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

• Register: EUCTR
• Last refreshed on: 16 November 2020
• Main ID: EUCTR2019-003563-22-IE
• Date of registration: 21/01/2020
• Prospective Registration: Yes
• Primary sponsor: Catabasis Pharmaceuticals Inc.
• Public title: A study to provide Edasalonexent to Pediatric Patients with Duchenne Muscular Dystrophy
• Scientific title: An Open-Label Extension Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy
• Date of first enrolment: 23/06/2020
• Target sample size: 140
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2019-003563-22
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
• 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, Sweden, United Kingdom, United States

Contacts

• Name: Valerie Fiolkoski
• Affiliation: Catabasis Pharmaceuticals Inc.

• Name: Valerie Fiolkoski
• Affiliation: Catabasis Pharmaceuticals Inc.

Key inclusion & exclusion 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 <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.


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

Health Condition(s) or Problem(s) studied

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

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

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: equal
Concentration number: 250-

Primary Outcome(s)

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

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

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).

Secondary Outcome(s)

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.

Secondary ID(s)

CAT-1004-302

2019-003563-22-GB

Source(s) of Monetary Support

Catabasis Pharmaceuticals Inc.

Ethics review

Status: Approved
Approval date: 23/06/2020
Contact: