A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia (MOVE-FA)
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|ClinicalTrials.gov Identifier: NCT04577352|
Recruitment Status : Recruiting
First Posted : October 6, 2020
Last Update Posted : August 25, 2021
|Condition or disease||Intervention/treatment||Phase|
|Friedreich Ataxia||Drug: Vatiquinone Drug: Placebo||Phase 2 Phase 3|
During the double-blind, placebo-controlled phase, participants will be stratified by baseline mFARS score (<40 versus ≥40), age of disease onset (<14 versus ≥14), and age at screening (≤21 years or >21 years) and randomized to receive either vatiquinone or placebo using interactive response technology (IRT). Following completion of the randomized, double-blind, placebo-controlled phase (72 weeks), participants will enter into an open-label extension phase (24 weeks) during which they will receive open-label treatment with vatiquinone at the dose they received in the randomized phase of the study (for participants entering the extension phase who initially received placebo, the dose of vatiquinone will be determined based on age and weight) and then a safety follow-up (approximately 30 days [±5 days] after last dose or termination visit, whichever is later).
The primary efficacy analysis will be based on change from baseline in mFARS score of participants between 7 and 21 years old. In order to explore the treatment efficacy and safety, approximately an additional 20 participants >21 years of age will be randomized for a total of approximately 126 participants.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||126 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Investigator)|
|Official Title:||A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study With Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich Ataxia (MOVE-FA)|
|Actual Study Start Date :||November 23, 2020|
|Estimated Primary Completion Date :||March 21, 2023|
|Estimated Study Completion Date :||July 6, 2023|
Participants will receive vatiquinone capsule at a dose of either 200 milligrams (mg) orally 3 times a day (TID) if ˂12 years of age and weighing ˂25 kilograms (kg) or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the placebo-controlled phase and for 24 weeks during the open-label extension phase.
Vatiquinone will be administered per dose and schedule specified in the arm.
Placebo Comparator: Placebo
Participants will receive placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the placebo-controlled phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label extension phase.
Vatiquinone will be administered per dose and schedule specified in the arm.
Placebo will be administered per schedule specified in the arm.
- Change From Baseline in the Modified Friedreich Ataxia Rating Scale (mFARS) Score at Week 72 [ Time Frame: Baseline, Week 72 ]Friedreich Ataxia Rating Scale (FARS) is a disease-specific scale that measures progression of neurological effects of FA. The mFARS is a validated and reliable 93- item scale; comprised of the neurologic component of the FARS and evaluates bulbar, upper limb, lower limb, and upright stability/gait function. For each item, responses categorize the corresponding neurological finding, and the findings are assigned a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment.
- Change From Baseline in Friedreich Ataxia Rating Scale Activities of Daily Living (FARS-ADL) Score at Week 72 [ Time Frame: Baseline, Week 72 ]The FARS-ADL is a subsection of the FARS questionnaire that assesses activities of daily living, including speech, personal hygiene, feeding, and mobility. Participants rank each category using a scale of 0 (normal) to 4 (severe disability/ inability to carry out activity independently), with lower scores indicative of "normal" function/activity.
- Change From Baseline in 1-Minute Walk Test (1MWT) at Week 72 [ Time Frame: Baseline, Week 72 ]The 1MWT is a timed performance test used to measure functional ability, walking endurance, balance, and muscle performance by measuring maximal walking speed in 1 minute. Participants will be instructed to walk as quickly as possible for 1 minute without running. Maximal walking speed will be measured upon completion of the walk and recorded.
- Number of Falls Through Week 72 [ Time Frame: Baseline through Week 72 ]The fall log directly relate to a participant's ability to ambulate during normal daily activities. Thus, each participant will be required to maintain a fall log, which will include the date and time of each fall. Falls as defined by World Health Organization as "inadvertently coming to rest on the ground, floor or other lower level, excluding intentional change in position to rest in furniture, wall or other objects," will be reported.
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): NCT04577352
|Contact: Patient Advocacyemail@example.com|
|United States, California|
|Los Angeles, California, United States, 90095|
|Contact: Study Coordinator 310-206-8153|
|United States, Florida|
|University of Florida||Not yet recruiting|
|Gainesville, Florida, United States, 32608|
|Contact: Study Coordinator 352-733-2435|
|University of South Florida||Recruiting|
|Tampa, Florida, United States, 33612|
|Contact: Study Coordinator 813-974-4685|
|United States, Iowa|
|University of Iowa||Recruiting|
|Iowa City, Iowa, United States, 52242|
|Contact: Study Coordinator 319-335-7617|
|United States, Pennsylvania|
|The Children's Hospital of Philadelphia||Recruiting|
|Philadelphia, Pennsylvania, United States, 19104|
|Contact: Study Coordinator 267-426-9567|
|Murdoch Children's Research Institute||Recruiting|
|Parkville, Victoria, Australia, 3052|
|Contact: Study Coordinator +613-83416374|
|University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology||Recruiting|
|São Paulo, Brazil, 13083-887|
|Contact: Study Coordinator 55 19 3521 9217|
|Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)||Recruiting|
|Montreal, Quebec, Canada, H2X 0A9|
|Contact: Study Coordinator 514-890-8000 ext 30154|
|CHU Sainte-Justine||Not yet recruiting|
|Montréal, Quebec, Canada, H3T1C5|
|Contact: Study Coordinator 514-345-4931 ext 5407|
|Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute)||Recruiting|
|PARIS cedex, France, 75646|
|Contact: Study Coordinator 33 1 57 27 46 91|
|Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE)||Recruiting|
|Tuebingen, Germany, 72076|
|Contact: Study Coordinator 0049 707129-85247|
|Ospedale Pediatrico Bambino Gesu' IRCCS||Not yet recruiting|
|Roma, Italy, 00165|
|Contact: Study coordinator 39 06 68592105|
|CBR Neurogenetic Research Clinic, University of Auckland||Recruiting|
|Auckland, New Zealand, 1023|
|Contact: Study Coordinator 0 274790725|
|Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares||Not yet recruiting|
|Barcelona, Spain, 08950|