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    Summary
    EudraCT Number:2022-002668-65
    Sponsor's Protocol Code Number:PTC743-NEU-003e-FA
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-12-23
    Trial 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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2022-002668-65
    A.3Full title of the trial
    Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia
    Studio in aperto a lungo termine per valutare la sicurezza e l’efficacia di vatiquinone in pazienti con atassia di Friedreich
    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
    Uno studio per determinare se Vatiquinone, il farmaco di studio, sia sicuro ed efficace per trattare una condizione neurologica chiamata atassia di Friedreich
    A.3.2Name or abbreviated title of the trial where available
    A study to determine if Vatiquinone is safe and effective to treat Friedreich ataxia
    Uno studio per determinare se Vatiquinone sia sicuro ed efficace per trattare atassia di Friedreich
    A.4.1Sponsor's protocol code numberPTC743-NEU-003e-FA
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05515536
    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 number0018662825873
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/21/2423
    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.3.9.4EV 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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Friedreich Ataxia (FA)
    Atassia di Friedreich (AF)
    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.
    L'atassia di Friedreich è una malattia neurodegenerativa autosomica recessiva che colpisce principalmente il sistema nervoso e il cuore.
    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
    To assess the long-term safety of vatiquinone in subjects with FA previously exposed to vatiquinone in Study PTC743-NEU-003-FA or Study PTC743-NEU-005-FA
    Valutare la sicurezza a lungo termine di vatiquinone nei soggetti affetti da AF precedentemente trattati con vatiquinone nell’ambito dello studio PTC743-NEU-003-FA o dello studio PTC743-NEU-005-FA
    E.2.2Secondary objectives of the trial
    To assess the effects of vatiquinone on the Modified Friedreich Ataxia Rating Scale (mFARS) in subjects with FA previously exposed to vatiquinone in Study PTC743-NEU-003-FA or Study PTC743-NEU-005-FA
    Valutare gli effetti di vatiquinone sulla base della Scala di valutazione dell’atassia di Friedreich modificata (mFARS) nei soggetti affetti da AF precedentemente trattati con vatiquinone nell’ambito dello studio PTC743-NEU-003-FA o dello studio PTC743-NEU-005-FA
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject provides consent to participate.

    2. Subjects with FA who completed and directly rolled over from a previous vatiquinone clinical study (PTC743-NEU-003-FA [ie, MOVE-FA] or PTC743-NEU-005-FA).

    3. Males and females of childbearing potential must be willing to use an effective method of contraception (eg, implants, injectables, transdermal patch, combined oral contraceptives, barrier methods, and intrauterine devices) from the time consent is signed until 30 days after 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. Note: Double-barrier method (ie, condom with spermicide) is
    required if no other methods of contraception are in use.
    1. Il soggetto fornisce il consenso alla partecipazione.

    2. Soggetti affetti da FA che hanno completato uno studio clinico precedente su vatiquinone (PTC743-NEU-003-FA [ossia MOVE-FA] o PTC743-NEU-005-FA) e sono stati arruolati direttamente da questi.

    3. Gli uomini e le donne potenzialmente fertili devono acconsentire a usare un metodo contraccettivo efficace (ad es. impianti, iniettabili, cerotto transdermico, contraccettivi orali combinati, metodi barriera e dispositivi intrauterini) dal momento della firma del modulo di consenso informato fino a 30 giorni dopo aver assunto l’ultima dose del farmaco dello studio o essere stati sottoposti alla visita di interruzione anticipata. I soggetti di sesso maschile devono acconsentire a non donare lo sperma durante lo studio e per almeno 30 giorni dopo aver assunto l’ultima dose del farmaco dello studio o essere stati sottoposti alla visita di interruzione anticipata. Nota: se non si utilizza nessun altro metodo contraccettivo è necessario utilizzare un metodo a doppia barriera (ossia profilattico con spermicida).
    E.4Principal exclusion criteria
    1. Current participation in any other interventional study

    2. Pregnancy or breastfeeding
    1. Attuale partecipazione a un qualsiasi altro studio interventistico in corso

    2. Gravidanza o allattamento al seno
    E.5 End points
    E.5.1Primary end point(s)
    Adverse events/serious adverse events (SAEs), laboratory abnormalities, vital signs, and electrocardiograms (ECGs)
    Eventi avversi (EA)/eventi avversi gravi (SAE), valori di laboratorio anomali, segni vitali ed elettrocardiogrammi (ECGs)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Frequencies of AEs will be tabulated by MedDRA System Organ Class, Preferred Term, severity, relationship to study drug, and seriousness.
    Vital signs, laboratory data, and ECG will be summarized by visit. Changes from baseline will be summarized by visit, where appropriate.
    Le frequenze degli EA saranno tabulate in base alla classificazione per MedDRA System Organ Class, Preferred Term, severità, relazione con il farmaco oggetto dello studio e gravità.
    I segni vitali, i dati di laboratorio e l'ECG saranno riassunti per visita. Le modifiche rispetto al basale saranno riassunte per visita, se del caso.
    E.5.2Secondary end point(s)
    Change from baseline in the mFARS
    Cambiamenti rispetto al basale nella mFARS
    E.5.2.1Timepoint(s) of evaluation of this end point
    Modified Friedreich Ataxia Rating Scale scores and the changes from baseline will be summarized by visit based on the Safety Analysis Set.
    I punteggi modificati della scala di valutazione dell'Atassia di Friedreich e le modifiche rispetto al basale saranno riassunte per visita in base al set di analisi della sicurezza.
    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 No
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Studio di estens. a lungo termine in soggetti con AF che hanno completato studio PTC743-NEU-003-FA
    Long-Term extension study in subject with FA who completed prior PTC743-NEU-003-FA study
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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.8.4.1Number of sites anticipated in Member State concerned1
    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 Information not present in EudraCT
    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
    Spain
    Germany
    Italy
    E.8.7Trial has a data monitoring committee No
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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: 25
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 65
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 45
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 35
    F.4.2.2In the whole clinical trial 140
    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)
    After withdrawal from the study or completion of the study, the patient should be treated according to normal clinical practice
    Dopo il ritiro dallo studio o il completamento dello studio, il paziente deve essere trattato secondo la normale pratica clinica
    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 Decision2023-02-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-04-26
    P. End of Trial
    P.End of Trial StatusOngoing
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