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

NCT03917719 | Terminated Phase 3 DMC FDA Drug

• 1 other identifier: CAT-1004-302
• Study Type: interventional
• Target: 130
• Locations: 6 countries
• Sites: 23

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.

Conditions

Duchenne Muscular Dystrophy

Keywords

Muscular Dystrophies; Musculoskeletal Diseases; Neuromuscular Diseases; DMD; dystrophin; dystrophy; Duchenne

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• 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

Study Arms (1)

Dose 1

EXPERIMENTAL

Edasalonexent 100mg/kg/day. Capsules taken by mouth three times per day.

Drug: Edasalonexent

Interventions

Edasalonexent DRUG

100 mg/kg/day

Also known as: Edasa, CAT-1004

Dose 1

Eligibility Criteria

• Age: 4 Years - 12 Years
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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:
• 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
• 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

Sponsors & Collaborators

• Catabasis Pharmaceuticals: lead

Study Sites (23)

UC Davis

Sacramento, California, 95817, United States

Location

Rare Disease Research, LLC

Atlanta, Georgia, 30318, United States

Location

University of Iowa Children's Hospital

Iowa City, Iowa, 52242, United States

Location

University of Kansas Medical Center

Fairway, Kansas, 66205, United States

Location

Kennedy Krieger Institute

Baltimore, Maryland, 21205, United States

Location

Johns Hopkins School of Medicine

Baltimore, Maryland, 21287, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Las Vegas Clinic

Las Vegas, Nevada, 89145, United States

Location

Shriners Hospital for Children

Portland, Oregon, 97239, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37212, United States

Location

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

Location

University of Texas Health Science Center at San Antonio

San Antonio, Texas, 78229, United States

Location

University of Utah

Salt Lake City, Utah, 84112, United States

Location

Royal Children's Hospital

Parkville, Victoria, 3052, Australia

Location

Children's Hospital of Eastern Ontario

Ottawa, Ontario, K1H 8L1, Canada

Location

University of Hamburg

Hamburg, 20246, Germany

Location

University of Munich

Munich, 80337, Germany

Location

Queen Silvia Children's Hospital

Gothenburg, 41685, Sweden

Location

Bristol Children's Hospital

Bristol, BS2 8AE, United Kingdom

Location

Great Ormond Street Hospital (GOSH)

London, WC1N 3JH, United Kingdom

Location

Royal Manchester Children's Hospital

Manchester, M13 9WL, United Kingdom

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Muscular Dystrophies; Musculoskeletal Diseases; Neuromuscular Diseases

Interventions

edasalonexent

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Joanne M Donovan, MD, PhD

  Catabasis Pharmaceuticals

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 12, 2019
• First Posted: April 17, 2019
• Study Start: March 14, 2019
• Primary Completion: October 26, 2020
• Study Completion: October 26, 2020
• Last Updated: November 23, 2020

Record last verified: 2020-11

Locations

SE (1); GB (3); CA (1); US (15); DE (2); AU (1)