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    Summary
    EudraCT Number:2016-001654-18
    Sponsor's Protocol Code Number:CN001016
    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:2018-02-07
    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 number2016-001654-18
    A.3Full title of the trial
    A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys with Duchenne Muscular Dystrophy
    Studio randomizzato, in doppio cieco, controllato con placebo, per valutare l’efficacia, la sicurezza e la tollerabilità di BMS-986089 in bambini deambulanti affetti da distrofia muscolare di Duchenne
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of BMS-
    986089 in Ambulatory Boys With Duchenne Muscular Dystrophy
    Sperimentazione clinica per valutare l’efficacia, la sicurezza e la tollerabilità di BMS-986089 in bambini deambulanti affetti da distrofia muscolare di Duchenne
    A.3.2Name or abbreviated title of the trial where available
    -
    -
    A.4.1Sponsor's protocol code numberCN001016
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberISRCTN00000000
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT00000000
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1181-8752
    A.5.4Other Identifiers
    Name:CN001-016Number:Amend 03
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/84/2017
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION
    B.1.3.4CountryBelgium
    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 supportBristol-Myers Squibb International Corporation
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBristol-Myers Squibb International Corporation
    B.5.2Functional name of contact pointGCT-SU
    B.5.3 Address:
    B.5.3.1Street AddressParc de l'Alliance - Avenue de Finlande, 4
    B.5.3.2Town/ cityBraine-l'Alleud
    B.5.3.3Post code1420
    B.5.3.4CountryBelgium
    B.5.4Telephone number0000000000
    B.5.5Fax number0000000000
    B.5.6E-mailclinical.trials@bms.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 nameBMS-986089-01 Injections, 7.5 mg/Syringe (10.7 mg/mL)
    D.3.2Product code BMS-986089
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAnti-Myostatin Adnectin
    D.3.9.2Current sponsor codeBMS-986089
    D.3.9.3Other descriptive nameBMS-986089-01, anti-myostatin
    D.3.9.4EV Substance CodeSUB181218
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10.7
    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 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 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.IMP: 2
    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 nameBMS-986089-01 Injections, 15 mg/Syringe (21.4 mg/mL)
    D.3.2Product code BMS-986089
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAnti-Myostatin Adnectin
    D.3.9.2Current sponsor codeBMS-986089
    D.3.9.3Other descriptive nameBMS-986089-01, anti-myostatin
    D.3.9.4EV Substance CodeSUB181218
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number21.4
    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 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 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.IMP: 3
    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 nameBMS-986089-01 Injections, 35 mg/Syringe (50 mg/mL)
    D.3.2Product code BMS-986089
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAnti-Myostatin Adnectin
    D.3.9.2Current sponsor codeBMS-986089
    D.3.9.3Other descriptive nameBMS-986089-01, anti-myostatin
    D.3.9.4EV Substance CodeSUB181218
    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 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 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.IMP: 4
    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 nameBMS-986089-01 Injections, 50 mg/Syringe (71.4 mg/mL)
    D.3.2Product code BMS-986089
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAnti-Myostatin Adnectin
    D.3.9.2Current sponsor codeBMS-986089
    D.3.9.3Other descriptive nameBMS-986089-01, anti-myostatin
    D.3.9.4EV Substance CodeSUB181218
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number71.4
    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 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 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Duchenne Muscular Dystrophy
    Distrofia muscolare di Duchenne
    E.1.1.1Medical condition in easily understood language
    Duchenne muscular dystrophy (DMD) is a genetic disorder characterized
    by progressive muscle degeneration and weakness.
    La distrofia muscolare di Duchenne (DMD) è un disturbo genetico caratterizzato da degenerazione e debolezza muscolare progressive.
    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 10013801
    E.1.2Term Duchenne muscular dystrophy
    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 compare the efficacy of BMS-986089 to placebo in ambulatory boys with Duchenne Muscular Dystrophy.
    Confrontare l'efficacia di BMS-986089 rispetto al placebo in ragazzi deambulanti con distrofia muscolare di Duchenne.
    E.2.2Secondary objectives of the trial
    tests: North Star Ambulatory Assessment (NSAA), Stand from supine
    velocity, 10 M walk/run velocity, PODCI transfers and basic mobility
    subscale, Proximal lower extremity flexor (knee extension and knee
    flexion) strength, measured using manual myometry, 6 Minute Walk
    Distance (6MWD).
    • To assess the safety and tolerability of BMS-986089 in boys with DMD
    as reflected by new or worsening lab abnormalities (as defined by CTCAE
    criteria), serious adverse events (SAEs) and adverse events (AEs)
    leading to discontinuation.
    • Confrontare l'efficacia di BMS-986089 rispetto al placebo utilizzando i seguenti test: Valutazione della deambulazione North Star (NSAA), Velocità di passaggio dalla posizione supina alla posizione eretta, Velocità di corsa/camminata per 10 M, Scala PODCI di valutazione degli spostamenti e della mobilità di base, Forza del flessore prossimale dell'arto inferiore (estensione e flessione del ginocchio), misurata mediante miometria manuale, Distanza percorsa in 6 minuti (6MWD).
    • Valutare la sicurezza e la tollerabilità di BMS-986089 in ragazzi con DMD sulla base di anomalie di laboratorio nuove o in peggioramento (come definite dai criteri CTCAE), eventi avversi seri (SAE) ed eventi avversi (AE)
    che conducono alla sospensione del trattamento.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Additional research collections and retention are optional for all subjects, except where prohibited by local laws or regulations. This protocol will include both sample collection and residual sample storage for additional research (AR).
    This serum collection for additional research is intended to expand the translational R&D capability at Bristol-Myers Squibb, and will support as yet undefined research aims that will advance our understanding of disease and options for treatment. For example, this sample may have
    potential in exploration of diagnostic or prognostic biomarkers contingent on scientific developments in the field of myostatin biology, DMD pathophysiology or unanticipated results in the study. This collection for additional research is intended to expand the translational R&D capability at Bristol-Myers Squibb, and will support as yet undefined research aims that will advance our understanding of disease and options for treatment. It may also be used to support health authority requests for analysis, and advancement of pharmacodiagnostic development to better target drugs to the right patients. This may also
    include genetic/genomic exploration aimed at exploring disease pathways, progression and response to treatment etc.
    Ulteriori raccolte e conservazione per la ricerca sono facoltative per tutti i sogg., ad eccezione dei casi in cui sia vietato da leggi o normative locali. Questo prot. includerà la raccolta di campioni e la conservazione dei campioni residui per l'ulteriore ricerca (AR). La raccolta di campioni di siero per l’ulteriore ricerca è volta ad ampliare la capacità traslazionale di R&D presso Bristol-Myers Squibb e fornirà supporto per eventuali scopi della ricerca non ancora definiti che permetteranno di compiere progressi nella conoscenza della malattia e delle opzioni di tratt. Ad es., questo campione potrebbe avere potenziale utilità nell’esplorazione di biomarcatori diagnostici o prognostici legati a sviluppi scientifici nel campo della biologia della miostatina, della fisiopatologia della DMD o di risultati dello studio non previsti. La raccolta di campioni per l’ulteriore ricerca è volta ad ampliare la capacità traslazionale di R&D presso Bristol-Myers Squibb e fornirà supporto a scopi della ricerca non ancora definiti che permetteranno di compiere progressi nella conoscenza della malattia e delle opzioni di trattamento. Potrà essere utilizzata a supporto delle richieste alle autorità sanitarie ai fini dell’analisi e del progresso dello sviluppo farmacodiagnostico per una migliore assegnazione mirata dei farmaci ai pazienti appropriati. Ciò potrà includere l'esplorazione genetica/genomica volta a indagare le vie della malattia, la progressione e la risposta al trattamento ec
    E.3Principal inclusion criteria
    Key Inclusion Criteria:
    • Males, 6 >= to < 12 years of age at time of randomization.
    • Diagnosis of DMD, confirmed by medical history (eg., onset of clinical
    signs or symptoms before 5 years of age together with an elevated
    serum creatine kinase level observed before or after initial diagnosis)
    and by genotyping.
    • Participants >= 15 kg.
    • Ambulatory without assistance.
    • Participants must be receiving corticosteroids (prednisone,
    prednisolone, or deflazacort) for at least 6 months prior to the start of
    study drug, with no significant change in dosage (> 0.2 mg/kg
    prednisone or > 0.24 mg/kg deflazacort) or dosing regimen for at least 3
    months prior to the start of study drug, with the expectation that dosage
    and dosing regimen will not change significantly for the duration of the
    study.
    • 4SC<= 8 seconds at screening.
    • Participants must agree to avoid major changes in their physical or
    respiratory therapy regimen during the double blind phase, to the extent
    possible.
    • Males who are sexually active with WOCBP must agree to follow
    instructions for method(s) of contraception for the duration of
    treatment with study treatment(s) plus 5 half-lives of the study
    treatment [BMS-986089; 50 days] plus 90 days (duration of sperm
    turnover) for a total of 140 days (5 months) post-treatment completion.
    For the rest of Inclusion Criteria, please refer to study protocol Section
    6.1 (page 50).
    Principali criteri di inclusione:
    • Maschi, di età compresa tra  6 e < 12 anni al momento della randomizzazione.
    • Diagnosi di DMD, confermata dall'anamnesi medica (ad es. insorgenza di segni o sintomi clinici prima dei 5 anni di età, associati ad alti livelli sierici di creatinchinasi osservati prima o dopo la diagnosi iniziale) e mediante genotipizzazione.
    • Partecipanti  15 kg.
    • Deambulanti senza assistenza.
    • I partecipanti devono essere in trattamento con corticosteroidi (prednisone, prednisolone o deflazacort) da almeno 6 mesi prima dell'inizio del trattamento con il farmaco in studio, al cui dosaggio (> 0,2 mg/kg) o regime di dosaggio non sia stata apportata alcuna modifica significativa per almeno 3 mesi prima dell'inizio del trattamento con il farmaco in studio, e per il cui dosaggio e regime di dosaggio non sia prevista alcuna modifica significativa per tutta la durata dello studio.
    4SC≤ 8 secondi allo screening.
    • I partecipanti devono accettare di evitare modifiche rilevanti nel loro regime di terapia fisica o respiratoria durante la fase in doppio cieco, nella misura possibile.
    • I soggetti di sesso maschile che sono sessualmente attivi con donne in età fertile devono acconsentire ad attenersi alle istruzioni relative ai metodi contraccettivi per tutta la durata del trattamento con il/i farmaco/i in studio più 5 emivite di trattamento in studio [BMS-986089; 50 giorni] più 90 giorni (durata della rotazione spermatica) per un totale di 140 giorni (5 mesi) dopo il completamento del trattamento. Per i criteri di inclusione rimanenti, fare riferimento alla Sezione 6.1 del protocollo di studio (pagina 50).
    E.4Principal exclusion criteria
    • Participants with cognitive impairment or behavioral issues that, in the judgement of the investigator, will compromise their ability to comply
    with study procedures.
    • Participants on intermittent corticosteroid regimens with off periods of 20 days or longer (eg.: 10 days on, 20 days off).
    • Any change (initiation, change in drug class, dose modification unrelated to change in body weight, interruption or re-initiation) in prophylaxis /treatment for congestive heart failure (CHF) within 3 months prior to start of study treatment.
    • Any change (initiation, change in drug class, dose modification unrelated to change in body weight, interruption or re-initiation) in prophylaxis/treatment for bone density within 3 months prior to start of study treatment.
    • Treatment with exon skipping therapies within 6 months prior to the start of study drug administration.
    • Treatment with ataluren, or any other investigational drug (excluding deflazacort and exon skipping therapies) currently or within 3 months prior to the start of study drug administration.
    • Participants with a FVC of < 50% (in participants able to produce a valid FVC, as judged by the clinical evaluator or respiratory therapist).
    • Cutaneous AEs sustained during participation in a prior clinical trial that resolved less than 3 months prior to the start of study drug
    administration.
    • Current or prior treatment within 3 months prior to the start of study drug administration with androgens or human growth hormone.
    • Prior treatment with BMS-986089 or any other anti-myostatin agent.
    • History of lower limb fracture within 3 months prior to the start of study drug administration.
    • History of upper limb fracture within 2 months prior to the start of study drug administration.
    • Any injury which may impact functional testing. Previous injuries must be fully healed prior to consenting.
    • Expectation of major surgical procedure, such as scoliosis surgery, during the double blind phase of this study.
    • Requirement of daytime ventilator assistance.
    • Initiation of nighttime ventilation less than 1 month prior to the start of study drug administration.
    • Expectation that daytime or nighttime ventilation may be initiated during the double blind phase of this study.
    • Uncontrolled clinical signs or symptoms of congestive heart failure (American College of Cardiology/American Heart Associated Stage C or
    Stage D).
    • For participants participating in cMRI substudy: implanted ferromagnetic metal (implanted metal that is not ferromagnetic, such as surgical steel or titanium implants may be allowed if the implants will not compromise the quality of the cMRI).
    • History of hypersensitivity to components of the study drug (histidine, trehalose, diethylenetriaminepentaacetic acid, polysorbate 80).
    • Unwilling or unable to administer study drug at home.
    For the rest of Exclusion Criteria, please refer to study protocol Section 6.2 (page 51).
    Principali criteri di esclusione:
    • Partecipanti con compromissione cognitiva o problemi comportamentali che, a giudizio dello sperimentatore, comprometteranno la loro capacità di rispettare le procedure dello studio.
    • Partecipanti che seguono regimi di trattamento intermittenti con corticosteroidi con periodi di
    sospensione di 20 giorni o più (per es.: 10 giorni di trattamento, 20 giorni di sospensione).
    • Qualsiasi modifica (inizio, variazione della classe di farmaci, modifica della dose non correlata a variazione di peso corporeo, interruzione o ripresa) di profilassi/trattamento per insufficienza cardiaca congestizia (ICC) entro 3 mesi prima dell'inizio del trattamento in studio.
    • Qualsiasi modifica (inizio, variazione della classe di farmaci, modifica della dose non correlata a variazione di peso corporeo, interruzione o ripresa) di profilassi/trattamento per la densità ossea entro 3 mesi prima dell'inizio del trattamento in studio.
    • Trattamento con terapie di "salto dell'esone" entro 6 mesi prima dell'inizio della somministrazione del farmaco in studio.
    • Trattamento con ataluren, o qualsiasi altro farmaco sperimentale (escluso il deflazacort e le terapie di salto dell'esone) in corso o entro 3 mesi prima dell'inizio della somministrazione del farmaco in studio.
    • Partecipanti con una FVC < 50% (tra i partecipanti in grado di produrre una FVC che possa essere ritenuta valida, in base al giudizio di un esaminatore clinico o di un fisioterapista respiratorio).
    • AE cutanei prolungati durante la partecipazione a una precedente sperimentazione clinica che si siano risolti meno di 3 mesi prima di iniziare la somministrazione del farmaco in studio.
    • Trattamento in corso o precedente con ormoni androgeni o ormone della crescita umano entro 3 mesi prima dell'inizio della somministrazione del farmaco in studio.
    • Trattamento precedente con BMS-986089 o qualsiasi altro agente anti-miostatina.
    • Anamnesi di frattura di arto inferiore entro 3 mesi prima dell'inizio della somministrazione del farmaco in studio.
    • Anamnesi di frattura di arto superiore entro 2 mesi prima dell'inizio della somministrazione del farmaco in studio.
    • Qualsiasi danno fisico che possa avere un impatto sui test di funzionalità. I danni precedenti devono essere completamente guariti prima del consenso.
    • Probabile intervento chirurgico rilevante, come intervento per la scoliosi, durante la fase in doppio cieco di questo studio.
    • Necessità di assistenza per ventilazione diurna.
    • Avvio di ventilazione notturna meno di 1 mese prima di iniziare la somministrazione del farmaco in studio.
    • Previsione di possibile avvio della ventilazione diurna o notturna durante la fase in doppio cieco di questo studio.
    • Segni o sintomi clinici incontrollati di insufficienza cardiaca congestizia (secondo l'American College of Cardiology/American Heart Association, stadio C o stadio D).
    • Per i partecipanti che prendono parte al sottostudio di cMRI: impianto in metallo ferromagnetico (impianti in metallo non ferromagnetico, come impianti in acciaio chirurgico o titanio, possono essere ammesso se non compromettono la qualità della cMRI).
    • Anamnesi di ipersensibilità ai componenti del farmaco in studio (istidina,trealosio, acido dietilentriamminopentacetico, polisorbato 80).
    • Partecipanti non disposti o non in grado di autosomministrarsi il farmaco in studio a domicilio.
    Per i criteri di esclusione rimanenti, fare riferimento alla Sezione 6.2 del protocollo di studio (pagina 51).
    E.5 End points
    E.5.1Primary end point(s)
    The change from baseline in the 4 stair climb velocity at Week 48 in
    BMS-986089 treated participants compared to placebo treated
    participants.
    Variazione rispetto alla baseline nella prova di velocità di salita di 4 gradini alla Settimana 48 nei partecipanti trattati con BMS-986089 rispetto ai partecipanti trattati con placebo.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day 1, week 12, week 24, week 36 and week 48 during double blind
    phase.
    Week 1, week 12, week 24, week 36 and week 48 during open label
    phase.
    Giorno 1, settimana 12, settimana 24, settimana 36 e settimana 48 durante la fase in doppio cieco.
    Settimana 1, settimana 12, settimana 24, settimana 36 e settimana 48 durante la fase in aperto.
    E.5.2Secondary end point(s)
    • Change from baseline at Week 48 in BMS-986089 treated participants compared to placebo treated participants in: North Star Ambulatory Assessment (NSAA), Stand from supine velocity, 10 M walk/run velocity, PODCI transfers and basic mobility subscale, Proximal lower extremity flexor (knee extension and knee flexion) strength, measured using manual myometry, 6 Minute Walk Distance (6MWD)
    •Tabulations of the numbers of unique participants with new or worsening laboratory abnormalities, SAEs and AEs leading to discontinuation, in BMS 986089 arms compared to the placebo arm.
    • Variazione rispetto alla baseline alla Settimana 48 nei partecipanti trattati con BMS-986089 rispetto ai partecipanti trattati con placebo di: Valutazione della deambulazione North Star (NSAA), Velocità di passaggio dalla posizione supina alla posizione eretta, Velocità di corsa/camminata per 10 M, Scala PODCI di valutazione degli spostamenti e della mobilità di base, Forza del flessore prossimale dell'arto inferiore (estensione e flessione del ginocchio) misurata mediante miometria manuale, Distanza percorsa in 6 minuti (6MWD)
    •Tabulazioni dei numeri di singoli partecipanti con anomalie di laboratorio nuove o in peggioramento, SAE e AE che conducono alla sospensione del trattamento nei bracci trattati con BMS-986089 rispetto al braccio trattato con placebo.

    E.5.2.1Timepoint(s) of evaluation of this end point
    Day 1, week 12, week 24, week 36 and week 48 during double blind phase. Week 1, week 12, week 24, week 36 and week 48 during open label
    phase.
    Giorno 1, settimana 12, settimana 24, settimana 36 e settimana 48 durante la fase in doppio cieco.
    Settimana 1, settimana 12, settimana 24, settimana 36 e settimana 48 durante la fase in aperto.
    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 Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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 Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    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 trial3
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA17
    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
    Argentina
    Australia
    Belgium
    Canada
    France
    Germany
    Italy
    Japan
    Netherlands
    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
    End of trial is defined as the last visit or scheduled procedure shown in the Schedule of Activities for the last participant. Study completion is defined as the final date on which data for the primary endpoint was or is expected to be collected, if this is not the same.
    La Fine della sperimentazione è definita come l’ultima visita o procedura programmata indicata nel Programma delle attività per l’ultimo partecipante. Il completamento dello studio è definito come l’ultima data in cui i dati per l’endpoint primario sono stati raccolti o in cui si prevedeva la raccolta, se non coincidono.
    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 months8
    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 months8
    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.1.1Number of subjects for this age range: 1
    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: 160
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    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 No
    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
    Study subjects are minors.
    I soggetti partecipanti allo studio sono minori
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 39
    F.4.2.2In the whole clinical trial 160
    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)
    At the conclusion of the study, participants who continue to demonstrate clinical benefit will be eligible to receive BMS supplied study treatment. Study treatment will be provided via an extension of
    the study, a rollover study requiring approval by responsible health authority and ethics committee or through another mechanism at the discretion of BMS.
    Alla conclusione dello studio, i partecipanti che continuano a mostrare beneficio clinico saranno eleggibili per ricevere il trattamento in studio fornito da BMS. Il trattamento in studio sarà fornito attraverso un'estensione dello studio, uno studio di rollover che richiede l'approvazione da parte dell'autorità sanitaria responsabile e del Comitato Etico o attraverso un altro meccanismo, a discrezione di BMS.
    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 Decision2017-09-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-06-22
    P. End of Trial
    P.End of Trial StatusOngoing
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
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