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    Summary
    EudraCT Number:2015-003681-87
    Sponsor's Protocol Code Number:BMN-044-201
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-11-16
    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 ConcernedBelgium - FPS Health-DGM
    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
    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)
    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.5.6E-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 injection/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 (company code)
    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 No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product 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 injection/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 (company code)
    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 No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product Yes
    D.3.11.13.1Other medicinal product typeAntisense oligonucleotide
    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
    E.1.1.1Medical condition in easily understood language
    Duchenne's disease
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.1
    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
    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.
    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 research-related procedures.
    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.
    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 non-standard 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
    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
    E.5.2Secondary end point(s)
    n/a
    E.5.2.1Timepoint(s) of evaluation of this end point
    n/a
    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
    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 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 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 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
    Belgium
    Italy
    Netherlands
    Sweden
    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
    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
    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.1.1Number of subjects for this age range: 50
    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
    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 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.
    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 Decision2015-12-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-01-12
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-09-09
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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