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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT04577352
Ethics application status
Date submitted
30/09/2020
Date registered
6/10/2020
Date last updated
29/04/2021

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 - 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.
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 - 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.
Timepoint [1] 0 0
Baseline, Week 72
Secondary outcome [2] 0 0
Change From Baseline in 1-Minute Walk Test (1MWT) at 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.
Timepoint [2] 0 0
Baseline, Week 72
Secondary outcome [3] 0 0
Number of Falls 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.
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

- 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. If the
participant is taking prohibited medications at screening, this difference is to be
assessed between the post-discontinuation mFARS and the baseline mFARS.

- 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

- Able to abstain from potent cytochrome P450 (CYP) 3A4 inducers/inhibitors (for
example, ketoconazole, rifampin, St. John's wort, grapefruit juice) for at least 4
weeks prior to enrollment

- Ability 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 treatment
discontinuation.
Minimum age
7 Years
Maximum age
No limit
Gender
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 or 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 or suicidal behavior based on Columbia-Suicide Severity
Rating Scale (C-SSRS) at screening or baseline

- 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

- 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 coenzyme Q10 (CoQ10), vitamin E, idebenone, or any other
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.

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 analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2/Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
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
Germany
State/province [7] 0 0
Tuebingen
Country [8] 0 0
Italy
State/province [8] 0 0
Roma
Country [9] 0 0
Spain
State/province [9] 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/show/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
Patient Advocacy
Address 0 0
Country 0 0
Phone 0 0
866-282-5873
Fax 0 0
Email 0 0
medinfo@ptcbio.com
Contact person for scientific queries

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