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    Summary
    EudraCT Number:2015-003681-87
    Sponsor's Protocol Code Number:BMN-044-201
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2021-01-19
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2015-003681-87
    A.3Full title of the trial
    A multi center, multi national, open label, extension study to evaluate the long-term efficacy and safety of BMN 044 (PRO044) in subjects with Duchenne muscular dystrophy
    Studio di estensione, in aperto, multicentrico, multinazionale, volto a valutare l'efficacia a lungo termine e la sicurezza di BMN 044 (PRO044) in soggetti affetti da Distrofia Muscolare di Duchenne (DMD).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to assess the effect of BMN 044 in subjects with Duchenne
    muscular dystrophy (Extension study)
    Studio per valutare l'effetto di BMN 044 in soggetti affetti da Distrofia Muscolare di Duchenne ( studio di estensione)
    A.3.2Name or abbreviated title of the trial where available
    NA
    NA
    A.4.1Sponsor's protocol code numberBMN-044-201
    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 SponsorBIOMARIN PHARMACEUTICAL 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 supportBioMarin Pharmaceutical Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBioMarin Pharmaceutical Inc.
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street Address105 Digital Drive
    B.5.3.2Town/ cityNovato
    B.5.3.3Post codeCA 94949
    B.5.3.4CountryUnited States
    B.5.4Telephone number000000000000000000
    B.5.5Fax number000000000000000000
    B.5.6E-mailclinicaltrials@bmrn.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/3/08/598
    D.3 Description of the IMP
    D.3.1Product nameBMN 044
    D.3.2Product code BMN 044
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    Subcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 1802402-63-6
    D.3.9.2Current sponsor codePS188
    D.3.9.3Other descriptive namePS188
    D.3.9.4EV Substance CodeSUB179970
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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 Yes
    D.3.11.13.1Other medicinal product typeAntisense oligonucleotide
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/08/598
    D.3 Description of the IMP
    D.3.1Product nameBMN 044
    D.3.2Product code BMN 044
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    Subcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPS188
    D.3.9.1CAS number 1802402-63-6
    D.3.9.2Current sponsor codePS188
    D.3.9.3Other descriptive namePS188
    D.3.9.4EV Substance CodeSUB179970
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 Yes
    D.3.11.13.1Other medicinal product typeOligonucleotide antisenso
    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
    Duchenne muscular dystrophy
    Distrofia Muscolare di Duchenne
    E.1.1.1Medical condition in easily understood language
    Duchenne's disease
    malattia di Duchenne
    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 evaluate the long-term safety of BMN 044 in subjects with DMD
    correctable by BMN 044 induced DMD exon 44 skipping who have
    previously participated in an eligible study
    ¿ valutare la sicurezza a lungo termine di BMN 044 in soggetti affetti da DMD trattabile con skipping BMN 044-indotto dell'esone DMD 44, che hanno partecipato precedentemente a uno studio idoneo
    E.2.2Secondary objectives of the trial
    Treatment Period 1:
    ¿ To evaluate the long-term efficacy of the dosing regimens used in prior
    BMN 044 studies.
    ¿ To evaluate the long-term safety and tolerability of the dosing
    regimens used in prior BMN 044 studies.
    Treatment Period 2:
    ¿ To evaluate the long-term efficacy of a selected dosing regimen(s) for
    BMN 044.
    ¿ To evaluate the long-term safety and tolerability of a selected dosing
    regimen(s) for BMN 044.
    ¿ To evaluate the long-term impact on patient reported outcomes of continued treatment with BMN 044.
    Periodo di trattamento 1:
    ¿ valutare l'efficacia a lungo termine dei regimi posologici impiegati negli studi precedenti su BMN 044.
    ¿ valutare la sicurezza e la tollerabilit¿ a lungo termine dei regimi posologici impiegati negli studi precedenti su BMN 044.
    Periodo di trattamento 2:
    ¿ valutare l'efficacia a lungo termine di un/alcuni regime(i) posologico(i) selezionato(i) di BMN 044.
    ¿ valutare la sicurezza e la tollerabilit¿ a lungo termine di un/alcuni regime(i) posologico(i) selezionato(i) di BMN 044.
    ¿ valutare l'influenza a lungo termine del trattamento continuo con BMN 044 sui risultati riferiti dai pazienti.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subjects previously treated with BMN 044 or a comparator treatment
    in a BMN 044 Sponsored Study or Investigator Initiated Trial and who
    are not eligible for another ongoing BMN 044 study. Subjects who
    withdrew from any previous BMN 044 study due to meeting laboratory safety
    stopping criteria may be eligible to enroll if the applicable laboratory
    parameter has resolved to be within normal limits or parent study baseline value, and benefit of further treatment with BMN 044
    outweighs the risk to the individual subject, as agreed in consultation
    with the Medical Monitor.
    2. Continued use of glucocorticosteroids for a minimum of 60 days prior to study entry with a reasonable expectation that the subject will remain
    on glucocorticosteroids for the duration of this study. Changes to or
    cessation of glucocorticosteroids will be at the discretion of the
    Investigator conducting this study in consultation with the
    subject/parent and Medical Monitor. Subjects who have discontinued
    glucocorticosteroids in their previous BMN 044 study, following
    consultation with the Medical Monitor may be enrolled.
    3. Willing and able to comply with all study requirements and
    procedures.
    4. Willing and able to provide written, signed informed consent, or in the
    case of subjects under the age of 18 years(or 16 years, depending on the
    region), provide written assent (if required) and written informed
    consent by a legally authorized representative after the nature of the
    study has been explained, and prior to the conduct of any researchrelated
    procedures.
    1. Soggetti trattati precedentemente con BMN 044 o con un comparatore in uno Studio Sponsorizzato su BMN 044 o intrapreso dallo Sperimentatore e non idonei per un altro studio su BMN 044 in corso. I soggetti che si sono ritirati da qualsiasi studio precedente BMN 044 per via di criteri di esclusione relativi alla sicurezza delle analisi di laboratorio possono essere idonei per l'arruolamento, se il parametro di laboratorio in questione è rientrato nei limiti dela norma o al valore del basale dello studio primario e il beneficio dell'ulteriore trattamento con BMN 044 è superiore al rischio individuale del soggetto, come stabilito dalla consultazione con il Monitor Medico.
    2. Uso continuo di glucocorticosteroidi per un minimo di 60 giorni prima dell'ingresso nello studio, con ragionevole aspettativa che il soggetto continui l'assunzione per la durata dello studio. Modifiche o sospensioni del trattamento con glucocorticosteroidi saranno a discrezione dello Sperimentatore che guida lo studio, consultandosi con il soggetto/familiare e il Monitor Medico. I soggetti che hanno sospeso il trattamento con glucocorticosteroidi nel loro studio precedente su BMN 044, previa consultazione con il Monitor Medico, possono essere arruolati.
    3. Soggetti capaci e disposti a rispettare tutti i requisiti e le procedure dello studio.
    4. Soggetti capaci e disposti a fornire un consenso informato scritto e firmato, o nel caso di soggetti di età inferiore a 18 anni (o 16 anni, a seconda della regione), a fornire consenso scritto (se necessario) e consenso informato scritto da parte di un rappresentante legalmente autorizzato, dopo spiegazione della natura dello studio e prima dell'esecuzione di qualunque procedura legata alla ricerca.
    E.4Principal exclusion criteria
    1. Subjects who have previously been treated with BMN 044 who
    had a serious adverse experience or met safety stopping criteria
    that remains unresolved, which in the opinion of the Investigator could
    have been attributable to BMN 044. Once resolved, subject may be
    eligible to enter the study following Investigator consultation with the
    Medical Monitor.
    2. History of significant medical disorder which may confound the
    interpretation of safety data (e.g. current or history of renal or liver
    disease/impairment, history of inflammatory illness, bleeding
    complications, mental retardation and/or behavioral problems).
    3. Acute illness within 4 weeks prior to the first dose of BMN 044 (Week
    1) which may interfere with the measurements.
    4. Symptomatic cardiomyopathy. If subject has a left ventricular ejection
    fraction <45% at start of this study, the Investigator should discuss
    inclusion of subject in this study with the Medical Monitor.
    5. Baseline aPTT above the upper limit of normal (ULN). A re test is
    possible at a later stage, and if within normal range, the subject may
    enter the study.
    6. Baseline platelet count below the lower limit of normal (LLN). A re
    test is possible at a later stage, and if within normal range, the subject
    may enter the study.
    7. Use of anti coagulants, anti thrombotics or anti platelet agents within
    28 days of the baseline visit. Chronic use of anti coagulants, anti
    thrombotics or anti platelet agents is prohibited during the study. As
    needed dosing (pro re nata – PRN) may be acceptable (except for
    aspirin) following discussion with the Medical Monitor.
    8. Prior use of any investigational product (other than BMN 044) or
    investigational medical device must be discussed with the Medical
    Monitor prior to screening.
    9. Current or history of drug and/or alcohol abuse.
    1. Soggetti trattati precedentemente con BMN 044 che hanno riportato una reazione avversa grave o in cui non si è verificata risoluzione di uno dei criteri di esclusione di sicurezza, che in base all'opinione dello Sperimentatore potrebbe essere attribuibile a BMN 044. Una volta risolta la questione, il soggetto può essere considerato idoneo all'ingresso nello studio previa consultazione dello Sperimentatore con il Monitor Medico.
    2.Anamnesi di disturbi medici significativi che potrebbero alterare l'interpretazione dei dati sulla sicurezza (ad es. anamnesi o attuale malattia/danno renale o epatico, anamnesi di malattie a carattere infiammatorio, complicanze emorragiche, ritardo mentale e/o problemi comportamentali).
    3. Patologia in fase acuta a 4 settimane dalla prima dose di BMN 044 (Settimana 1) che può interferire con le misurazioni.
    4. Cardiomiopatia sintomatica. Se il soggetto ha una frazione dell'eiezione ventricolare sinistra <45% all'inizio dello studio, lo Sperimentatore deve discutere della sua inclusione con il Monitor Medico.
    5. Tempo di tromboplastina parziale attivata (aPTT) basale al di sopra del limite superiore della norma (ULN). È possibile eseguire un secondo test in una fase successiva; se i valori rientrano nella norma il soggetto può entrare a far parte dello studio.
    6. Conta delle piastrine basale al di sotto del limite inferiore della norma (LLN). È possibile eseguire un secondo test in una fase successiva; se i valori rientrano nella norma il soggetto può entrare a far parte dello studio.
    7. Uso di anticoagulanti, agenti antitrombotici o antiaggreganti nei 28 giorni precedenti la visita basale. L'uso cronico di anticoagulanti, agenti antitrombotici o antiaggreganti non è consentito durante lo studio. Le somministrazioni in caso di necessità (pro re nata - PRN) possono essere accettate (ad eccezione dell'aspirina) previa discussione con il Monitor Medico.
    8.L'uso precedente di qualsiasi prodotto (diverso da BMN 044) o dispositivo medico sperimentale deve essere discusso con il Monitor Medico prima dello screening.
    9.Anamnesi o storia attuale di abuso di droghe e/o alcol.
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy:
    Ambulant subjects:
    • Muscle function using:
    - 6 minute walking distance (6MWD) test
    - Timed function tests (4 stair climb, Rise from floor, 10 meter walk/run)North Star Ambulatory Assessment
    • Patient Reported Outcomes Measure (PROM), Patient Outcomes Data
    Collection Instrument (PODCI), EQ 5D 5L
    • Pulmonary function (forced expiratory volume in the 1st second of
    exhalation [FEV1], forced vital capacity [FVC], Maximum Inspiratory
    Pressure [MIP], Maximum Expiratory Pressure [MEP], Peak Cough Flow
    [PCF], and Peak Flow [PF])
    • Time to major disease milestones (e.g. loss of ambulation, night time
    ventilation)
    • Performance Upper Limb (PUL)
    Non-ambulant subjects:
    • Pulmonary function (FEV1, FVC, MIP, MEP, PCF, and PF)
    • Time to major disease milestones (e.g. night time ventilation)
    • PUL
    • Egen Klassification
    • PROM, PODCI, EQ 5D 5L
    Safety:
    • Incidence and severity of adverse events
    • Vital signs
    • ECG parameters
    • Injection Site Reactions
    • Safety hematology and biochemistry parameters including nonstandard
    parameters such as coagulation parameters (in particular
    aPTT), serum cystatin C, Complement Factor C3/C4/H, haptoglobin,
    fibrinogen, high sensitivity C-reactive protein (hsCRP)
    • Urinalysis (including quantitative protein and creatinine and their ratio)• Anti-dystrophin antibodies and anti-BMN 044 antibodies
    Pharmacodynamic:
    • Serum Creatine Kinase (CK), lactate dehydrogenase (LDH) and other
    biomarkers to be defined
    Pharmacokinetic:
    • Plasma PK pre-dose every 24 weeks
    Efficacia:
    Soggetti ambulanti:
    Uso della funzionalità muscolare:
    - Test del cammino dei 6 minuti (6MWD)
    - Test della funzionalità a tempo (salita 4 gradini, sollevarsi da terra, 10 metri cammino/corsa)
    • North Star Ambulatory Assessment
    • Patient Reported Outcomes Measure (PROM), Patient Outcomes Data Collection Instrument (PODCI), EDEQ-5D-5L
    • Funzionalità polmonare (Flusso espiratorio forzato nel primo secondo di espirazione [FEV1], Capacità vitale forzata [FVC],
    Massima Pressione Inspiratoria [MIP], Massima Pressione Espiratoria [MEP], Picco di Flusso della Tosse [PCF] e Picco di Flusso [PF])
    • Tempo delle fasi principali della malattia (ad es. perdita della deambulazione, ventilazione notturna)
    • Prestazioni degli arti superiori (Performance Upper Limb-PUL).
    Soggetti non ambulanti:
    Funzionalità polmonare (FEV1, FVC, MIP, MEP, PCF e PF)
    • Tempo delle fasi principali della malattia (ad es. ventilazione notturna)
    • PUL
    • Egen Klassification
    • PROM, PODCI, EDEQ-5D-5L
    Sicurezza
    • Incidenza e gravità degli eventi avversi
    • Segni vitali
    • Parametri ECG
    • Reazioni in sede di iniezione
    • Parametri di sicurezza ematologici e biochimici inclusi parametri non standard come quelli relativi a coagulazione (in particolare
    aPTT), cistatina C sierica, Fattore del Complemento C3/C4/H, aptoglobina, fibrinogeno, Proteina C reattiva ad alta sensibilità
    (hsCRP)
    • Esame delle urine (inclusa la valutazione quantitativa di proteine e creatinina e il loro rapporto)
    • Anticorpi antidistrofina e anticorpi anti-BMN044
    Farmacodinamica:
    • Creatinchinasi sierica (CK), lattato deidrogenasi (LDH) ed altri biomarcatori da definire
    Farmacocinetica:
    • Farmacocinetica plasmatica pre-dose ogni 24 settimane
    E.5.1.1Timepoint(s) of evaluation of this end point
    Please refer to Section 12.3.2 Study period procedures and assessments in the Protocol
    Si prega di far riferimento alla Sez. 12.3.2 del protocollo, procedure e test del periodo di studio
    E.5.2Secondary end point(s)
    NA
    NA
    E.5.2.1Timepoint(s) of evaluation of this end point
    NA
    NA
    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 No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    dose exploration
    esplorazione della dose
    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 No
    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 No
    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.8.3 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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA5
    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
    United States
    Belgium
    Italy
    Netherlands
    Sweden
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    A subject will continue in BMN-044-201 until one of the following
    occurs:
    ¿ The subject withdraws consent and discontinues from the study.
    ¿ The subject is discontinued from the study at the discretion of the
    Investigator or BioMarin.
    ¿ The study is terminated.
    ¿ The study drug receives marketing authorization in their country of
    residence
    LVLS
    I soggetti continueranno il trattamento con BMN-044-201 fino a quando non si verifica una delle seguenti condizioni:

    ¿ Il soggetto ritira il consenso e interrompe la partecipazione allo studio.
    ¿ Il soggetto viene escluso dallo studio a discrezione dello Sperimentatore o di BioMarin.
    ¿ Lo studio viene terminato.
    ¿ Il farmaco dello studio riceve l'autorizzazione all'immissione in commercio nel proprio paese di residenza.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    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 years5
    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: 23
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 2
    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
    Children under the age of 18
    Bambini di et¿ inferiore ai 18
    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 30
    F.4.2.2In the whole clinical trial 50
    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)
    This study will be active until marketing authorization or termination.
    Once BMN044 is commercially available, subjects can keep on their
    treatment with commercial supplies.
    Questo studio sar¿ attivo fino all'autorizzazione in commercio oppure fino alla cessazione. Una volta che BMN044 sar¿ disponibile in commercio, i soggetti potranno continuare il loro trattamento con il farmaco commerciale.
    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 Decision2016-03-08
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
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