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    Summary
    EudraCT Number:2019-003563-22
    Sponsor's Protocol Code Number:CAT-1004-302
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2020-01-31
    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 number2019-003563-22
    A.3Full title of the trial
    An Open-Label Extension 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 provide Edasalonexent to Pediatric Patients with Duchenne Muscular Dystrophy
    A.4.1Sponsor's protocol code numberCAT-1004-302
    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 pointValerie Fiolkoski
    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 number003034259596
    B.5.5Fax number006172732637
    B.5.6E-mailvfiolkoski@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 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
    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 (DMD) is an inherited (genetic) condition which affects the muscles, causing muscle weakness.
    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 safety and tolerability of long-term treatment of edasalonexent in pediatric patients with Duchenne muscular dystrophy (DMD)
    E.2.2Secondary objectives of the trial
    To assess the durability of effects of edasalonexent as measured by North Star Ambulatory Assessment (NSAA), 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
    For patients from CAT-1004-201 OR CAT-1004-301:
    Inclusion Criteria

    1. Written consent/assent by patient and/or parent or legal guardian as per Regulatory and/or Institutional Review Board
    (IRB)/Independent Ethics Committee (IEC) requirements.
    2. Completion of either CAT-1004-201 or CAT-1004-301.

    For siblings of patients who completed CAT-1004-201 OR CAT-1004-301:
    Inclusion Criteria (siblings)

    A patient must meet all criteria to be eligible for this study
    1. Written consent/assent by patient and/or parent or legal guardian as per Regulatory and/or Institutional Review Board
    (IRB)/Independent Ethics Committee (IEC) requirements.
    2. A sibling of a patient who completed either CAT-1004-201 OR CAT-1004-301.
    3. 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.
    4. Male sex by birth
    5. Age ≥4.0 to <13.0 years (at the time of consent)
    6. Followed by a doctor or medical professional who coordinates DMD care on a regular basis and willingness to
    disclose patient’s study participation with medical professionals.
    A patient must meet all criteria to be eligible for this study
    1. Written consent/assent by patient and/or parent or legal guardian as per Regulatory and/or Institutional Review Board
    (IRB)/Independent Ethics Committee (IEC) requirements.
    2. A sibling of a patient who completed either CAT-1004-201 OR CAT-1004-301.
    3. 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.
    4. Male sex by birth
    5. Age ≥4.0 to <13.0 years (at the time of consent)
    6. Followed by a doctor or medical professional who coordinates DMD care on a regular basis and willingness to
    disclose patient’s study participation with medical professionals.
    E.4Principal exclusion criteria
    For patients from CAT-1004-201 OR CAT-1004-301:

    In the Investigator’s opinion, unwilling or unable for any reason (e.g., medical conditions) to complete all study
    assessments and laboratory tests and comply with scheduled visits, administration of drug, and all other study
    procedures.

    For siblings of patients who completed CAT-1004-201 OR CAT-1004-301:
    A patient who meets any of the following criteria will be excluded from this study
    1. Use of oral corticosteroids at screening; use of inhaled, intranasal and topical corticosteroids is permitted.
    Corticosteroid use should not be discontinued expressly for the trial, so boys in the trial would be those for whom
    corticosteroid use is not yet suitable, deferred by parent or guardian decision, or discontinued because of side effects.
    2. Use of an investigational drug, idebenone, or dystrophin-focused therapy such as ataluren 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.
    In regions were ataluren is approved, ataluren should not be discontinued for the purposes of this study, nor should
    patients be enrolled who would be eligible for ataluren in the future. Exception: Patients who are currently on or plan
    to initiate treatment with approved oligonucleotide exon-skipping therapies, and expected to continue treatment
    throughout the study, will be eligible.
    3. Use of the following within 4 weeks prior to Day 1: immunosuppressive therapy, anticoagulants, cyclosporine,
    dihydroergotamine, ergotamine, fentanyl, alfentanil, pimozide, quinidine, sirolimus, or tacrolimus.
    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. Abnormal gamma-glutamyl transferase (GGT) (>laboratory’s upper limit of normal [ULN]).
    7. Other prior or ongoing medical condition, known hypersensitivity to edasalonexent, salicylates, omega-3 fatty acids,
    excipients, or soy products, physical findings, electrocardiogram (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).
    8. In the Investigator’s opinion, unwilling or unable for any reason (e.g., medical conditions) 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)
    Safety will be evaluated in terms of all treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs), as well
    as physical examination, growth parameters, vital signs, clinical laboratory parameters (including chemistry, hematology), and
    adrenal function (adrenocorticotrophic hormone [ACTH] and cortisol levels).

    E.5.1.1Timepoint(s) of evaluation of this end point
    Treatment Emergent Adverse Events and Serious Adverse Events will be assessed continuously through out the course of the study.
    Physical Examination; Screening visit, Week 52 and Week 104
    Height and Weight; Screening visit, baseline visit, Week 26, Week 52, Week 78, Week 104.
    Vital Signs; Screening visit, Week 26, Week 52, Week 78, Week 104
    Clinical Laboratory parameters; Screening visit, Week 26, Week 52, Week 78, Week 104

    E.5.2Secondary end point(s)
    Durability of Effect:
    The durability of the effect of edasalonexent will be assessed via:
    • Timed function testing (TFT), which includes the 10MWT, 4-stair climb, and stand from supine
    • The North Star Ambulatory Assessment (NSAA)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Timed Function Testing and The North Star Ambulatory Assessment (NSAA) at screening (siblings only), week 52 and week 104.
    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 No
    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 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 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 concerned4
    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
    Canada
    Israel
    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
    Last Patient Last Visit
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    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: 140
    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: 135
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 5
    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 state14
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 140
    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 will return to standard of care
    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 Decision2020-04-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-04-16
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2020-10-27
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