A study to provide Edasalonexent to Pediatric Patients with Duchenne Muscular Dystrophy

Phase 1

• Conditions: Duchenne Muscular Dystrophy; MedDRA version: 20.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2019-003563-22-IE
• Lead Sponsor: Catabasis Pharmaceuticals Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-01-21
• Last Update Posted: 16 November 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 140

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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 140
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified