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)

Plain English Summary

A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia is a Phase 3 clinical trial sponsored by PTC Therapeutics studying Friedreich Ataxia. Tests the effectiveness and safety of Vatiquinone for Friedreich Ataxia. For adults diagnosed with Friedreich Ataxia, aged 18+. Participation involves taking Vatiquinone or placebo for 72 weeks, with regular check-ups. Alternative treatments include physical therapy and supportive care. The trial aims to enroll 146 participants.

Official 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).

Who Can Participate

Here is what you need to know about eligibility for this trial. Eligible if aged 18+, diagnosed with Friedreich Ataxia, can walk at least 10 feet in a minute, and meet other health criteria. Not eligible if allergic to Vatiquinone or have certain health conditions like uncontrolled diabetes or bleeding disorders. This trial is studying Friedreich Ataxia, so participants generally need a confirmed diagnosis.

What They're Measuring

The primary outcome measures the improvement in daily living activities and walking ability over 72 weeks. The specific primary outcome measures are: Change From Baseline in the mFARS Score at Week 72 - Modified Intent-to-treat (mITT) Analysis Set (Baseline, Week 72); Change From Baseline in the mFARS Score at Week 72 - Intent-to-treat (ITT) Analysis Set (Baseline, Week 72). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This trial is in Phase 3, the final and most rigorous stage before seeking FDA approval. Phase 3 trials involve 300-3,000+ patients across multiple sites and compare the new treatment directly against the current standard of care. These pivotal trials generate the evidence needed for regulatory review. About 58% of Phase 3 drugs receive FDA approval. Successful Phase 3 results typically lead to a New Drug Application submission.

Why This Trial Matters

This trial addresses a critical gap in treatment options for Friedreich Ataxia, a rare genetic disorder affecting movement. As a Phase 3 trial, positive results could directly lead to FDA approval, making this treatment available to the broader patient population. This research targets Friedreich Ataxia, where improved treatment options are needed.

Investor Insight

The large international scope and high enrollment suggest strong market interest and potential for approval. Phase 3 trials have approximately a 50-60% chance of gaining FDA approval if they reach this stage.

Is This Trial Right for Me?

Ask your doctor about your eligibility and the potential benefits and risks. Participation involves regular check-ups and taking Vatiquinone or placebo daily. The trial is being conducted at 14 sites. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

• Study Type: INTERVENTIONAL
• Allocation: RANDOMIZED
• Model: PARALLEL
• Masking: QUADRUPLE
• Enrollment: 146 participants

Interventions

• 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 Outcomes

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

Secondary Outcomes

• Change From Baseline in Friedreich Ataxia Rating Scale Activities of Daily Living (FARS-ADL) Score at Week 72 - mITT Analysis Set (Baseline, Week 72)
• Change From Baseline in FARS-ADL Score at Week 72 - ITT Analysis Set (Baseline, Week 72)
• Change From Baseline in 1-Minute Walk Test (1MWT) at Week 72 - mITT Analysis Set (Baseline, Week 72)
• Change From Baseline in 1MWT at Week 72 - ITT Analysis Set (Baseline, Week 72)
• Number of Falls Per 28 Days Over Every 24-Week Period - mITT Analysis Set (Week 1-24, Week 25-48, and Week 49-72)

Full Eligibility Criteria

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.

Trial Locations

• UCLA, Los Angeles, California, United States
• University of Florida, Gainesville, Florida, United States
• University of South Florida, Tampa, Florida, United States
• University of Iowa, Iowa City, Iowa, United States
• The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
• Murdoch Children's Research Institute, Parkville, Victoria, Australia
• University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology, São Paulo, Brazil
• Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM), Montreal, Quebec, Canada
• CHU Sainte-Justine, Montreal, Quebec, Canada
• Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute), Paris, France
• ...and 4 more locations

Frequently Asked Questions

Related Conditions

• Friedreich Ataxia, Mitochondrial Disorders, Neurological Disorders, Genetic Disorders, Rare Diseases

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