Back to resultsA Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
Primary Purpose
Friedreich Ataxia
Status
Active
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Vatiquinone
Sponsored by
About this trial
This is an interventional treatment trial for Friedreich Ataxia
Eligibility Criteria
Inclusion Criteria:
- Friedreich ataxia diagnosis (homozygous for GAA repeat expansion in intron-1 of FXN gene), confirmed by clinical genetic testing.
- Must be able to abstain from anticoagulants and any aspirin (including 81 mg) for 30 days before the Baseline Visit and for the duration of the study; any possible discontinuation of anticoagulants should be monitored and indicated by a specialist (for example, cardiologist, neurologist, or hematologist), and discontinuation will be noted by the prescribing physician.
- Must be able to abstain from strong cytochrome P450 (CYP) 3A4 inducers/inhibitors (for example, ketoconazole, rifampin, St. John's wort, grapefruit juice, or any grapefruit product) for at least 30 days prior to enrollment and for the duration of the study.
- Must be able to take vatiquinone oral solution with food.
Exclusion Criteria:
- Previous treatment with vatiquinone.
- Allergy to vatiquinone or sesame oil.
- Ejection fraction <50%.
- Participation in any other interventional clinical trial or receipt of any study drug in any other clinical trial within 60 days prior to the Baseline Visit. Participants may be rescreened after the exclusionary period of 60 days has passed.
- Concomitant use of interventional CoQ10, vitamin E, or any approved or non-approved medication for FA within 30 days prior to the Baseline Visit. These prohibited medications can be discontinued at the Screening Visit.
Note: Other inclusion and exclusion criteria may apply.
Sites / Locations
- The Children's Hospital of Philadelphia
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Vatiquinone
Arm Description
Participants will receive an oral solution (100 milligrams [mg]/milliliter [mL]) of vatiquinone (15 mg/kilogram [kg] if body weight <13 kg and 200 mg if body weight ≥13 kg) 3 times a day (TID) for 72 weeks.
Outcomes
Primary Outcome Measures
Plasma Concentration of Vatiquinone
Area Under the Curve (AUC) of Vatiquinone
Number of Participants With Adverse Events
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05485987
Brief Title
A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
Official Title
An Open-Label Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Vatiquinone in Children With Friedreich Ataxia Younger Than 7 Years of Age
Study Type
Interventional
2. Study Status
Record Verification Date
October 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
October 13, 2022 (Actual)
Primary Completion Date
October 5, 2024 (Anticipated)
Study Completion Date
October 5, 2024 (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
Data Monitoring Committee
No
5. Study Description
Brief Summary
The primary objective of the study is to assess the pharmacokinetics (PK) and safety of vatiquinone administered in participants with Friedreich ataxia (FA) younger than 7 years.
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 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
5 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Vatiquinone
Arm Type
Experimental
Arm Description
Participants will receive an oral solution (100 milligrams [mg]/milliliter [mL]) of vatiquinone (15 mg/kilogram [kg] if body weight <13 kg and 200 mg if body weight ≥13 kg) 3 times a day (TID) for 72 weeks.
Intervention Type
Drug
Intervention Name(s)
Vatiquinone
Other Intervention Name(s)
PTC743, EPI-743
Intervention Description
Vatiquinone will be administered per dose and schedule specified in the arm.
Primary Outcome Measure Information:
Title
Plasma Concentration of Vatiquinone
Time Frame
Pre-morning dose (0 hour) at Week 4; 1 to 3 hours and 3 to 6 hours post-morning dose at Weeks 4, 12, and 24
Title
Area Under the Curve (AUC) of Vatiquinone
Time Frame
Pre-morning dose (0 hour) at Week 4; 1 to 3 hours and 3 to 6 hours post-morning dose at Weeks 4, 12, and 24
Title
Number of Participants With Adverse Events
Time Frame
Baseline up to Week 76
10. Eligibility
Sex
All
Maximum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Friedreich ataxia diagnosis (homozygous for GAA repeat expansion in intron-1 of FXN gene), confirmed by clinical genetic testing.
Must be able to abstain from anticoagulants and any aspirin (including 81 mg) for 30 days before the Baseline Visit and for the duration of the study; any possible discontinuation of anticoagulants should be monitored and indicated by a specialist (for example, cardiologist, neurologist, or hematologist), and discontinuation will be noted by the prescribing physician.
Must be able to abstain from strong cytochrome P450 (CYP) 3A4 inducers/inhibitors (for example, ketoconazole, rifampin, St. John's wort, grapefruit juice, or any grapefruit product) for at least 30 days prior to enrollment and for the duration of the study.
Must be able to take vatiquinone oral solution with food.
Exclusion Criteria:
Previous treatment with vatiquinone.
Allergy to vatiquinone or sesame oil.
Ejection fraction <50%.
Participation in any other interventional clinical trial or receipt of any study drug in any other clinical trial within 60 days prior to the Baseline Visit. Participants may be rescreened after the exclusionary period of 60 days has passed.
Concomitant use of interventional CoQ10, vitamin E, or any approved or non-approved medication for FA within 30 days prior to the Baseline Visit. These prohibited medications can be discontinued at the Screening Visit.
Note: Other inclusion and exclusion criteria may apply.
Facility Information:
Facility Name
The Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
12. IPD Sharing Statement
Learn more about this trial
A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
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