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    Summary
    EudraCT Number:2018-000464-29
    Sponsor's Protocol Code Number:CAT-1004-301
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-09-24
    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 ConcernedGermany - BfArM
    A.2EudraCT number2018-000464-29
    A.3Full title of the trial
    A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, GLOBAL PHASE 3 STUDY OF EDASALONEXENT IN PEDIATRIC 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 compare edasalonexent with placebo in patients with Duchenne Muscular dystrophy
    A.3.2Name or abbreviated title of the trial where available
    POLARIS DMD
    A.4.1Sponsor's protocol code numberCAT-1004-301
    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 SponsorCatabasis Pharmaceuticals, 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 supportCatabasis Pharmaceuticals Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCatabasis Pharmaceuticals Inc.
    B.5.2Functional name of contact pointJoe Johnston
    B.5.3 Address:
    B.5.3.1Street Address100 High Street, 28th Floor
    B.5.3.2Town/ cityBoston
    B.5.3.3Post codeMA 02110
    B.5.3.4CountryUnited States
    B.5.4Telephone number0016174752428
    B.5.5Fax number0016172732637
    B.5.6E-mailjjohnston@catabasis.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/3/15/1560
    D.3 Description of the IMP
    D.3.1Product nameEdasalonexent
    D.3.2Product code CAT-1004
    D.3.4Pharmaceutical form Capsule, soft
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNEDASALONEXENT
    D.3.9.2Current sponsor codeCAT-1004
    D.3.9.4EV Substance CodeSUB193884
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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 No
    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/3/15/1560
    D.3 Description of the IMP
    D.3.1Product nameEdasalonexent
    D.3.2Product code CAT-1004
    D.3.4Pharmaceutical form Capsule, soft
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNEDASALONEXENT
    D.3.9.2Current sponsor codeCAT-1004
    D.3.9.4EV Substance CodeSUB193884
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    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 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 placeboCapsule, soft
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 2
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule, soft
    D.8.4Route of administration of the placeboOral use
    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 muscular dystrophy
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    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 assess the efficacy of edasalonexent as measured by change from Baseline (CFB) on North Star Ambulatory
    Assessment (NSAA) Total Score in pediatric patients with Duchenne muscular dystrophy (DMD)
    E.2.2Secondary objectives of the trial
    To assess the safety and tolerability of edasalonexent in pediatric patients with DMD

    To assess the effects of edasalonexent on physical function as measured by the 10-meter walk/run test (10MWT), time
    to stand from supine, and the 4-stair climb in pediatric patients with DMD
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    A patient must meet all the following criteria to be eligible for this study.
    1. Written consent/assent by patient and/or legal guardian as per regional and/or Institutional Review Board
    (IRB)/Independent Ethics Committee (IEC) requirements
    2. Diagnosis of DMD based on a clinical phenotype with increased serum creatine kinase (CK) and documentation of
    mutation(s) in the dystrophin gene known to be associated with a DMD phenotype
    3. Male sex by birth
    4. Age ≥4.0 to <8.0 years (at the time of consent)
    5. Able to perform stand from supine without assistance in ≤ 10 seconds
    6. Able to perform the 10MWT and 4-stair climb
    7. Able to swallow placebo capsules at the Screening Visit
    8. Followed by a doctor or medical professional who coordinates Duchenne care on a regular basis and willingnessto disclose patient's study participation with medical professionals.
    E.4Principal exclusion criteria
    A patient who meets any of the following criteria will be excluded from this study.
    1. Use of corticosteroids within 24 weeks prior to Day 1Íž use of inhaled, intranasal, and topical corticosteroids is
    permitted
    2. Use of an investigational drug, idebenone, or dystrophin-focused therapy within 4 weeks or a period of 5 half-lives duration prior to Day 1 (whichever is longer) or ongoing participation in any other therapeutic clinical trial. Exception: Patients who have received at least 24 weeks of a stable dose of eteplirsen prior to Day 1, and expected to continue treatment, will be eligible.
    3. Use of the following within 4 weeks prior to Day 1: immunosuppressive therapy, warfarin, phenytoin, S-mephenytoin,
    cyclosporine, dihydroergotamine, ergotamine, fentanyl, alfentanil, pimozide, quinidine, sirolimus, tacrolimus, or
    paclitaxel
    4. Use of human growth hormone within 3 months prior to Day 1
    5. Documented positive hepatitis B surface antigen, hepatitis C antibody, or human immunodeficiency virus (HIV) or a
    known risk factor for hepatitis such as a blood transfusion within 12 weeks prior to Day 1
    6. Hemoglobin <10.5 g/dL
    7. Abnormal gamma-glutamyl transferase (GGT) (>laboratory’s upper limit of normal [ULN])
    8. Other prior or ongoing medical condition, known hypersensitivity to omega-3 fatty acids, physical findings, ECG
    findings, or laboratory abnormality (including but not limited to renal insufficiency or impaired hepatic function) that, in the Investigator’s opinion, could adversely affect the safety of the patient, make it unlikely that the course of Treatment or follow-up would be completed, or impair the assessment of study results (e.g., a gastrointestinal condition that would impair fat absorption)
    9. In the Investigator’s opinion, unwilling or unable for any reason (e.g., attentional or behavioral issues) to complete all study assessments and laboratory tests and comply with scheduled visits, administration of drug, and all other study procedures.
    E.5 End points
    E.5.1Primary end point(s)
    To assess the efficacy of edasalonexent as measured by change from Baseline (CFB) on
    North Star Ambulatory Assessment (NSAA) Total Score in pediatric patients with DMD
    E.5.1.1Timepoint(s) of evaluation of this end point
    NSAA is assessed at the following timepoints:
    Screening
    Baseline
    Week 13
    Week 26
    Week 39
    Week 52
    E.5.2Secondary end point(s)
    To assess the safety and tolerability of edasalonexent in pediatric patients with DMD
    To assess the effects of edasalonexent on physical function as measured by the 10 Meter Walking Test,time to stand
    from supine, and the 4-stair climb in pediatric patients with DMD
    E.5.2.1Timepoint(s) of evaluation of this end point
    Safety (in the form of AEs) will be assessed continuously throughout the study.

    The 10Meter Walking Test, time to stand from supine and the 4 stair climb will be assessed at Screening, Baseline, Week 13, Week 26, Week 39, 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 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 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 No
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA10
    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
    Canada
    Germany
    Ireland
    Israel
    Sweden
    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 years1
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months8
    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: 126
    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: 126
    F.1.1.6Adolescents (12-17 years) No
    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 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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 31
    F.4.2.2In the whole clinical trial 126
    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)
    Patients may elect to continue in the open-label extension study, pending approval by appropriate ethics committees
    and regulatory authorities
    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 Decision2019-01-31
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-01-31
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-09-22
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