A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
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ClinicalTrials.gov Identifier: NCT05485987 |
Recruitment Status :
Active, not recruiting
First Posted : August 3, 2022
Last Update Posted : April 9, 2024
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Condition or disease | Intervention/treatment | Phase |
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Friedreich Ataxia | Drug: Vatiquinone | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 5 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
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 |
Actual Study Start Date : | October 13, 2022 |
Estimated Primary Completion Date : | October 5, 2024 |
Estimated Study Completion Date : | October 5, 2024 |
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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Drug: Vatiquinone
Vatiquinone will be administered per dose and schedule specified in the arm.
Other Names:
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- 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 ]
- 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 ]
- Number of Participants With Adverse Events [ Time Frame: Baseline up to Week 76 ]
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Ages Eligible for Study: | up to 6 Years (Child) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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.
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): NCT05485987
United States, Pennsylvania | |
The Children's Hospital of Philadelphia | |
Philadelphia, Pennsylvania, United States, 19104 |
Responsible Party: | PTC Therapeutics |
ClinicalTrials.gov Identifier: | NCT05485987 |
Other Study ID Numbers: |
PTC743-NEU-005-FA |
First Posted: | August 3, 2022 Key Record Dates |
Last Update Posted: | April 9, 2024 |
Last Verified: | April 2024 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Ataxia Cerebellar Ataxia Friedreich Ataxia Dyskinesias 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 |