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    Summary
    EudraCT Number:2013-003605-26
    Sponsor's Protocol Code Number:PRO044-CLIN-02
    National Competent Authority:Sweden - MPA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2014-09-26
    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 ConcernedSweden - MPA
    A.2EudraCT number2013-003605-26
    A.3Full title of the trial
    A phase II, open label, extension study to assess the effect of PRO044 in patients 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 PRO044 in patients with Duchenne muscular dystrophy (Extension study to PRO044-CLIN-01)
    A.4.1Sponsor's protocol code numberPRO044-CLIN-02
    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 Nederland B.V.
    B.1.3.4CountryNetherlands
    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 Nederland B.V.
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBioMarin Nederland B.V.
    B.5.2Functional name of contact pointMichael Odontiadis
    B.5.3 Address:
    B.5.3.1Street AddressJ. H. Oortweg 21
    B.5.3.2Town/ cityLeiden
    B.5.3.3Post code2333 CH
    B.5.3.4CountryNetherlands
    B.5.4Telephone number442074200800
    B.5.5Fax number442074200829
    B.5.6E-mailinfo@bmrn.nl
    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 namePRO044
    D.3.2Product code PRO044
    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.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 assess the safety, tolerability and efficacy of two different doses of intravenous (IV) PRO044 and one dose of subcutaneous (SC) PRO044 in subjects with DMD after 48 weeks treatment.
    E.2.2Secondary objectives of the trial
    •To assess the safety, tolerability and efficacy of two different doses of intravenous (IV) PRO044 and one dose of subcutaneous (SC) PRO044 in subjects with DMD over time.
    •To assess the pharmacokinetic profile of two different dosages and different routes of administration of PRO044 in subjects with DMD.
    •To assess the pharmacodynamic (PD) effects of two different dosages and different routes of administration of PRO044 in subjects with DMD.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subjects previously treated with PRO044 in the PRO044-CLIN-01 study.

    2. Continued use of glucocorticoids for a minimum of 60 days prior to study entry with a reasonable expectation that the subject will remain on steroids for the duration of the study. Changes to the dose regimen or cessation of glucocorticoids will be at the discretion of the Principle Investigator (PI) in consultation with the subject/parent and the Medical Monitor. If the subject is not on steroids, involvement in the study needs to be discussed with the medical monitor.
    E.4Principal exclusion criteria
    1. Current, or history of, liver or renal disease.
    2. Acute illness within 4 weeks prior to the first dose of PRO044 (Week 1) which may interfere with the measurements.
    3. Severe cardiac myopathy which in the opinion of the Investigator prohibits participation in this study
    4. Need for daytime mechanical ventilation.
    5. Screening aPTT above the upper limit of normal (ULN).
    6. Screening platelet count below the lower limit of normal (LLN).
    7. Use of anti-coagulants, anti-thrombotics or anti-platelet agents.
    8. Use of any investigational product within 6 months prior to the start of screening for the study.
    9. Current or history of drug and/or alcohol abuse.
    E.5 End points
    E.5.1Primary end point(s)
    Safety Parameters:
    •Adverse events
    •Tolerability
    •Physical examination
    •Vital signs (temperature, blood pressure, pulse rate, respiration rate)
    •Laboratory assessments including:
    -Routine biochemistry and hematology
    -Urinalysis (routine parameters plus alpha1-microglobulin, microscopy) and 24-hour urine (additionally including protein electrophoresis, urine cystatin C, KIM 1)
    -Coagulation parameters (aPTT, PT [INR], fibrinogen)
    -Complement C3 (including split fragments C3a, SC5b and Bb)
    -Complement C4 and complement Factor H
    -Pro-inflammatory markers haptoglobulin, MCP 1
    -Anti-dystrophin antibodies
    -Anti-PRO044 antibodies
    •ECG
    •Echocardiography
    •Renal ultrasound.
    Efficacy parameters:
    •Muscle Function
    -6 Minute Walk Distance (6MWD)
    -North Star Ambulatory Assessment
    -Timed tests (10-meter walk/run, rising from floor, stair climb)
    -DMD Functional Outcomes Questionnaire (DMD-FOS) –for ambulant subjects only
    -Egen Klassification - for non-ambulant subjects.
    •Muscle strength
    -Pulmonary Function (Spirometry)
    -Handheld myometry.
    •Exploratory:
    -Performance Upper Limb (PUL).
    -Patient Reported Outcome measure (PROM).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 48
    E.5.2Secondary end point(s)
    Pharmacokinetic parameters:
    •t ½
    •AUC: 0-24h, 0-∞ (where applicable)
    •Cmax
    •tmax
    •CL (for IV subjects) or CL/F (for SC subjects)
    •PRO044 concentrations in muscle tissue.
    Pharmacodynamic parameters:
    •Presence of dystrophin expression after treatment (muscle biopsy)
    •Nuclear Magnetic Resonance imaging (MRI and MRS) in some subjects in study centers with appropriate imaging capabilities and training.
    •Exploratory biomarkers (e.g., MMP 9, miRNA 1, miRNA-133).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 48
    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 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) 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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial3
    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 No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    Last patient last visit
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    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: 18
    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: 5
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 3
    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 18
    F.4.2.2In the whole clinical trial 18
    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)
    Following completion of the 48 weeks study duration, subjects will be offered continued PRO044 dosing in an extension study, BMN-044-201.
    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 Decision2014-11-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-11-26
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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