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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT05515536
Recruitment Status : Not yet recruiting
First Posted : August 25, 2022
Last Update Posted : October 24, 2022
Information provided by (Responsible Party):
PTC Therapeutics

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 in Study PTC743-NEU-003-FA (NCT04577352) or Study PTC743-NEU-005-FA.

Condition or disease Intervention/treatment Phase
Friedreich Ataxia Drug: Vatiquinone Phase 3

Detailed Description:
Treatment will continue until the participant withdraw consent, regulatory authority and/or PTC discontinue the study, participant's condition substantially worsens after initiating vatiquinone treatment in this study, investigator withdraws the participant from vatiquinone treatment, if, in the investigator's clinical judgment, it is not in the participant's best interest to continue, or vatiquinone becomes commercially available in a participant's country.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 140 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia
Estimated Study Start Date : November 1, 2022
Estimated Primary Completion Date : December 31, 2027
Estimated Study Completion Date : December 31, 2027

Arm Intervention/treatment
Experimental: 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 (PTC743-NEU-003-FA or PTC743-NEU-005-FA) 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
Vatiquinone will be administered per dose and schedule specified in the arm.

Primary Outcome Measures :
  1. Number of Participants With Adverse Events (AEs) [ Time Frame: Baseline up to approximately 5 years ]

Secondary Outcome Measures :
  1. Change From Baseline in the Modified Friedreich Ataxia Rating Scale (mFARS) at Year 5 [ Time Frame: Baseline, Year 5 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Participants with FA who completed and directly rolled over from a previous vatiquinone clinical study (PTC743-NEU-003-FA [that is, MOVE-FA] or PTC743-NEU-005-FA).
  • 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:

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05515536

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Contact: Patient Advocacy 1-866-562-4620 medinfo@ptcbio.com

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United States, California
Los Angeles, California, United States, 90095
Contact: Study Coordinator    310-206-8153      
United States, Florida
University of Florida
Gainesville, Florida, United States, 32608
Contact: Study Coordinator    352-733-2435      
University of South Florida
Tampa, Florida, United States, 33612
Contact: Study Coordinator    813-974-4685      
United States, Iowa
University of Iowa
Iowa City, Iowa, United States, 52242
Contact: Study Coordinator    319-335-7617      
United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Contact: Study Coordinator    267-426-9567      
Australia, Victoria
Murdoch Children's Research Institute
Parkville, Victoria, Australia, 3052
Contact: Study Coordinator    +613-83416374      
University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology
São Paulo, Brazil, 13083-887
Contact: Study Coordinator    55 19 3521 9217      
Canada, Quebec
Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)
Montreal, Quebec, Canada, H2X 0A9
Contact: Study Coordinator    514-890-8000 30154      
CHU Sainte-Justine
Montréal, Quebec, Canada, H3T1C5
Contact: Study Coordinator    514-345-4931 5407      
Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute)
PARIS cedex, France, 75646
Contact: Study Coordinator    33 1 57 27 46 91      
Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE)
Tuebingen, Germany, 72076
Contact: Study Coordinator    0049 707129-85247      
Ospedale Pediatrico Bambino Gesu' IRCCS
Roma, Italy, 00165
Contact: Study coordinator    39 06 68592105      
New Zealand
CBR Neurogenetic Research Clinic, University of Auckland
Auckland, New Zealand, 1023
Contact: Study Coordinator    0 274790725      
Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares
Barcelona, Spain, 08950
Sponsors and Collaborators
PTC Therapeutics
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Responsible Party: PTC Therapeutics
ClinicalTrials.gov Identifier: NCT05515536    
Other Study ID Numbers: PTC743-NEU-003e-FA
2022-002668-65 ( EudraCT Number )
First Posted: August 25, 2022    Key Record Dates
Last Update Posted: October 24, 2022
Last Verified: October 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cerebellar Ataxia
Friedreich Ataxia
Neurologic Manifestations
Nervous System Diseases
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinocerebellar Degenerations
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases