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| ID | Type | Description | Link |
|---|---|---|---|
| 2022-002668-65 | EudraCT Number | ||
| 2024-516505-23-00 | Other Identifier | EU CT Number |
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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.
This long-term, open-label study will serve as a continued access study for participants with FA who have previously participated in a vatiquinone study. The purpose of this study is to assess continued safety and efficacy of vatiquinone dosing in previously treated participants. This study addresses the medical need for participants to continue vatiquinone with a planned study duration of 3 years.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Vatiquinone | Experimental | 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 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. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Vatiquinone | Drug | Vatiquinone will be administered per dose and schedule specified in the arm. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Events (AEs) | Baseline up to 3 years |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in the Modified Friedreich Ataxia Rating Scale (mFARS) and its 4 Subscales (Upright Stability, Upper Limb, Lower Limb, Bulbar) Scores at Year 3 | Baseline, Year 3 |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCLA | Los Angeles | California | 90095 | United States | ||
| University of Florida |
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| Gainesville |
| Florida |
| 32608 |
| United States |
| University of South Florida | Tampa | Florida | 33612 | United States |
| University of Iowa | Iowa City | Iowa | 52242 | United States |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Murdoch Children's Research Institute | Parkville | Victoria | 3052 | Australia |
| University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology | São Paulo | 13083-887 | Brazil |
| Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM) | Montreal | Quebec | H2X 0A9 | Canada |
| CHU Sainte-Justine | Montreal | Quebec | H3T1C5 | Canada |
| Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute) | Paris | 75646 | France |
| Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE) | Tübingen | 72076 | Germany |
| Ospedale Pediatrico Bambino Gesu' IRCCS | Roma | 00165 | Italy |
| CBR Neurogenetic Research Clinic, University of Auckland | Auckland | 1023 | New Zealand |
| Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares | Barcelona | 08950 | Spain |
| ID | Term |
|---|---|
| D005621 | Friedreich Ataxia |
| ID | Term |
|---|---|
| D013132 | Spinocerebellar Degenerations |
| D002526 | Cerebellar Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D013118 | Spinal Cord Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D028361 | Mitochondrial Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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| ID | Term |
|---|---|
| C571746 | alpha-tocotrienol quinone |
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