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

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

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.

Study Overview

• Status: Terminated
• Conditions: Duchenne Muscular Dystrophy
• Intervention / Treatment: Drug: Edasalonexent

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.

• Study Type: Interventional
• Enrollment (Actual): 130
• Phase: Phase 3

Contacts and Locations

Study Locations

• Australia
  • Victoria
    • Parkville, Victoria, Australia, 3052
      • Royal Children's Hospital
• Canada
  • Ontario
    • Ottawa, Ontario, Canada, K1H 8L1
      • Children's Hospital of Eastern Ontario
• Germany
  • Hamburg, Germany, 20246
    • University of Hamburg
  • Munich, Germany, 80337
    • University of Munich
• Sweden
  • Gothenburg, Sweden, 41685
    • Queen Silvia Children's Hospital
• United Kingdom
  • Bristol, United Kingdom, BS2 8AE
    • Bristol Children's Hospital
  • London, United Kingdom, WC1N 3JH
    • Great Ormond Street Hospital (GOSH)
  • Manchester, United Kingdom, M13 9WL
    • Royal Manchester Children's Hospital
• United States
  • California
    • Sacramento, California, United States, 95817
      • UC Davis
  • Georgia
    • Atlanta, Georgia, United States, 30318
      • Rare Disease Research, LLC
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa Children's Hospital
  • Kansas
    • Fairway, Kansas, United States, 66205
      • University of Kansas Medical Center
  • Maryland
    • Baltimore, Maryland, United States, 21205
      • Kennedy Krieger Institute
    • Baltimore, Maryland, United States, 21287
      • Johns Hopkins School of Medicine
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan
  • Nevada
    • Las Vegas, Nevada, United States, 89145
      • Las Vegas Clinic
  • Oregon
    • Portland, Oregon, United States, 97239
      • Shriners Hospital for Children
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Children's Hospital of Philadelphia
  • Tennessee
    • Nashville, Tennessee, United States, 37212
      • Vanderbilt University Medical Center
  • Texas
    • Fort Worth, Texas, United States, 76104
      • Cook Children's Medical Center
    • San Antonio, Texas, United States, 78229
      • University of Texas Health Science Center at San Antonio
  • Utah
    • Salt Lake City, Utah, United States, 84112
      • University of Utah

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 years to 12 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Dose 1; Edasalonexent 100mg/kg/day. Capsules taken by mouth three times per day.	Drug: Edasalonexent; 100 mg/kg/day; Other Names: Edasa; CAT-1004

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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 Outcome Measures

Outcome Measure	Time Frame
Durability of effects of edasalonexent on physical function as measured by the North Star Ambulatory Assessment (NSAA)	104 Weeks
Durability of effects of edasalonexent on physical function as measured by the 10-meter walk/run test	104 Weeks
Durability of effects of edasalonexent on physical function as measured by the time to stand from supine	104 Weeks
Durability of effects of edasalonexent on physical function as measured by the 4-stair climb	104 Weeks

Collaborators and Investigators

• Sponsor: Catabasis Pharmaceuticals
• Investigators: Study Chair: Joanne M Donovan, MD, PhD, Catabasis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (ACTUAL): March 14, 2019
• Primary Completion (ACTUAL): October 26, 2020
• Study Completion (ACTUAL): October 26, 2020

Study Registration Dates

• First Submitted: April 12, 2019
• First Submitted That Met QC Criteria: April 16, 2019
• First Posted (ACTUAL): April 17, 2019

Study Record Updates

• Last Update Posted (ACTUAL): November 23, 2020
• Last Update Submitted That Met QC Criteria: November 19, 2020
• Last Verified: November 1, 2020

More Information

Terms related to this study

• Keywords: Musculoskeletal Diseases; Neuromuscular Diseases; DMD; Duchenne; Muscular Dystrophies; dystrophin; dystrophy
• Additional Relevant MeSH Terms: Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Muscular Disorders, Atrophic; Muscular Dystrophies; Muscular Dystrophy, Duchenne
• Other Study ID Numbers: CAT-1004-302

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No