An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy Clinical Trial

— GalaxyDMD  

Official title:

An Open-Label Extension Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03917719
• Other study ID #: CAT-1004-302
• Status: Terminated
• Phase: Phase 3
• Start date: March 14, 2019
• Est. completion date: October 26, 2020

Study information

• Verified date: November 2020
• Source: Catabasis Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The GalaxyDMD study is a global Phase 3, open-label, treatment extension study to evaluate the safety, tolerability, and durability of effect in long-term dosing of edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD. Patients who completed CAT-1004-201 or CAT-1004-301 or siblings of these boys from 4-12 years of age (up to 13th birthday) will be enrolled.

Edasalonexent is an orally administered small molecule that inhibits NF-kB, which is a key link between loss of dystrophin and disease pathology and plays a fundamental role in the initiation and progression of skeletal and cardiac muscle disease in DMD.

Description:

The study includes a 104-week open-label treatment period with edasalonexent. Patients who completed CAT-1004-201 or CAT-1004-301 and eligible siblings of these boys will be enrolled in this trial.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 130
• Est. completion date: October 26, 2020
• Est. primary completion date: October 26, 2020
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 4 Years to 12 Years

Eligibility

For Patients who Completed CAT-1004-201 or CAT-1004-301:

Inclusion Criteria:

• Written consent/assent by patient and/or legal guardian as per regional and/or Institutional Review Board (IRB)/Independent Ethics Committee (IEC) requirements

• Completion of either CAT-1004-201 or CAT-1004-301

Exclusion Criteria:

• In the Investigator's opinion, unwilling or unable for any reason 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:

Inclusion Criteria:

• Written consent/assent by patient and/or legal guardian as per regional and/or Institutional Review Board (IRB)/Independent Ethics Committee (IEC) requirements

• A sibling of a patient who completed either CAT-1004-201 or CAT-1004-301

• 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

• 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

Exclusion Criteria:

• Use of oral corticosteroids at screening; use of inhaled, intranasal, and topical corticosteroids is permitted

• Use of another investigational drug, idebenone, or dystrophin-focused therapy within 4 weeks. 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

• Use of the following within 4 weeks prior to Day 1: immunosuppressive therapy, anticoagulants, cyclosporine, dihydroergotamine, ergotamine, fentanyl, alfentanil, pimozide, quinidine, sirolimus or tacrolimus

• Use of human growth hormone within 3 months prior to Day 1

• Other prior or ongoing significant medical conditions

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• Edasalonexent
100 mg/kg/day

Locations

Country	Name	City	State
Australia	Royal Children's Hospital	Parkville	Victoria
Canada	Children's Hospital of Eastern Ontario	Ottawa	Ontario
Germany	University of Hamburg	Hamburg
Germany	University of Munich	Munich
Sweden	Queen Silvia Children's Hospital	Gothenburg
United Kingdom	Bristol Children's Hospital	Bristol
United Kingdom	Great Ormond Street Hospital (GOSH)	London
United Kingdom	Royal Manchester Children's Hospital	Manchester
United States	University of Michigan	Ann Arbor	Michigan
United States	Rare Disease Research, LLC	Atlanta	Georgia
United States	Johns Hopkins School of Medicine	Baltimore	Maryland
United States	Kennedy Krieger Institute	Baltimore	Maryland
United States	Boston Children's Hospital	Boston	Massachusetts
United States	University of Kansas Medical Center	Fairway	Kansas
United States	Cook Children's Medical Center	Fort Worth	Texas
United States	University of Iowa Children's Hospital	Iowa City	Iowa
United States	Las Vegas Clinic	Las Vegas	Nevada
United States	Vanderbilt University Medical Center	Nashville	Tennessee
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Shriners Hospital for Children	Portland	Oregon
United States	UC Davis	Sacramento	California
United States	University of Utah	Salt Lake City	Utah
United States	University of Texas Health Science Center at San Antonio	San Antonio	Texas

Sponsors (1)

Lead Sponsor	Collaborator
Catabasis Pharmaceuticals

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and tolerability of long-term treatment with edasalonexent measured by number of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)		104 Weeks
Secondary	Durability of effects of edasalonexent on physical function as measured by the North Star Ambulatory Assessment (NSAA)		104 Weeks
Secondary	Durability of effects of edasalonexent on physical function as measured by the 10-meter walk/run test		104 Weeks
Secondary	Durability of effects of edasalonexent on physical function as measured by the time to stand from supine		104 Weeks
Secondary	Durability of effects of edasalonexent on physical function as measured by the 4-stair climb		104 Weeks