A study to compare edasalonexent with placebo in pediatric patients with Duchenne Muscular dystrophy.

• Register: EUCTR
• Last refreshed on: 13 October 2020
• Main ID: EUCTR2018-000464-29-IE
• Date of registration: 02/10/2018
• Prospective Registration: Yes
• Primary sponsor: Catabasis Pharmaceuticals, Inc.
• Public title: A study to compare edasalonexent with placebo in pediatric patients with Duchenne Muscular dystrophy.
• Scientific title: A RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED, GLOBAL PHASE 3 STUDY OF EDASALONEXENT IN PEDIATRIC PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY - Phase 3 Study of Edasalonexent in Duchenne Muscular Dystrophy
• Date of first enrolment: 16/01/2019
• Target sample size: 126
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2018-000464-29
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: yes; Open: no; Single blind: no; Double blind: yes; Parallel group: yes; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: yes; Other: no; Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Australia, Canada, Germany, Ireland, Israel, Sweden, United Kingdom, United States

Contacts

• Name: Leslie Cowen
• Affiliation: Catabasis Pharmaceuticals Inc.

• Name: Leslie Cowen
• Affiliation: Catabasis Pharmaceuticals Inc.

Key inclusion & exclusion 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 <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 willingness to disclose patient's study participation with medical professionals.
Are the trial subjects under 18? yes
Number of subjects for this age range: 126
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range 0
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

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 <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.


Age minimum:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

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

Intervention(s)

Product Name: Edasalonexent
Product Code: CAT-1004
Pharmaceutical Form: Capsule, soft
INN or Proposed INN: Edasalonexent
Current Sponsor code: CAT-1004
Concentration unit: mg milligram(s)
Concentration type: range
Concentration number: 100-250
Pharmaceutical form of the placebo: Capsule, soft
Route of administration of the placebo: Oral use

Primary Outcome(s)

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)

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

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

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

Secondary Outcome(s)

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.

Secondary ID(s)

CAT-1004-301

2018-000464-29-DE

Source(s) of Monetary Support

Catabasis Pharmaceuticals Inc.

Ethics review

Status: Approved
Approval date: 16/01/2019
Contact: