9 countries
• Sites: 14

Brief Summary

The primary objective of this study is to assess the long-term safety of vatiquinone in participants with Friedreich ataxia (FA) previously exposed to vatiquinone.

Conditions

Friedreich Ataxia

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Adverse Events (AEs)

  Baseline up to 3 years

Secondary Outcomes (1)

• Change From Baseline in the Modified Friedreich Ataxia Rating Scale (mFARS) and its 4 Subscales (Upright Stability, Upper Limb, Lower Limb, Bulbar) Scores at Year 3

  Baseline, Year 3

Study Arms (1)

Vatiquinone

EXPERIMENTAL

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.

Drug: Vatiquinone

Interventions

Vatiquinone DRUG

Vatiquinone will be administered per dose and schedule specified in the arm.

Vatiquinone

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants with FA who completed and directly rolled over from a previous vatiquinone clinical study.
• 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.

You may not qualify if:

• Current participation in any other interventional study
• Pregnancy or breastfeeding

Sponsors & Collaborators

• PTC Therapeutics: lead

Study Sites (14)

UCLA

Los Angeles, California, 90095, United States

Location

University of Florida

Gainesville, Florida, 32608, United States

Location

University of South Florida

Tampa, Florida, 33612, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Murdoch Children's Research Institute

Parkville, Victoria, 3052, Australia

Location

University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology

São Paulo, 13083-887, Brazil

Location

Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)

Montreal, Quebec, H2X 0A9, Canada

Location

CHU Sainte-Justine

Montreal, Quebec, H3T1C5, Canada

Location

Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute)

Paris, 75646, France

Location

Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE)

Tübingen, 72076, Germany

Location

Ospedale Pediatrico Bambino Gesu' IRCCS

Roma, 00165, Italy

Location

CBR Neurogenetic Research Clinic, University of Auckland

Auckland, 1023, New Zealand

Location

Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares

Barcelona, 08950, Spain

Location

MeSH Terms

Conditions

Friedreich Ataxia

Interventions

alpha-tocotrienol quinone

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 23, 2022
• First Posted: August 25, 2022
• Study Start: December 8, 2022
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: October 30, 2025

Record last verified: 2025-10

Locations

DE (1); IT (1); NZ (1); FR (1); US (5); AU (1); BR (1); ES (1); CA (2)