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    Summary
    EudraCT Number:2020-000698-26
    Sponsor's Protocol Code Number:FGCL-3019-093
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2020-10-14
    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 ConcernedUK - MHRA
    A.2EudraCT number2020-000698-26
    A.3Full title of the trial
    A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory Duchenne Muscular Dystrophy (DMD)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Evaluation of Pamrevlumab for the Treatment of Male Patients Affected by Non-ambulatory Duchenne Muscular Dystrophy and just Treated with Corticosteroids
    A.4.1Sponsor's protocol code numberFGCL-3019-093
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04371666
    A.5.4Other Identifiers
    Name:IND (US)Number:126630
    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 SponsorFibroGen, 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 supportFibroGen, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationFibroGen, Inc.
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street Address409 Illinois Street
    B.5.3.2Town/ citySan Francisco
    B.5.3.3Post code94158
    B.5.3.4CountryUnited States
    B.5.6E-mailFG3019_093@Fibrogen.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/3/19/2234
    D.3 Description of the IMP
    D.3.1Product namePamrevlumab
    D.3.2Product code FG-3019
    D.3.4Pharmaceutical form 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 INNPAMREVLUMAB
    D.3.9.1CAS number 946415-13-0
    D.3.9.2Current sponsor codeFG-3019
    D.3.9.4EV Substance CodeSUB198085
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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) 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
    D.8.3Pharmaceutical form of the placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Non-ambulatory Duchenne Muscular Dystrophy
    E.1.1.1Medical condition in easily understood language
    Duchenne Muscle 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
    Evaluation of the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids in subjects with Duchenne muscular dystrophy (non-ambulatory, age 12 years and older).
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Double-blind phase:
    Age, Consent, and Contraception
    1. Males at least 12 years of age, non-ambulatory at screening initiation;
    2. Written consent by patient and/or legal guardian as per regional/ country and/or IRB/IEC requirements;
    3. Male subjects with partners of childbearing potential must use contraception during the conduct of the study, and for 3 months after the last dose of study drug.

    DMD Diagnosis:
    4. Medical history includes diagnosis of DMD and confirmed Duchenne mutation using a validated genetic test.

    Performance criteria:
    5. Brooke Score for Arms and Shoulders ≤5:
    6. Able to undergo MRI test for the upper arm extremities (Biceps Brachii muscle) andcardiac muscle;
    7. Able to perform spirometry.

    Pulmonary and Cardiac criteria:
    8. Reproducible (+/- 5% difference between screening and baseline) percent predicted FVC between 45 and 85, inclusive;
    9. Left ventricular ejection fraction ≥50% as determined by cardiac MRI at screening or within 3 months prior to randomization (Day 0);
    10. Prior diagnosis of cardiomyopathy, subjects must be on a stable regimen dose for cardiomyopathy/ heart failure medications (e.g., angiotensin converting enzyme inhibitors, aldosterone receptors blockers, angiotensin-receptor blockers, and beta-blockers) for at least 1 month prior to screening;
    11. On a stable dose of systemic corticosteroids for a minimum of 6 months, with nos ubstantial change in dosage for a minimum of 3 months (except for adjustments for changes in body weight) prior to screening. Corticosteroid dosage should be in compliance with the DMD Care Considerations Working Group recommendations (e.g. prednisone or prednisolone 0.75 mg/kg per day or deflazacort 0.9 mg/kg per day) orstable dose. A reasonable expectation is that dosage and dosing regimen would not change significantly for the duration of the study.

    Vaccination:
    12. Received pneumococcal vaccine (PPSV23) (or any other pneumococcal polysaccharidevaccine as per national recommendations) and is receiving annual influenza vaccinations.

    Laboratory criteria:
    13. Adequate renal function: cystatin C ≤1.4 mg/L;
    14. Adequate hematology and electrolytes parameters:
    a. Platelets >100,000/mcL
    b. Hemoglobin >12 g/dL
    c. Absolute neutrophil count >1500 /μL
    d. Serum calcium (Ca), potassium (K), sodium (Na), magnesium (Mg) and phosphorus(P) levels are within a clinically accepted range;
    15. Adequate hepatic function:
    a. No history or evidence of liver disease
    b. Gamma glutamyl transferase (GGT) ≤3x upper limit of normal (ULN)
    c. Total bilirubin ≤1.5xULN.

    Open-label extension phase:
    All patients must have completed treatment through the primary endpoint and completed the Week 52 visit on the double-blind phase. The study investigator must consider the subject medically stable for continued treatment. Written consent/assent by the patients and/or their legal guardian must be obtained prior to the patient’s participation in the open-label extension phase.
    E.4Principal exclusion criteria
    Both phases:
    General criteria:
    1. Previous exposure to pamrevlumab;
    2. BMI ≥40 kg/m2 or weight >117 kg;
    3. History of allergic or anaphylactic reaction to human, humanized, chimeric or murine monoclonal antibodies;
    4. Exposure to any investigational drug (for DMD or not), in the 30 days prior to screening initiation or use of approved DMD therapies (e.g., eteplirsen, ataluren) within 5 half-lives of screening, whichever is longer, with the exception of the systemic corticosteroids,including deflazacort.

    Cardiac and Pulmonary assessment:
    5.Severe uncontrolled heart failure (NYHA Classes III-IV), including any of the following:
    a. Need for intravenous diuretics or inotropic support within 8 weeks prior to screening
    b. Hospitalization for a heart failure exacerbation or arrhythmia within 8 weeks prior to screening;
    6. Arrhythmia requiring anti-arrhythmic therapy;
    7. Requires ≥16 hours continuous ventilation;
    8. Hospitalization due to respiratory failure within the 8 weeks prior to screening;
    9. Poorly controlled asthma or underlying lung disease such as bronchitis, bronchiectasis, emphysema, recurrent pneumonia that in the opinion of the investigator might impact respiratory function.

    Clinical judgments:
    10.The Investigator judges that the subject will be unable to fully participate in the study and complete it for any reason, including inability to comply with study procedures and treatment, or any other relevant medical or psychiatric conditions which could confound efficacy assessment and/or safety assessment.
    E.5 End points
    E.5.1Primary end point(s)
    Double-blind phase:
    Function assessment:
    • Change in the total score of Performance of Upper Limb Version 2.0

    Open-label extension phase:
    Function assessment:
    • Annual mean change in the total score of Performance of Upper Limb Version 2.0.

    Pulmonary assessment:
    • Annual mean change in percent predicted forced vital capacity, assessed by spirometry;
    • Annual mean change in percent predicted peak expiratory flow, assessed by spirometry.

    Performance assessment:
    • Annual mean change in the Grip strength of the hands extremities, assessed by Hand Held Myometry.

    Cardiac assessment:
    • Annual mean change in Left Ventricular Ejection Fraction percentage (LVEF %),assessed by MRI.

    Fibrosis/MRI assessments:
    • Annual mean change in fibrosis score of the biceps brachii, assessed by MRI;
    • Annual mean change in Cardiac fibrosis score, assessed by Late Gadolinium Enhancement;
    • Annual mean change in Myocardial Circumferential Strain percentage, assessed by cardiac MRI.

    Both phases:
    Safety assessments
    • Treatment-emergent adverse events (TEAEs), treatment-emergent serious adverse events (TESAEs), clinically significant laboratory test abnormalities, and discontinuation of treatment due to TEAEs serve as the safety assessments for the open-label extension phase.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Double-blind phase:
    Baseline
    Week 52

    Open-label extension phase:
    Week 52
    Week 104
    Week 156
    Week 208

    Safety:
    From screening throughout week 208
    E.5.2Secondary end point(s)
    Double-blind phase:
    Pulmonary assessment:
    • Change in percent predicted forced vital capacity,assessed by spirometry;
    • Change in percent predicted peak expiratory flow,assessed by spirometry.

    Performance assessment:
    • Change in the Grip strength of the hands from baseline to Week 52, assessed by Hand Held Myometry.

    Cardiac assessment:
    • Change in Left Ventricular Ejection Fraction percentage (LVEF %), assessed by MRI.

    Open-label extension phase:
    Not applicable
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline
    Week 52
    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 No
    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) 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 Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Open-label extension
    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 trial2
    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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA29
    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
    Belgium
    Canada
    Czech Republic
    France
    Israel
    Italy
    Netherlands
    Slovakia
    Spain
    Switzerland
    Turkey
    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
    LVLS
    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 months7
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months5
    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: 30
    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) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 30
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 55
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 No
    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 No
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 34
    F.4.2.2In the whole clinical trial 90
    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)
    None
    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 Decision2020-12-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-12-16
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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