Back to resultsA Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
Primary Purpose
Friedreich Ataxia
Status
Enrolling by invitation
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Vatiquinone
Sponsored by
About this trial
This is an interventional treatment trial for Friedreich Ataxia
Eligibility Criteria
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
Sites / Locations
- UCLA
- University of Florida
- University of South Florida
- University of Iowa
- The Children's Hospital of Philadelphia
- Murdoch Children's Research Institute
- University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology
- Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)
- CHU Sainte-Justine
- Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute)
- Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE)
- Ospedale Pediatrico Bambino Gesu' IRCCS
- CBR Neurogenetic Research Clinic, University of Auckland
- Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Vatiquinone
Arm Description
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.
Outcomes
Primary Outcome Measures
Number of Participants With Adverse Events (AEs)
Secondary Outcome Measures
Change From Baseline in the Modified Friedreich Ataxia Rating Scale (mFARS) at Year 5
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05515536
Brief Title
A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
Official Title
Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia
Study Type
Interventional
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
December 8, 2022 (Actual)
Primary Completion Date
December 31, 2027 (Anticipated)
Study Completion Date
December 31, 2027 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
PTC Therapeutics
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Friedreich Ataxia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
140 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Vatiquinone
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Vatiquinone
Intervention Description
Vatiquinone will be administered per dose and schedule specified in the arm.
Primary Outcome Measure Information:
Title
Number of Participants With Adverse Events (AEs)
Time Frame
Baseline up to approximately 5 years
Secondary Outcome Measure Information:
Title
Change From Baseline in the Modified Friedreich Ataxia Rating Scale (mFARS) at Year 5
Time Frame
Baseline, Year 5
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
Facility Name
UCLA
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
University of Florida
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32608
Country
United States
Facility Name
University of South Florida
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
University of Iowa
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
The Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Murdoch Children's Research Institute
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
Facility Name
University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology
City
São Paulo
ZIP/Postal Code
13083-887
Country
Brazil
Facility Name
Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H2X 0A9
Country
Canada
Facility Name
CHU Sainte-Justine
City
Montréal
State/Province
Quebec
ZIP/Postal Code
H3T1C5
Country
Canada
Facility Name
Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute)
City
PARIS cedex
ZIP/Postal Code
75646
Country
France
Facility Name
Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE)
City
Tuebingen
ZIP/Postal Code
72076
Country
Germany
Facility Name
Ospedale Pediatrico Bambino Gesu' IRCCS
City
Roma
ZIP/Postal Code
00165
Country
Italy
Facility Name
CBR Neurogenetic Research Clinic, University of Auckland
City
Auckland
ZIP/Postal Code
1023
Country
New Zealand
Facility Name
Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares
City
Barcelona
ZIP/Postal Code
08950
Country
Spain
12. IPD Sharing Statement
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A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
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