Duchenne Muscular Dystrophy MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
Catabasis Pharmaceuticals, Inc.
Eligibility
Sex/Gender
Male
Inclusion criteria
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 =65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
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 1; 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 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 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);Secondary Objective: 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;Primary 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;Timepoint(s) of evaluation of this end point: NSAA is assessed at the following timepoints: Screening Baseline Week 13 Week 26 Week 39 Week 52 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary 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;Timepoint(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. | — |
Countries
Australia, Canada, Germany, Ireland, Israel, Sweden, United Kingdom, United States
Contacts
Public ContactLeslie Cowen
Catabasis Pharmaceuticals Inc.
Outcome results
None listed