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    Summary
    EudraCT Number:2017-002541-29
    Sponsor's Protocol Code Number:PVO-1A-301
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2017-09-28
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2017-002541-29
    A.3Full title of the trial
    A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP)
    Estudio de fase 3, de la eficacia y la seguridad de palovaroteno oral para el tratamiento de la fibrodisplasia osificante progresiva (FOP)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical trial to evaluate the Efficacy and Safety of Oral Palovarotene as a treatment for Fibrodysplasia Ossificans Progressiva (FOP)
    Estudio clinica para evaluar la eficacia y la seguridad de palovaroteno oral para el tratamiento de la fibrodisplasia osificante progresiva (FOP)
    A.3.2Name or abbreviated title of the trial where available
    MOVE Trial
    A.4.1Sponsor's protocol code numberPVO-1A-301
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/127/2015
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorClementia Pharmaceuticals Inc
    B.1.3.4CountryCanada
    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 supportClementia Pharmaceuticals Inc
    B.4.2CountryCanada
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationClementia Pharmaceuticals Inc.
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street Address4150 Ste-Catherine Street West, Suite 550
    B.5.3.2Town/ cityMontreal, Quebec
    B.5.3.3Post codeH3Z 2Y5
    B.5.3.4CountryCanada
    B.5.4Telephone number900834223
    B.5.6E-mailRegistroEspanolDeEstudiosClinicos@druginfo.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/3/14/1368
    D.3 Description of the IMP
    D.3.1Product namePALOVAROTENE
    D.3.4Pharmaceutical form Capsule, hard
    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 INNPALOVAROTENE
    D.3.9.1CAS number 410528-02-8
    D.3.9.3Other descriptive namePALOVAROTENE
    D.3.9.4EV Substance CodeSUB75998
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number1 to 10
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Fibrodysplasia Ossificans Progressiva (FOP)
    Fibrodisplasia Osificante progresiva (FOP)
    E.1.1.1Medical condition in easily understood language
    FOP is a rare, severely disabling disease characterized by heterotopic ossification in muscles, tendons, and ligaments often associated with painful and recurrent episodes of soft tissue swelling.
    FOP:enfermedad rara,severamente incapacitante por la osificación heterotópica en músculos,tendones y ligamentos amenudo asociados con episodios dolorosos y recurrentes de hinchazón de tejidos blandos.
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10068715
    E.1.2Term Fibrodysplasia ossificans progressiva
    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 evaluate the efficacy of palovarotene in decreasing heterotopic ossification (HO) in adult and pediatric subjects with FOP as assessed by
    low-dose, whole body computed tomography (WBCT), excluding head, as compared to untreated subjects from Clementia’s FOP natural history study.
    • To evaluate the safety of palovarotene in adult and pediatric subjects with FOP.
    •Evaluar la eficacia del palovaroteno en la reducción de la osificación heterotópica (OH) en pacientes adultos y pediátricos con FOP, evaluada mediante tomografía computarizada de cuerpo entero (TCCE) de dosis bajas, excepto la cabeza, en comparación con sujetos no tratados del estudio de la evolución natural de la FOP de Clementia.
    •Evaluar la seguridad del palovaroteno en pacientes adultos y pediátricos con FOP.
    E.2.2Secondary objectives of the trial
    • To evaluate the effect of palovarotene on flare-up rate and proportion of subjects reporting at least one flare-up.
    • To evaluate the effect of palovarotene on range of motion (ROM) as assessed by the Cumulative Analogue Joint Involvement Scale for FOP (CAJIS).
    • To evaluate the effect of palovarotene on physical function using age-appropriate forms of the FOP-Physical Function Questionnaire (FOP-PFQ).
    • To evaluate the effect of palovarotene on physical and mental health using age-appropriate forms of the Patient Reported Outcomes Measurement
    Information System (PROMIS) Global Health Scale.
    • To evaluate the pharmacokinetics of palovarotene.
    •Evaluar el efecto del palovaroteno sobre la tasa de exacerbaciones y la proporción de sujetos que notifican al menos una exacerbación.
    •Evaluar el efecto del palovaroteno sobre la amplitud de movimiento (ADM), evaluada mediante la Escala analógica de afectación articular acumulada (CAJIS) para la FOP.
    •Evaluar el efecto del palovaroteno sobre la función física utilizando los formularios adecuados para la edad del Cuestionario de función física en la FOP (FOP PFQ).
    •Evaluar el efecto del palovaroteno sobre la salud física y mental utilizando los formularios adecuados para la edad de la Escala de salud global del Sistema de información de resultados comunicados por el paciente (PROMIS).
    •Evaluar la farmacocinética del palovaroteno.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects must meet all of the following criteria to be eligible for enrollment:
    1. Written, signed, and dated informed subject/parent consent; and for subjects who are minors, age-appropriate assent (performed according to local
    regulations).
    2. Male or female at least 4 years of age.
    3. Clinically diagnosed with FOP, with the R206H ACVR1 mutation.
    4. No flare-up symptoms within the past 4 weeks, including at the time of enrollment.
    5. Females of child-bearing potential must have a negative blood or urine pregnancy test (with sensitivity of at least 50 mIU/mL) prior to administration of palovarotene. Male and FOCBP subjects must agree to remain abstinent during treatment and for 1 month after treatment or, if sexually active, to use two highly effective methods of birth control during and for 1 month after treatment. Additionally, sexually active FOCBP subjects must already be using two highly effective methods of birth control 1 month before treatment is to start. Specific risk of the use of retinoids during pregnancy, and the agreement to remain abstinent or use two highly effective methods of birth control will be clearly defined in the informed consent and the subject or legally authorized representatives (eg, parents, caregivers, or legal guardians) must specifically sign this section.
    6. Must be accessible for treatment and follow-up, and be able to undergo all study procedures. Subjects living at distant locations from the
    investigational site must be able and willing to travel to a site for the initial and all on-site follow-up visits. Subjects must be able to undergo low-dose,
    WBCT (excluding head).
    Los sujetos deben cumplir todos los criterios siguientes para poder ser incluidos en este estudio:
    1.Consentimiento informado del sujeto/progenitor escrito, firmado y fechado; en los sujetos menores de edad, asentimiento apropiado para la edad (conforme a la normativa local).
    2.Sujetos de ambos sexos mayores de 4 años.
    3.Diagnóstico clínico de FOP con la mutación R206H ACVR1.
    4.Ausencia de síntomas de exacerbación en las 4 últimas semanas, hasta el momento del reclutamiento inclusive.
    5.Las mujeres en edad fértil deberán dar negativo en una prueba de embarazo en sangre u orina (con una sensibilidad mínima de 50 mUI/ml) antes de la administración del palovaroteno. Los varones y las MEF deberán comprometerse a mantener la abstinencia durante el tratamiento y durante un mes después del tratamiento o, si son sexualmente activos, a utilizar dos métodos anticonceptivos muy eficaces durante el tratamiento y durante un mes después del mismo. Además, las MEF sexualmente activas deberán estar utilizando dos métodos anticonceptivos muy eficaces desde un mes antes de empezar el tratamiento. El riesgo específico del uso de retinoides durante el embarazo y el compromiso de mantener la abstinencia o utilizar dos métodos anticonceptivos muy eficaces se definirán claramente en el consentimiento informado y el sujeto o sus representantes legales (p. ej., padres, cuidadores o tutores legales) deberán firmar específicamente esta sección.
    6.Deben estar accesibles para recibir tratamiento y seguimiento y ser capaces de someterse a todos los procedimientos del estudio. Los sujetos que vivan en lugares alejados del centro de investigación deberán ser capaces y estar dispuestos a desplazarse a un centro para la visita inicial y para todas las visitas de seguimiento en el centro. Los sujetos deben ser capaces de someterse a una TCCE de dosis bajas (excepto la cabeza).
    E.4Principal exclusion criteria
    Subjects meeting any of the following criteria are not eligible for enrollment:
    1. Weight <10 kg.
    2. If currently using vitamin A or beta carotene, multivitamins containing vitamin A or beta carotene, or herbal preparations, fish oil, and unable or
    unwilling to discontinue use of these products during palovarotene treatment.
    3. Exposure to synthetic oral retinoids other than palovarotene within 4 weeks prior to screening.
    4. Concurrent treatment with tetracycline or any tetracycline derivatives due to the potential increased risk of pseudotumor cerebri.
    5. History of allergy or hypersensitivity to retinoids or lactose (note that lactose intolerance is not exclusionary).
    6. Concomitant medications that are strong inhibitors or inducers of cytochrome P450 (CYP450) 3A4 activity; or kinase inhibitors such as
    imatinib (see Section 5.2.1).
    7. Amylase or lipase >2x above the upper limit of normal (ULN) or with a history of chronic pancreatitis.
    8. Elevated aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >2.5x ULN.
    9. Fasting triglycerides >400 mg/dL with or without therapy.
    10. Female subjects who are breastfeeding.
    11. Subjects with uncontrolled cardiovascular, hepatic, pulmonary, gastrointestinal, endocrine, metabolic, ophthalmologic, immunologic,
    psychiatric, or other significant disease.
    12. Subjects experiencing suicidal ideation (type 4 or 5) or any suicidal behavior within the past month as defined by the Columbia-Suicide Severity
    Rating Scale (C-SSRS).
    13. Simultaneous participation in another clinical research study within 4 weeks prior to Screening; or within five half-lives of the investigational agent,
    whichever is longer.
    14. Any reason that, in the opinion of the Investigator, would lead to the inability of the subject and/or family to comply with the protocol.
    No podrán ser incluidos los sujetos que cumplan alguno de los criterios siguientes:
    1.Peso <10 kg.
    2.En caso estar utilizando vitamina A o betacaroteno, polivitamínicos que contengan vitamina A o betacaroteno, preparados de herbolario o aceite de pescado, el sujeto no puede o no está dispuesto a suspender el uso de estos productos durante el tratamiento con palovaroteno.
    3.Exposición a retinoides orales sintéticos distintos del palovaroteno en las 4 semanas previas a la selección.
    4.Tratamiento concomitante con tetraciclinas o cualquier derivado de las tetraciclinas debido al posible aumento del riesgo de seudotumor cerebral.
    5.Antecedentes de alergia o hipersensibilidad a los retinoides o la lactosa (obsérvese que la intolerancia a la lactosa no es motivo de exclusión).
    6.Medicamentos concomitantes que sean inhibidores o inductores potentes de la actividad del citocromo P450 (CYP450) 3A4, o inhibidores de quinasas como imatinib (véase la sección 5.2.1).
    7.Valor de amilasa o lipasa > 2 veces por encima del límite superior de la normalidad (LSN) o antecedentes de pancreatitis crónica.
    8.Elevación de la aspartato aminotransferasa (AST) o la alanina aminotransferasa (ALT) > 2,5 veces el LSN.
    9.Triglicéridos en ayunas > 400 mg/dl con o sin tratamiento.
    10.Mujeres lactantes.
    11.Sujetos con enfermedades cardiovasculares, hepáticas, pulmonares, digestivas, endocrinas, metabólicas, oftalmológicas, inmunológicas, psiquiátricas o de otra naturaleza significativas y no controladas.
    12.Sujetos que hayan experimentado ideas suicidas (tipo 4 o 5) o cualquier comportamiento suicida en el último mes, según la definición de la Escala de valoración del riesgo de suicidio de Columbia (C-SSRS).
    13.Participación simultánea en otro estudio de investigación clínica en las 4 semanas previas a la selección o en el plazo de cinco semividas del fármaco en investigación, lo que suponga más tiempo.
    14.Cualquier motivo que, en opinión del investigador, pueda impedir que el sujeto o su familia cumplan el protocolo.
    E.5 End points
    E.5.1Primary end point(s)
    The annualized change in new HO volume as assessed by low-dose, WBCT (excluding head) compared to untreated subjects from the NHS.
    Variación anualizada del volumen de nueva OH, evaluada mediante TCCE de dosis bajas (excepto la cabeza), en comparación con los sujetos no tratados del EEN.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Month 6, 12, 18 and 24 months. It will also being evaluated at End of trial or End of study visit.
    Meses 6,12,18 y 24.También se evaluará al final del ensayo o al final de la visita de estudio.
    E.5.2Secondary end point(s)
    1. The proportion of subjects with any new HO.
    2. The change from baseline in the number of body regions with new HO.
    3. The proportion of subjects reporting flare-ups.
    4. The flare-up rate per subject-month exposure.
    1.Proporción de sujetos con nueva OH.
    2.Variación con respecto al valor basal del número de regiones corporales con nueva OH.
    3.Proporción de sujetos que notifiquen exacerbaciones.
    4.Tasa de exacerbaciones por exposición mensual-sujeto.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Month 6, 12, 18 and 24 months. It will also being evaluated at End of trial or End of study visit.
    Meses 6,12,18 y 24.También se evaluará al final del ensayo o al final de la visita de estudio.
    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 No
    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 Yes
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    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
    El ensayo tiene control externo (estudio del EEN - sujetos no tratados)
    The trial has en external control (NHS study - untreated subjects)
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    The trial has en external control (NHS study - untreated subjects)
    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 Yes
    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 EEA8
    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
    Argentina
    Australia
    Brazil
    Canada
    France
    Germany
    Italy
    Japan
    Russian Federation
    South Africa
    Spain
    Sweden
    United Kingdom
    United States
    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
    Last Visit Last Patient
    UVUP
    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 days15
    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 days15
    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: 20
    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: 10
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 60
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Subjects of study are male of female at least 4 years old
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 27
    F.4.2.2In the whole clinical trial 80
    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)
    If the risk/benefit profile based on the emerging Phase 3 data warrants it, subjects will be offered to continue treatment until commercial availability of the product in a subject’s country of residence.
    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 Decision2017-11-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-10-31
    P. End of Trial
    P.End of Trial StatusOngoing
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