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A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia (MOVE-FA)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT04577352
Recruitment Status : Completed
First Posted : October 6, 2020
Last Update Posted : March 27, 2024
Information provided by (Responsible Party):
PTC Therapeutics

Brief Summary:
The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale [mFARS]) and safety of vatiquinone in participants with Friedreich ataxia (FA).

Condition or disease Intervention/treatment Phase
Friedreich Ataxia Drug: Vatiquinone Drug: Placebo Phase 2 Phase 3

Detailed Description:

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 146 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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 : December 17, 2020
Actual Primary Completion Date : April 4, 2023
Actual Study Completion Date : October 2, 2023

Arm Intervention/treatment
Experimental: Vatiquinone
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.
Drug: Vatiquinone
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.
Drug: Vatiquinone
Vatiquinone will be administered per dose and schedule specified in the arm.

Drug: Placebo
Placebo will be administered per schedule specified in the arm.

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   7 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • mFARS ≥20 to ≤70 at baseline
  • Must be able to ambulate at least 10 feet in 1 minute with or without assistance (non-wheelchair).
  • Friedreich ataxia diagnosis (homozygous for guanine-adenine-adenine [GAA] repeat expansion in intron-1 of frataxin [FXN] gene), confirmed by clinical testing (Note: size of GAA repeat is not required for eligibility)
  • Consent to comply with study procedures. For participants under the age of 18 (or age of consent), parent(s)/legal guardian(s) of the participant must agree to comply with the requirements of the study, including the need for frequent and prolonged follow up; parent(s)/legal guardian(s) with custody of the participant must give their consent for participant to enroll in the study.
  • Difference in the mFARS at screening and baseline of no more than 4 points.
  • Must be able to abstain from anticoagulants and any aspirin (including 81 mg) for 30 days prior to 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 potent 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
  • Must be able to swallow capsules
  • Males and females of childbearing potential must be willing to use an effective method of contraception from the time consent is signed until 30 days after the last dose of study drug or early termination visit. Male participants must agree not to donate sperm during the study and for at least 30 days after the last dose of study drug or early termination visit.

Exclusion Criteria:

  • Individuals with clinical diagnosis of FA who have point mutations or deletions or other non-GAA expansion mutations
  • Previous treatment with vatiquinone
  • Allergy to vatiquinone, sesame oil, gelatin (bovine and/or porcine), titanium dioxide, or red iron oxide
  • Ejection fraction <50%
  • Uncontrolled diabetes (glycated hemoglobin [HbA1c] >7.0%) at the time of screening
  • Has current suicidal ideation based on Columbia-Suicide Severity Rating Scale (C-SSRS) within 3 months prior to screening or between screening and baseline at the baseline visit or suicidal behavior within the last year at the screening visit or between screening and baseline at the baseline visit
  • Pregnant or lactating participants or those sexually active participants who are unwilling to comply with proper birth control methods; females of childbearing potential must have a negative pregnancy test at screening and during the baseline visit
  • Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) ≥2 * upper limit of normal (ULN) at time of screening
  • International normalized ratio (INR) ≥1.5 * ULN at time of screening or clinically significant (CS) bleeding, as determined by the investigator
  • Serum creatinine ≥1.5 * ULN at time of screening
  • Comorbidities that may confound study results (for example, fat malabsorption syndrome, other mitochondrial disorder) in the opinion of the investigator
  • Participation in any other interventional clinical trial or received any investigational 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 coenzyme Q10 (CoQ10), vitamin E, or any approved or non-approved medication for FA within 30 days prior to the screening visit. These prohibited medications can be discontinued at the screening visit; if this is the case, the mFARS assessment must be repeated to confirm inclusion eligibility after a minimum of 30 days post-discontinuation and there must be no more than a 4-point difference in mFARS assessed from the post-discontinuation visit to the baseline visit.
  • Illicit drug use 30 days prior to screening and during the study is prohibited.

Information from the National Library of Medicine

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 identifier (NCT number): NCT04577352

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United States, California
Los Angeles, California, United States, 90095
United States, Florida
University of Florida
Gainesville, Florida, United States, 32608
University of South Florida
Tampa, Florida, United States, 33612
United States, Iowa
University of Iowa
Iowa City, Iowa, United States, 52242
United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Australia, Victoria
Murdoch Children's Research Institute
Parkville, Victoria, Australia, 3052
University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology
São Paulo, Brazil, 13083-887
Canada, Quebec
Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)
Montreal, Quebec, Canada, H2X 0A9
CHU Sainte-Justine
Montréal, Quebec, Canada, H3T1C5
Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute)
PARIS cedex, France, 75646
Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE)
Tuebingen, Germany, 72076
Ospedale Pediatrico Bambino Gesu' IRCCS
Roma, Italy, 00165
New Zealand
CBR Neurogenetic Research Clinic, University of Auckland
Auckland, New Zealand, 1023
Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares
Barcelona, Spain, 08950
Sponsors and Collaborators
PTC Therapeutics
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Responsible Party: PTC Therapeutics Identifier: NCT04577352    
Other Study ID Numbers: PTC743-NEU-003-FA
First Posted: October 6, 2020    Key Record Dates
Last Update Posted: March 27, 2024
Last Verified: March 2024

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cerebellar Ataxia
Friedreich Ataxia
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