Clinical trial NCT03917719: An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
Official title: An Open-Label Extension Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Duchenne Muscular Dystrophy
Keywords: Muscular Dystrophies
Musculoskeletal Diseases
Neuromuscular Diseases
DMD
dystrophin
dystrophy
Duchenne
Lead sponsor: Catabasis Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2019-04-17
Enrollment: 130
Sex: MALE
Minimum age (years): 4
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: Safety and tolerability of long-term treatment with edasalonexent measured by number of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
timeFrame: 104 Weeks
Secondary outcomes:
measure: Durability of effects of edasalonexent on physical function as measured by the North Star Ambulatory Assessment (NSAA)
timeFrame: 104 Weeks
measure: Durability of effects of edasalonexent on physical function as measured by the 10-meter walk/run test
timeFrame: 104 Weeks
measure: Durability of effects of edasalonexent on physical function as measured by the time to stand from supine
timeFrame: 104 Weeks
measure: Durability of effects of edasalonexent on physical function as measured by the 4-stair climb
timeFrame: 104 Weeks
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: UC Davis
state: California
city: Atlanta
country: United States
facility: Rare Disease Research, LLC
state: Georgia
city: Iowa City
country: United States
facility: University of Iowa Children's Hospital
state: Iowa
city: Fairway
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Baltimore
country: United States
facility: Kennedy Krieger Institute
state: Maryland
city: Baltimore
country: United States
facility: Johns Hopkins School of Medicine
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Las Vegas
country: United States
facility: Las Vegas Clinic
state: Nevada
city: Portland
country: United States
facility: Shriners Hospital for Children
state: Oregon
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Fort Worth
country: United States
facility: Cook Children's Medical Center
state: Texas
city: San Antonio
country: United States
facility: University of Texas Health Science Center at San Antonio
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Parkville
country: Australia
facility: Royal Children's Hospital
state: Victoria
city: Ottawa
country: Canada
facility: Children's Hospital of Eastern Ontario
state: Ontario
city: Hamburg
country: Germany
facility: University of Hamburg
city: Munich
country: Germany
facility: University of Munich
city: Gothenburg
country: Sweden
facility: Queen Silvia Children's Hospital
city: Bristol
country: United Kingdom
facility: Bristol Children's Hospital
city: London
country: United Kingdom
facility: Great Ormond Street Hospital (GOSH)
city: Manchester
country: United Kingdom
facility: Royal Manchester Children's Hospital
Study officials:
affiliation: Catabasis Pharmaceuticals
name: Joanne M Donovan, MD, PhD
role: STUDY_CHAIR
References: