- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05485987
A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
April 8, 2024 updated by: PTC Therapeutics
An Open-Label Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Vatiquinone in Children With Friedreich Ataxia Younger Than 7 Years of Age
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.
Study Overview
Status
Active, not recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
5
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104
- The Children's Hospital of Philadelphia
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
No older than 6 years (Child)
Accepts Healthy Volunteers
No
Description
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Vatiquinone
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.
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Vatiquinone will be administered per dose and schedule specified in the arm.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
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
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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
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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
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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
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Number of Participants With Adverse Events
Time Frame: Baseline up to Week 76
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Baseline up to Week 76
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
October 13, 2022
Primary Completion (Estimated)
October 5, 2024
Study Completion (Estimated)
October 5, 2024
Study Registration Dates
First Submitted
August 1, 2022
First Submitted That Met QC Criteria
August 1, 2022
First Posted (Actual)
August 3, 2022
Study Record Updates
Last Update Posted (Actual)
April 9, 2024
Last Update Submitted That Met QC Criteria
April 8, 2024
Last Verified
April 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Neurologic Manifestations
- Genetic Diseases, Inborn
- Neurodegenerative Diseases
- Dyskinesias
- Spinal Cord Diseases
- Heredodegenerative Disorders, Nervous System
- Mitochondrial Diseases
- Cerebellar Diseases
- Spinocerebellar Degenerations
- Ataxia
- Cerebellar Ataxia
- Friedreich Ataxia
Other Study ID Numbers
- PTC743-NEU-005-FA
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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