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

NCT03917719 · Status: TERMINATED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 130

Last updated 2020-11-23

No results posted yet for this study

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

Interventions

DRUG

Edasalonexent

100 mg/kg/day

Sponsors & Collaborators

  • Catabasis Pharmaceuticals

    lead INDUSTRY

Principal Investigators

  • Joanne M Donovan, MD, PhD · Catabasis Pharmaceuticals

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Max Age
12 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-03-14
Primary Completion
2020-10-26
Completion
2020-10-26
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Canada
  • Germany
  • Sweden
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03917719 on ClinicalTrials.gov