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    Summary
    EudraCT Number:2016-001615-21
    Sponsor's Protocol Code Number:B5161004
    National Competent Authority:Bulgarian Drug Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2018-04-12
    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 ConcernedBulgarian Drug Agency
    A.2EudraCT number2016-001615-21
    A.3Full title of the trial
    A Multicenter, Open-Label Extension Study to Evaluate the Long Term Safety of PF-06252616 in Boys with Duchenne Muscular Dystrophy
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An extension to Protocol B5161002 to evaluate the long-term safety of as well as to collect data regarding maintenance of effect up to 4 years
    A.4.1Sponsor's protocol code numberB5161004
    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 SponsorPfizer Inc. 235 East 42nd Street, New York, NY 10017
    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 supportPfizer Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPfizer Inc.
    B.5.2Functional name of contact pointClinical Trials.gov Call Centre
    B.5.3 Address:
    B.5.3.1Street Address235 East 42nd Street
    B.5.3.2Town/ cityNew York, NY
    B.5.3.3Post code10017
    B.5.3.4CountryUnited States
    B.5.4Telephone number+18007181021
    B.5.5Fax number+13037391119
    B.5.6E-mailClinicaltrials.gov_Inquiries@pfizer.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/3/13/1105
    D.3 Description of the IMP
    D.3.2Product code PF-06252616
    D.3.4Pharmaceutical form Powder for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPF-06252616
    D.3.9.2Current sponsor codePF-06252616
    D.3.9.4EV Substance CodeSUB88240
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number260
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) Yes
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Duchenne's Muscular Dystrophy
    E.1.1.1Medical condition in easily understood language
    Duchenne's Muscular Dystrophy
    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 IV dosing of PF-06252616 in boys with DMD.
    E.2.2Secondary objectives of the trial
    - To evaluate the long-term efficacy of PF-06252616 using functional assessments and
    strength.
    - To assess the PK and immunogenicity of PF-06252616.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subjects with Duchenne muscular dystrophy who enrolled and completed through Week 97 of Study B5161002.
    2. Evidence of a personally signed and dated informed consent document (ICD) indicating that the subject’s parent or legal guardian/caregiver has been informed of all pertinent aspects of the study. Subjects may be required to provide assent in compliance with local regulations and IRB requirements.
    3. Subjects and their legal guardians/caregivers who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
    4. The following inclusion criteria will be assessed using data from the B5161002 study, and if data are unavailable, these assessments must be completed prior to enrollment:
    a. Adequate hepatic function on screening laboratory assessments from the Week 97 visit.
    b. GLDH ≤ 20 units/liter (2 x upper limit of normal [ULN]) from the Week 97 visit.
    c. Iron content estimate on the liver MRI within the normal range as determined by R2* value (R2*≤75 Hz at 1.5 T or R2*≤139 Hz at 3.0 T) from the Week 93 visit.
    E.4Principal exclusion criteria
    1. Unwilling or unable (eg, metal implants) to undergo examination with closed MRI. If subjects required sedation in the B5161002 study they will be permitted to enroll in the OLE. In the event that a subject becomes intolerant to MRI scanning, during the OLE, the subject may be separately consented to be administered sedation in order to complete the MRI.
    2. All male subjects who are able to father children and are sexually active and at risk for impregnating a female partner, who are unwilling or unable to use with their female partner(s) a highly effective method of contraception consistently and correctly for the duration of the active treatment period and through the final study visit. In addition, all sexually active male subjects who are unwilling or unable to prevent potential transfer of and exposure to drug through semen to their partners by using a condom consistently and
    correctly, beginning with the first dose of investigational product and continuing through the final study visit.
    3. Subjects who are investigational site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the Investigator, or subjects who are related to Pfizer employees directly involved in the conduct of the study.
    4. Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for entry into this study.
    5. Participation in other studies involving investigational drug(s), with the exception of B5161002, for a minimum of 30 days or within 5 half-lives (whichever is longer) prior to signing the informed consent and/or during study participation.
    6. History of allergic or anaphylactic reaction to a therapeutic or diagnostic protein or additives of this investigational product (histidine, sucrose, edetic acid [ethylenediaminetetraacetic acid], and polysorbate 80).
    E.5 End points
    E.5.1Primary end point(s)
    Safety:
    - Incidence and/or rate of intolerability or dose limiting treatment related AEs following up to 4 years of treatment.
    - Incidence and/or rate, severity and causal relationship of TEAEs and withdrawals due to TEAEs following up to 4 years of treatment.
    - Incidence and magnitude of abnormal laboratory findings (clinical laboratory tests: hematology, chemistry, GGT, GLDH, PT, aPTT, creatine kinase, amylase, serum ferritin, serum iron, total iron binding capacity (TIBC), % transferrin saturation. Hormones: luteinizing hormone [LH], follicle stimulating hormone [FSH], thyroxine [T4], thyroid stimulating hormone [TSH]. Fecal occult blood, cardiac Troponin I and urinalysis) following up to 4 years of treatment.
    - Abnormal and clinically relevant changes in liver MRI and physical examinations (including nose and throat mucosal exam and Tanner stage and Testicular Volume), weight, vitals, electrocardiogram (ECG), LVEF measured by cardiac MRI (or echocardiogram), bone mineral density by DXA, x-ray (hand and wrist for bone age evaluation) and C-SSRS.

    Efficacy:
    - Mean change from baseline following up to 4 years of treatment in the following functional assessment tests PFTs (to include FVC, FEV1 and PEFR), 4SC, NSAA, PUL, ROM and 6MWD.
    - Mean change from baseline in muscle strength measured by myometry following up to 4 years of treatment.
    E.5.1.1Timepoint(s) of evaluation of this end point
    To evaluate change from baseline of Lean Body Mass (LBM) determined via whole body DXA after up to 4 years of treatment.
    E.5.2Secondary end point(s)
    Efficacy:
    - Mean change from baseline following up to 4 years of treatment in the following functional assessment tests PFTs (to include FVC, FEV1 and PEFR), 4SC, NSAA, PUL, ROM and 6MWD.
    - Mean change from baseline in muscle strength measured by myometry following up to 4 years of treatment.

    PK and Immunogenicity:
    Trough serum PF-06252616 concentrations for all subjects receiving active drug.
    - Incidence of ADA and NAb.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 193
    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 No
    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) No
    E.8.2.2Placebo No
    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 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 EEA8
    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
    Australia
    Bulgaria
    Canada
    Italy
    Japan
    Poland
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    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
    LSLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    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: 105
    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: 53
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 52
    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 No
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Subjects with Duchenne muscular dystrophy who enrolled and complete through Week97 of Study B5161002
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 24
    F.4.2.2In the whole clinical trial 105
    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)
    Depending on when subjects begin the OLE study, their participation may be as long as 4 years, by which time commercial drug supply should be available.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation TREAT-NMD, The University of Newcastle upon Tyne
    G.4.3.4Network Country United Kingdom
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2018-07-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-06-26
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2018-08-16
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