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    Summary
    EudraCT Number:2020-002812-36
    Sponsor's Protocol Code Number:PTC743-NEU-003-FA
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2020-11-06
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedGermany - BfArM
    A.2EudraCT number2020-002812-36
    A.3Full title of the trial
    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)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to determine if Vatiquinone, the study drug, is safe and effective to treat a neurological condition called Friedreich ataxia
    A.3.2Name or abbreviated title of the trial where available
    MOVE-FA
    A.4.1Sponsor's protocol code numberPTC743-NEU-003-FA
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04577352
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorPTC Therapeutics, Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportPTC Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPTC Therapeutics, Inc.
    B.5.2Functional name of contact pointPatient Advocacy
    B.5.3 Address:
    B.5.3.1Street Address100 Corporate Court
    B.5.3.2Town/ citySouth Plainfield
    B.5.3.3Post codeNJ 07080
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1 866-282-5873
    B.5.6E-mailmedinfo@ptcbio.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameVatiquinone
    D.3.2Product code PTC743
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNVATIQUINONE
    D.3.9.1CAS number 1213269-98-7
    D.3.9.2Current sponsor codePTC743
    D.3.9.3Other descriptive namealpha-tocotrienolquinone
    D.3.9.4EV Substance CodeSUB188275
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Friedreich Ataxia (FA)
    E.1.1.1Medical condition in easily understood language
    Friedreich ataxia is an autosomal recessive, neurodegenerative disease that primarily affects the nervous system and heart.
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10017374
    E.1.2Term Friedreich's ataxia
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale [mFARS]) and safety of vatiquinone in subjects with FA.
    E.2.2Secondary objectives of the trial
    • Demonstrate the effects of vatiquinone on activities of daily living as assessed by the Friedreich Ataxia Rating Scale Activities of Daily Living (FARS-ADL) scale
    • Demonstrate the effects of vatiquinone on ambulation as assessed by the 1-minute walk test (1MWT)
    • Demonstrate the effects of vatiquinone on falls as assessed by a fall log
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. mFARS ≥20 to ≤70 at baseline

    2. Minimum age 7 years at the time of Screening Visit

    3. Must be able to ambulate at least 10 feet in one minute with or without assistance (non-wheelchair)

    4. Friedreich ataxia diagnosis (homozygous for guanine-adenine-adenine [GAA] repeat expansion in intron-1 of frataxin gene), confirmed by clinical genetic testing (Note: size of GAA repeat is not required for eligibility)

    5. Consent to comply with study procedures. For subjects under the age of 18 (or age of consent), parent(s)/legal guardian(s) of the subject 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 subject must give their consent for subject to enroll in the study.

    6. Difference in the mFARS between screening and baseline of no more than 4 points

    7. 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 (eg, cardiologist, neurologist, or hematologist), and discontinuation will be noted by the prescribing physician (see Appendix 1)

    8. Must be able to abstain from potent cytochrome P450 (CYP) 3A4 inducers/inhibitors (eg, ketoconazole, rifampin, St. John’s wort, grapefruit juice or any grapefruit product) for at least 30 days prior to enrollment (see Appendix 1)

    9. Must be able to swallow capsules

    10. Males and females of childbearing potential must be willing to use an effective method of contraception as defined in protocol Section 7.5.10 from the time consent is signed until 30 days after the last dose of study drug or early termination visit. Male subjects 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
    E.4Principal exclusion criteria
    1. Individuals with clinical diagnosis of FA who have point mutations or deletions or other non-GAA expansion mutations

    2. Previous treatment with vatiquinone

    3. Allergy to vatiquinone, sesame oil, gelatin (bovine and/or porcine), titanium dioxide, or red iron oxide

    4. Ejection fraction <50%

    5. Uncontrolled diabetes (HbA1c >7.0%) at the time of screening

    6. 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

    7. Pregnant or lactating subjects or those sexually active subjects 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

    8. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) ≥2×ULN at time of screening

    9. INR ≥1.5×ULN at time of screening or clinically significant (CS) bleeding, as determined by the investigator

    10. Serum creatinine ≥1.5×ULN at time of screening

    11. Comorbidities that may confound study results (eg, fat malabsorption syndrome, other mitochondrial disorder) in the opinion of the investigator

    12. 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. Subjects may be rescreened after the exclusionary period of 60 days has passed.

    13. Concomitant use of interventional CoQ10, vitamin E, or any approved or non-approved medication for FA within 30 days prior to the Screening Visit (Appendix 1). 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.

    14. Illicit drug use 30 days prior to screening and during the study is prohibited.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint will be the change from baseline in the modified Friedreich Ataxia Rating Scale (mFARS) in the mITT population at Week 72.
    E.5.1.1Timepoint(s) of evaluation of this end point
    A completer analysis will be performed for all subjects who took medication till the end of double-blind period as specified by the protocol and have mFARS score at Week 72 visit.
    E.5.2Secondary end point(s)
    Secondary endpoints will include change in baseline in the following assessments at Week 72.
    • FARS-ADL scale (key secondary endpoint)
    • 1MWT
    • Fall log
    E.5.2.1Timepoint(s) of evaluation of this end point
    Comparisons between treatment groups for change from baseline in FARS-ADL scale, one minute timed walk, MFIS score, EQ-5D-5L, and Upright Stability subscale of the mFARS to Week 72 will be analyzed using the same model as the primary efficacy variable by using its respective baseline and age as covariates. Summary statistics will be provided for total score at each visit as well as change from baseline at each visit.

    A summary of the number of falls will be presented, and the number of falls over time will be compared between the two treatment groups.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Study with Open-Label Extension
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Brazil
    Canada
    New Zealand
    United States
    France
    Germany
    Italy
    Spain
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days7
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months7
    E.8.9.2In all countries concerned by the trial days9
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 90
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 30
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 60
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 36
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 24
    F.4.2.2In the whole clinical trial 126
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Subjects who complete the study may have the option to enter an open-label expanded access program to continue receiving vatiquinone.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-01-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-02-18
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2023-10-02
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