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Completed

NCT Number: NCT04577352

A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia

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).

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Key information

Conditions

Friedreich Ataxia Brain Diseases Central Nervous System Diseases Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolic Diseases Mitochondrial Diseases Nervous System Diseases Neurodegenerative Diseases Nutritional and Metabolic Diseases Spinal Cord Diseases Spinocerebellar Degenerations

Age range

7 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Murdoch Children's Research Institute, Parkville, Victoria, Australia

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Vatiquinone

Drug

Vatiquinone will be administered per dose and schedule specified in the arm.

Placebo

Drug

Placebo will be administered per schedule specified in the arm.

Primary outcomes

  1. Change From Baseline in the mFARS Score at Week 72 - Modified Intent-to-treat (mITT) Analysis Set

    Time frame: Baseline, Week 72

    mFARS is a 93-item scale; comprised of neurologic component of FARS. For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. Total mFARS scores for each subscale: bulbar (0 to 5), upper limb coordination (0 to 36), lower limb coordination (0 to 16), and upright stability (0 to 36). mFARS total score was a composite score of all 4 subscales, ranging from 0 (normal) to 93 (greater impairment). A lower score = better neurological function. Missing data was imputed using pattern mix model multiple imputation. Least square (LS) mean and standard error (SE) was calculated using mixed-model repeated measures (MMRM).

  2. Change From Baseline in the mFARS Score at Week 72 - Intent-to-treat (ITT) Analysis Set

    Time frame: Baseline, Week 72

    mFARS is a 93-item scale; comprised of neurologic component of FARS. For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. Total mFARS scores for each subscale: bulbar (0 to 5), upper limb coordination (0 to 36), lower limb coordination (0 to 16), and upright stability (0 to 36). mFARS total score was a composite score of all 4 subscales, ranging from 0 (normal) to 93 (greater impairment). A lower score = better neurological function. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM.

Secondary outcomes

  1. Change From Baseline in Friedreich Ataxia Rating Scale Activities of Daily Living (FARS-ADL) Score at Week 72 - mITT Analysis Set

    Time frame: Baseline, Week 72

    The ADL component of the FARS includes 9 subscales: speech, swallowing, cutting food and handling utensils, dressing, personal hygiene, falling, walking, quality of sitting position, and bladder function. Each of these subscales is rated on a 5-point scale where 0=normal to 4=severe disability/inability to carry out activity independently for a total possible score of 0 to 36, with higher scores representing greater disability/dependency. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM.

  2. Change From Baseline in FARS-ADL Score at Week 72 - ITT Analysis Set

    Time frame: Baseline, Week 72

    The ADL component of the FARS includes 9 subscales: speech, swallowing, cutting food and handling utensils, dressing, personal hygiene, falling, walking, quality of sitting position, and bladder function. Each of these subscales is rated on a 5-point scale where 0=normal to 4=severe disability/inability to carry out activity independently for a total possible score of 0 to 36, with higher scores representing greater disability/dependency. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM.

  3. Change From Baseline in 1-Minute Walk Test (1MWT) at Week 72 - mITT Analysis Set

    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 were instructed to walk as quickly as possible for 1 minute without running. Maximal walking speed was measured upon completion of the walk and distance was recorded. The mean change from baseline in the distance the participant walked is reported. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM.

  4. Change From Baseline in 1MWT at Week 72 - ITT Analysis Set

    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 were instructed to walk as quickly as possible for 1 minute without running. Maximal walking speed was measured upon completion of the walk and distance was recorded. The mean change from baseline in the distance the participant walked is reported. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM.

  5. Number of Falls Per 28 Days Over Every 24-Week Period - mITT Analysis Set

    Time frame: Week 1-24, Week 25-48, and Week 49-72

    Each participant was required to maintain a fall log, which included 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," were reported. Number of falls per 28 days during a time interval was calculated as the number of falls during the period divided by the number of days during the interval, and multiplied by 28. The falls that occurred on or after the first Loss of Ambulation visit were excluded from the analysis.

  6. Number of Falls Per 28 Days Over Every 24-Week Period - ITT Analysis Set

    Time frame: Week 1-24, Week 25-48, and Week 49-72

    Each participant was required to maintain a fall log, which included 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," were reported. Number of falls per 28 days during a time interval was calculated as the number of falls during the period divided by the number of days during the interval, and multiplied by 28. The falls that occurred on or after the first Loss of Ambulation visit were excluded from the analysis.

Other outcomes

  1. Change From Baseline in the Upright Stability Subscale of the mFARS at Week 72 - mITT Analysis Set

    Time frame: Baseline, Week 72

    mFARS is a 93-item scale; comprised of neurologic component of FARS. For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. The Upright Stability subscale of the mFARS includes following items: 1. Sitting posture (score: 0 to 4); 2a. Stance with feet apart and eyes open (score: 0 to 4); 2b. Stance with feet apart and eyes closed (score: 0 to 4); 3a. Stance with feet together and eyes open (score: 0 to 4); 3b. Stance with feet together and eyes closed (score: 0 to 4); 4. Tandem stance (score: 0 to 4); 5. Stance on dominant foot (score: 0 to 4); 6. Tandem walk (score: 0 to 3); 7. Gait (score: 0 to 5). The upright stability subscale total score ranges from 0 (normal) to 36 (greater impairment). A lower score = better neurological function. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM.

  2. Change From Baseline in the Upright Stability Subscale of the mFARS at Week 72 - ITT Analysis Set

    Time frame: Baseline, Week 72

    mFARS is a 93-item scale; comprised of neurologic component of FARS. For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. The Upright Stability subscale of the mFARS includes following items: 1. Sitting posture (score: 0 to 4); 2a. Stance with feet apart and eyes open (score: 0 to 4); 2b. Stance with feet apart and eyes closed (score: 0 to 4); 3a. Stance with feet together and eyes open (score: 0 to 4); 3b. Stance with feet together and eyes closed (score: 0 to 4); 4. Tandem stance (score: 0 to 4); 5. Stance on dominant foot (score: 0 to 4); 6. Tandem walk (score: 0 to 3); 7. Gait (score: 0 to 5). The upright stability subscale total score ranges from 0 (normal) to 36 (greater impairment). A lower score = better neurological function. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM.

  3. Change From Baseline in the Modified Fatigue Impact Scale (MFIS) Score at Week 72 - mITT Analysis Set

    Time frame: Baseline, Week 72

    The MFIS is a 21-item, reliable, validated instrument that has been utilized in many neurological disorders. It is a modified form of the Fatigue Impact Scale, a component of the Multiple Sclerosis Quality of Life Inventory. Each item was scored on a scale of 0 (never) to 4 (almost always). The total score was the sum of all item's score and ranged from 0 (no fatigue impact) to 84 (almost always impacted by fatigue). Higher scores indicated greater impact of fatigue on participant function.

  4. Change From Baseline in the MFIS Score at Week 72 - ITT Analysis Set

    Time frame: Baseline, Week 72

    The MFIS is a 21-item, reliable, validated instrument that has been utilized in many neurological disorders. It is a modified form of the Fatigue Impact Scale, a component of the Multiple Sclerosis Quality of Life Inventory. Each item was scored on a scale of 0 (never) to 4 (almost always). The total score was the sum of all item's score and ranged from 0 (no fatigue impact) to 84 (almost always impacted by fatigue). Higher scores indicated greater impact of fatigue on participant function.

Sponsors and collaborators

Lead sponsor

PTC Therapeutics

Industry

Registry information

Official study 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)

Acronym: MOVE-FA

Important dates

Study start
2020
Primary completion
2023
Study completion
2023
First posted
Oct 6, 2020
Registry last updated
Apr 13, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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