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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04577352




Registration number
NCT04577352
Ethics application status
Date submitted
30/09/2020
Date registered
6/10/2020
Date last updated
27/03/2024

Titles & IDs
Public title
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
Scientific 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)
Secondary ID [1] 0 0
PTC743-NEU-003-FA
Universal Trial Number (UTN)
Trial acronym
MOVE-FA
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Friedreich Ataxia 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Neurodegenerative diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Vatiquinone
Treatment: Drugs - Placebo

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.

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.


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

Treatment: Drugs: Placebo
Placebo will be administered per schedule specified in the arm.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in the Modified Friedreich Ataxia Rating Scale (mFARS) Score at Week 72
Timepoint [1] 0 0
Baseline, Week 72
Secondary outcome [1] 0 0
Change From Baseline in Friedreich Ataxia Rating Scale Activities of Daily Living (FARS-ADL) Score at Week 72
Timepoint [1] 0 0
Baseline, Week 72
Secondary outcome [2] 0 0
Change From Baseline in 1-Minute Walk Test (1MWT) at Week 72
Timepoint [2] 0 0
Baseline, Week 72
Secondary outcome [3] 0 0
Number of Falls Through Week 72
Timepoint [3] 0 0
Baseline through Week 72

Eligibility
Key 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.
Minimum age
7 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
17/12/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
2/10/2023
Sample size
Target
Accrual to date
Final
146
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Murdoch Children's Research Institute - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Iowa
Country [4] 0 0
United States of America
State/province [4] 0 0
Pennsylvania
Country [5] 0 0
Brazil
State/province [5] 0 0
São Paulo
Country [6] 0 0
Canada
State/province [6] 0 0
Quebec
Country [7] 0 0
France
State/province [7] 0 0
PARIS cedex
Country [8] 0 0
Germany
State/province [8] 0 0
Tuebingen
Country [9] 0 0
Italy
State/province [9] 0 0
Roma
Country [10] 0 0
New Zealand
State/province [10] 0 0
Auckland
Country [11] 0 0
Spain
State/province [11] 0 0
Barcelona

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
PTC Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
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).
Trial website
https://clinicaltrials.gov/study/NCT04577352
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04577352