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    Summary
    EudraCT Number:2016-005035-33
    Sponsor's Protocol Code Number:R2477-FOP-1623
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Temporarily Halted
    Date on which this record was first entered in the EudraCT database:2019-01-22
    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 number2016-005035-33
    A.3Full title of the trial
    A randomized, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, and effects on heterotopic bone formation of REGN2477 in patients with Fibrodysplasia Ossificans Progressiva
    Estudio aleatorizado y controlado con placebo para evaluar la seguridad, la tolerabilidad, la farmacocinética y los efectos en la osificación heterotópica de REGN2477 en pacientes con fibrodisplasia osificante progresiva
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to examine the safety, tolerability and effects on abnormal bone formation of REGN2477 in patients with Fibrodysplasia Ossificans Progressiva
    Estudio para examinar la seguridad, la tolerabilidad y los efectos en la osificación anormal de REGN2477 en pacientes con fibrodisplasia osificante progresiva
    A.3.2Name or abbreviated title of the trial where available
    LUMINA-1
    A.4.1Sponsor's protocol code numberR2477-FOP-1623
    A.5.4Other Identifiers
    Name:IND NumberNumber:130595
    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 SponsorRegeneron Pharmaceuticals, 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 supportRegeneron Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRegeneron Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointClinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address777 Old Saw Mill River Road
    B.5.3.2Town/ cityTarrytown, NY
    B.5.3.3Post code10591
    B.5.3.4CountryUnited States
    B.5.6E-mailclinicaltrials@regeneron.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/3/16/1779
    D.3 Description of the IMP
    D.3.1Product nameREGN2477
    D.3.2Product code REGN2477
    D.3.4Pharmaceutical form Powder for solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNN/A
    D.3.9.1CAS number N/A
    D.3.9.2Current sponsor codeREGN2477
    D.3.9.3Other descriptive nameREGN2477
    D.3.9.4EV Substance CodeSUB182187
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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 placeboPowder for solution for injection/infusion
    D.8.4Route of administration of the placeboIntravenous use
    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
    Genetic condition which causes abnormal formation of bone at abnormal locations such as in the muscles, tendons and ligaments.
    Alteración genética que provoca una osificación anormal en ubicaciones inusuales, como los músculos, tendones y ligamentos.
    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
    The primary safety objective of the study is to assess the safety and tolerability of REGN2477 in male and female patients with fibrodysplasia ossificans progressiva (FOP).
    The primary efficacy objective of the study is to assess the effect of REGN2477 versus placebo on the change from baseline in heterotopic ossification (HO) in patients with FOP, as determined by 18-NaF uptake in HO lesions by positron emission tomography (PET) and in total volume of HO lesions by computed tomography (CT).
    En lo que respecta a la seguridad, el objetivo principal del estudio es evaluar la seguridad y tolerabilidad de REGN2477 en pacientes de sexo masculino y femenino con fibrodisplasia osificante progresiva (FOP).
    En lo referente a la eficacia, el objetivo principal del estudio es evaluar el efecto de REGN2477, en comparación con el del placebo, en el cambio con respecto al momento basal en la osificación heterotópica (OH) en pacientes con FOP, determinada por la captación de 18F-NaF en las lesiones de OH mediante tomografía por emisión de positrones (PET), así como en el volumen total de las lesiones de OH determinado mediante tomografía computarizada (TC).
    E.2.2Secondary objectives of the trial
    - To compare the effect of REGN2477 versus placebo on pain due to FOP, as measured by the area under the curve for pain based on daily numeric rating scale scores
    - To assess the effect of REGN2477 versus placebo on the change from baseline in:
    - HO, as determined by the number of new HO lesions identified by 18F-NaF PET or by CT
    - 18F-NaF standardized uptake value maximum of individual active HO site(s) by PET
    - biochemical markers of bone formation
    - To assess the effect of REGN2477 between wk28 and wk56, on the number, activity, and volume of HO lesions identified by 18F-NaF PET or by CT in patients who switch from placebo to REGN2477 at wk 28 versus the same patients between baseline and wk28
    - To characterize the concentrations of total activin A at baseline and over time following the first dose of study drug
    - To characterize the concentration-time profile (pharmacokinetics) of REGN2477 in patients with FOP
    - To assess the immunogenicity of REGN2477
    -Comparar efecto REGN2477 vs placebo en dolor provocado por FOP, según área bajo la curva para dolor según puntuaciones diarias en escala calificación numérica (NRS)
    -Evaluar efecto REGN2477 vs placebo en cambio respecto al momento basal en:
    - OH, determinada por núm. de nuevas lesiones de OH identificadas mediante PET con 18F-NaF o mediante TC
    - valor captación normalizado máx (VCNmáx) de 18F-NaF de focos OH activos (mediante PET)
    - marcadores bioquímicos osificación
    -Evaluar efecto REGN2477 entre sem 28 -56 en el núm, actividad y volumen de lesiones de OH identificadas mediante PET con 18F-NaF o mediante TC en pacs que cambien de placebo a REGN2477 en sem. 28, en comparación con los de los mismos pacientes entre el momento basal y sem. 28
    -Caracterizar concentraciones activina A total en momento basal y a lo largo del tiempo tras 1a. dosis de medicamento estudio
    -Caracterizar perfil concentración-tiempo (FC) REGN2477 en pacs con FOP
    -Evaluar inmunogenia REGN2477
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Men and women 18 to 60 years of age at screening.
    2. Clinical diagnosis of FOP (based on findings of congenital malformation of the great toes, episodic soft tissue swelling, and/or progressive heterotopic ossification).
    3. Confirmation of FOP diagnosis with documentation of any ACVR1 mutation.
    4. FOP disease activity within 1 year of screening visit. FOP disease activity is defined as pain, swelling, stiffness, and other signs and symptoms associated with FOP flare-ups; or worsening of joint function, or radiographic progression of heterotopic ossifications (increase in site or number of HO lesions) with/without being associated with flare-up episodes.
    5. Willing and able to undergo PET and CT imaging procedures and other procedures as defined in this study.
    1. Hombres y mujeres de entre 18 y 60 años de edad en el momento de la selección.
    2. Diagnóstico clínico de FOP (basado en los hallazgos de malformación congénita en los dedos gordos de los pies, inflamación episódica de los tejidos blandos y/u osificación heterotópica progresiva).
    3. Confirmación del diagnóstico de FOP con documentación de cualquier mutación en ACVR1.
    4. FOP activa durante el año anterior a la visita de selección. La FOP activa se define como dolor, inflamación, rigidez y otros signos y síntomas asociados a reagudizaciones de la FOP, o empeoramiento de la función articular o progresión radiográfica de las osificaciones heterotópicas (aumento del foco o del número de lesiones de OH) con o sin asociación a episodios de reagudización.
    5. Capacidad y disposición para someterse a una PET y TC y a otros procedimientos definidos en este estudio.
    E.4Principal exclusion criteria
    1. Significant concomitant illness or history of significant illness such as, but not limited to cardiac, renal, rheumatologic, neurologic, psychiatric, endocrine, metabolic or lymphatic disease, that in the opinion of the study investigator might confound the results of the study or pose additional risk to the patient by their participation in the study.
    2. Use of bisphosphonate within 1 year of screening.
    3. Concurrent participation in another interventional clinical study, or a non-interventional study with radiographic measures or invasive procedures (e.g. collection of blood or tissue samples). Participation in the FOP Connection Registry or other studies in which patients complete study questionnaires are allowed.
    4. Pregnant or breastfeeding women.
    5. Male and women of childbearing potential patients who are unwilling to practice highly effective contraception.
    6. Previous history or diagnosis of cancer.
    1. Enfermedad concomitante importante o antecedentes de enfermedad importante como, por ejemplo, enfermedad cardiaca, renal, reumatológica, neurológica, psiquiátrica, endocrina, metabólica o linfática que, en opinión del investigador del estudio, podría provocar confusiones en los resultados del estudio o aumentar el riesgo que conlleva para el paciente la participación en el estudio.
    2. Uso de bisfosfonatos en el año anterior a la selección.
    3. Participación simultánea en otro estudio clínico intervencionista o en un estudio no intervencionista en el que se realicen evaluaciones radiográficas o procedimientos invasivos (por ejemplo, extracción de sangre o de muestras de tejido). Se permite la participación en el Registro Conexión FOP o en otros estudios en los que los pacientes cumplimenten cuestionarios.
    4. Mujeres embarazadas o en periodo de lactancia.
    5. Pacientes de sexo masculino y femenino con posibilidad de procrear que no estén dispuestos a utilizar métodos anticonceptivos de gran eficacia.
    6. Antecedentes o diagnóstico de cáncer.
    E.5 End points
    E.5.1Primary end point(s)
    Primary safety end point:
    Incidence and severity of treatment-emergent adverse events (TEAEs) through the end of the Period 1 at week 28

    Primary efficacy endpoints:
    - Time-weighted average (standardized area under the curve [AUC]) of the percent change from baseline in total lesion activity by 18F-NaF PETover 28 weeks (AHOC - Baseline active HO classic ACVR1[R206H] mutation analysis set)
    - Percent change from baseline in the total volume of HO lesions as assessed by CT at week 28 (AHOC)
    - Time-weighted average (standardized AUC) of the percent change from baseline in total lesion activity by 18F-NaF PET over 28 weeks (AHO - Baseline active HO analysis set)
    - Percent change from baseline in the total volume of HO lesions as assessed by CT at week 28 (AHO)
    Criterio principal de valoración de la seguridad:

    Incidencia y gravedad de los acontecimientos adversos surgidos durante el tratamiento (AAST) hasta el final del periodo 1 en la semana 28

    Criterios principales de valoración de la eficacia:

    - Cambio porcentual promedio ponderado en el tiempo (área bajo la curva [ABC] estandarizada) con respecto al momento basal en la actividad total de las lesiones determinada mediante PET con 18F-NaF a lo largo de 28 semanas (OHAC: grupo de análisis con mutación en ACVR1[R206H] clásica y OH activa en el momento basal)
    - Cambio porcentual con respecto al momento basal en el volumen total de lesiones de OH evaluado mediante TC en la semana 28 (OHAC)
    - Cambio porcentual promedio ponderado en el tiempo (ABC estandarizada) con respecto al momento basal en la actividad total de las lesiones determinada mediante PET con 18F-NaF a lo largo de 28 semanas (OHA: grupo de análisis con OH activa en el momento basal)
    - Cambio porcentual con respecto al momento basal en el volumen total de lesiones de OH evaluado mediante TC en la semana 28 (OHA)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline and end of Period 1 at week 28
    Momento basal y final del periodo 1 en la semana 28
    E.5.2Secondary end point(s)
    The key efficacy secondary endpoints:

    - Time-weighted average (standardized AUC) of the change from baseline in daily pain due to FOP, as measured using the daily NRS over 28 weeks (AHOC)
    - Time-weighted average (standardized AUC) of the change from baseline in daily pain due to FOP, as measured using the daily NRS over 28 weeks (AHO)

    Other secondary endpoints are:
    - Percent change from baseline in 18F-NaF SUVmax of individual active HO site(s) by PET at week 8 (AHOC)
    - Percent change from baseline in 18F-NaF SUVmax of individual active HO site(s) by PET at week 8 (AHO)
    - Change from baseline in number of HO lesions as assessed by 18F-NaF PET at week 28 (AHOC)
    - Change from baseline in number of HO lesions as assessed by 18F-NaF PET at week 28 (AHO)
    - Change from baseline in number of HO lesions as assessed by 18F-NaF PET at week 28 (FAS - Full Analysis Set)
    - Change from baseline in number of HO lesions detectable by CT at week 28 (AHOC)
    - Change from baseline in number of HO lesions detectable by CT at week 28 (AHO)
    - Change from baseline in number of HO lesions detectable by CT at week 28 (FAS)
    - Percent change from week 28 in total lesion activity by 18F-NaF PET to week 56 (in patients switching from placebo in double-blind period and who have active HO lesions at week 28) versus the same patients between baseline and week 28 (AHOC)
    - Percent change from week 28 in total lesion activity by 18F-NaF PET to week 56 (in patients switching from placebo in double-blind period and who have active HO lesions at week 28) versus the same patients between baseline and week 28 (AHO)
    - Percent change from week 28 in the total volume of HO lesions as assessed by CT to week 56 (in patients switching from placebo in double-blind period and who have active HO lesions at week 28) versus the same patients between baseline and week 28 (AHOC)
    - Percent change from week 28 in the total volume of HO lesions as assessed by CT to week 56 (in patients switching from placebo in double-blind period and who have active HO lesions at week 28) versus the same patients between baseline and week 28 (AHO)
    - Change from week 28 in the total volume of HO lesions as assessed by CT to week 56 (in patients switching from placebo in double-blind period and who have active HO lesions at week 28) versus the same patients between baseline and week 28 (FAS)
    - Change from week 28 in number of HO lesions by 18F-NaF PET to week 56 (in patients switching from placebo in double-blind period and who have active HO lesions at week 28) versus the same patients between baseline and week 28 (AHOC)
    - Change from week 28 in number of HO lesions by 18F-NaF PET to week 56 (in patients switching from placebo in double-blind period and who have active HO lesions at week 28) versus the same patients between baseline and week 28 (AHO)
    - Change from week 28 in number of HO lesions by CT to week 56 (in patients switching from placebo in double-blind period and who have active HO lesions at week 28) versus the same patients between baseline and week 28 (AHOC)
    - Change from week 28 in number of HO lesions by CT to week 56 (in patients switching from placebo in double-blind period and who have active HO lesions at week 28) versus the same patients between baseline and week 28 (AHO)
    - Change from week 28 in number of HO lesions by CT to week 56 (in patients switching from placebo in double-blind period and who have active HO lesions at week 28) versus the same patients between baseline and week 28 (FAS)
    - Percent change from baseline in total lesion activity by 18F-NaF PET to week 56 (AHOC)
    - Percent change from baseline in total lesion activity by 18F-NaF PET to week 56 (AHO)
    - Percent change from baseline in the total volume of HO lesions as assessed by CT to week 56 (AHOC)
    - Percent change from baseline in the total volume of HO lesions as assessed by CT to week 56 (AHO)
    - Change from baseline in the total volume of HO lesions as assessed by CT to week 56 (FAS)
    - Time-weighted average (standardized AUC) of the change from baseline in daily pain due to FOP, as measured using the daily NRS over 28 weeks (FAS)
    - Incidence and severity of TEAEs
    - Time weighted average (standardized AUC) of the percent change from baseline in biomarkers of bone formation levels in serum over 28 weeks, including Total Procollagen Type 1 N-Terminal Propeptide (P1NP), bone specific alkaline phosphatase (BSAP), and total alkaline phosphatase (tAP) (FAS)
    - Concentration of total activin A in serum over time
    - PK profile of REGN2477, assessed as concentrations of REGN2477 in serum over time
    - Immunogenicity of REGN2477, as determined by the incidence, titer, and clinical impact of treatment-emergent ADA to REGN2477 over time
    Criterios 2os clave de valoración eficacia:
    -Cambio promedio ponderado en tiempo (ABC estandarizada) respecto momento basal en dolor diario provocado por FOP, evaluado por NRS diaria durante 28 sem -OHAC
    -Cambio promedio ponderado en tiempo (ABC estandarizada) respecto momento basal en dolor diario provocado por FOP, evaluado por NRS diaria durante 28 sem -OHA
    Otros criterios 2os valoración son:
    -Cambio % respecto momento basal en VCEmáx de 18F-NaF de cada foco de OH activo según PET en sem 8 -OHAC
    -Cambio % respecto momento basal en VCEmáx de 18F-NaF de cada foco de OH activo según PET en sem 8 -OHA
    -Cambio respecto momento basal en núm de lesiones OH evaluadas por PET con 18F-NaF en sem 28 -OHAC
    -Cambio respecto momento basal en núm lesiones OH evaluadas por PET con 18F-NaF en sem 28 -OHA
    -Cambio respecto momento basal en núm lesiones OH evaluadas por PET con 18F-NaF en sem 28 -GCA: grupo completo análisis
    -Cambio respecto momento basal en núm lesiones OH detectables por TC en sem 28 -OHAC
    -Cambio respecto momento basal en núm lesiones OH detectables por TC en sem 28 -OHA
    -Cambio respecto momento basal en núm lesiones OH detectables por TC en sem 28 -GCA
    -Cambio % en actividad total de lesiones, según PET con 18F-NaF, desde sem 28 hasta 56 (en pacs que cambien desde placebo en período doble enmascaramiento y presenten lesiones OH activas en sem 28) frente a mismos pacs entre momento basal y sem 28 -OHAC
    -Cambio % en actividad total de lesiones, según PET con 18F-NaF, desde sem 28 hasta 56 (en pacs que cambien desde placebo en período doble enmascaramiento y presenten lesiones OH activas en sem 28) frente a mismos pacs entre momento basal y sem 28 -OHA
    -Cambio % en volumen total de lesiones OH, evaluado por TC, desde sem 28 hasta 56 (en pacs que cambien desde placebo en período doble enmascaramiento y presenten lesiones OH activas en sem 28) frente a mismos pacs entre momento basal y sem 28 -OHAC
    -Cambio % en volumen total de lesiones OH, evaluado por TC, desde sem 28 hasta 56 (en pacs que cambien desde placebo en período doble enmascaramiento y presenten lesiones OH activas en sem 28) frente a mismos pacs entre momento basal y sem 28 -OHA
    -Cambio en volumen total de lesiones OH, evaluado por TC, desde sem 28 hasta 56 (en pacs que cambien desde placebo en período doble enmascaramiento y presenten lesiones OH activas en sem 28) frente a mismos pacis entre momento basal y sem 28 -GCA
    -Cambio en núm de lesiones OH, según PET con 18F-NaF, desde sem 28 hasta 56 (en pacs que cambien desde placebo en período doble enmascaramiento y presenten lesiones OH activas en sem 28) frente a mismos pacs entre momento basal y sem 28 -OHAC
    -Cambio en núm de lesiones OH, según PET con 18F-NaF, desde sem 28 hasta 56 (en pacs que cambien desde placebo en período doble enmascaramiento y presenten lesiones OH activas en la sem 28) frente a mismos pacs entre momento basal y sem 28 -OHA
    -Cambio en núm de lesiones OH, según TC, desde sem 28 hasta 56 (en pacs que cambien desde placebo en período doble enmascaramiento y presenten lesiones OH activas en sem 28) frente a mismos pacs entre momento basal y sem 28 -OHAC
    -Cambio en núm de lesiones OH, según TC, desde la sem 28 hasta la 56 (en pacs que cambien desde placebo en período doble enmascaramiento y presenten lesiones OH activas en sem 28) frente a mismos pacs entre el momento basal y la sem 28 OHA
    -Cambio en núm de lesiones OH, según TC, desde sem 28 hasta 56 (en pacs que cambien desde placebo en período doble enmascaramiento y presenten lesiones OH activas en sem 28) frente a mismos pacs entre momento basal y sem 28 -GCA
    -Cambio % respecto momento basal en la actividad total de lesiones determinada por PET con 18F-NaF en sem 56 -OHAC
    -Cambio % respecto momento basal en la actividad total de lesiones determinada por PET con 18F-NaF en sem 56 -OHA
    -Cambio % respecto momento basal en volumen total de lesiones OH evaluado por TC en sem 56 -OHAC
    -Cambio % respecto momento basal en volumen total de lesiones OH evaluado por TC en sem 56 -OHA
    -Cambio respecto momento basal en volumen total de lesiones OH evaluado por TC en sem 56 -GCA
    -Cambio promedio ponderado en tiempo (ABC estandarizada) respecto momento basal en dolor diario provocado por FOP, evaluado por NRS diaria durante 28 sems -GCA
    -Incidencia y gravedad de AAST
    -Cambio % promedio ponderado en tiempo (ABC estandarizada) respecto momento basal en biomarcadores de niveles de osificación en suero durante 28 sem, incluidos propéptido aminoterminal del procolágeno de tipo 1 (P1NP) total, fosfatasa alcalina específica de hueso (FAEH) y fosfatasa alcalina total (FAt) -GCA
    -Concentración activina A total en suero a lo largo del tiempo
    - Perfil FC REGN2477, evaluado a partir de concentraciones de REGN2477 en suero a lo largo del tiempo
    - Inmunogenia de REGN2477, según la incidencia, valor cuantitativo e impacto clínico de AAF contra REGN2477 surgidos a lo largo del tiempo durante el tratamiento
    E.5.2.1Timepoint(s) of evaluation of this end point
    Through study (week 8, 28 and 56) and end of study, week 76.
    A lo largo del estudio (semanas 8, 28 y 56) y al final del estudio, semana 76.
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    Tolerabilidad
    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) No
    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
    Estudio 3 periodos -1:descrito arriba; 2:abierto REGN2477; 3:tto de seguimiento RGN2477 hasta sem 76
    3-period study - 1: per above; 2: open label REGN2477; 3: Follow-up Trt with RGN2477 till wk76
    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 EEA7
    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
    Canada
    Colombia
    France
    Italy
    Mexico
    Netherlands
    Poland
    Spain
    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
    The end of study for this study is defined as the last visit of the last patient.
    El final del estudio se define como la última visita del último paciente.
    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 months5
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months5
    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 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 40
    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 40
    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)
    Standard treatment
    Tratamiento convencional
    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 Decision2019-01-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-01-16
    P. End of Trial
    P.End of Trial StatusTemporarily Halted
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