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: 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 =65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the safety and tolerability of long-term treatment of edasalonexent in pediatric patients with Duchenne muscular dystrophy (DMD);Secondary Objective: 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;Primary 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). ;Timepoint(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 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary 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);Timepoint(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. | — |
Countries
Australia, Canada, Germany, Ireland, Sweden, United Kingdom, United States
Contacts
Public ContactValerie Fiolkoski
Catabasis Pharmaceuticals Inc.
Outcome results
None listed