An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
NCT ID: NCT03917719
Last Updated: 2020-11-23
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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TERMINATED
PHASE3
130 participants
INTERVENTIONAL
2019-03-14
2020-10-26
Brief Summary
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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
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Conditions
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Keywords
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Dose 1
Edasalonexent 100mg/kg/day. Capsules taken by mouth three times per day.
Edasalonexent
100 mg/kg/day
Interventions
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Edasalonexent
100 mg/kg/day
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Completion of either 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
Exclusion Criteria
For Siblings of Patients who Completed CAT-1004-201 or CAT-1004-301:
* 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
4 Years
12 Years
MALE
No
Sponsors
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Catabasis Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Joanne M Donovan, MD, PhD
Role: STUDY_CHAIR
Catabasis Pharmaceuticals
Locations
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UC Davis
Sacramento, California, United States
Rare Disease Research, LLC
Atlanta, Georgia, United States
University of Iowa Children's Hospital
Iowa City, Iowa, United States
University of Kansas Medical Center
Fairway, Kansas, United States
Kennedy Krieger Institute
Baltimore, Maryland, United States
Johns Hopkins School of Medicine
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
University of Michigan
Ann Arbor, Michigan, United States
Las Vegas Clinic
Las Vegas, Nevada, United States
Shriners Hospital for Children
Portland, Oregon, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
Cook Children's Medical Center
Fort Worth, Texas, United States
University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States
University of Utah
Salt Lake City, Utah, United States
Royal Children's Hospital
Parkville, Victoria, Australia
Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada
University of Hamburg
Hamburg, , Germany
University of Munich
Munich, , Germany
Queen Silvia Children's Hospital
Gothenburg, , Sweden
Bristol Children's Hospital
Bristol, , United Kingdom
Great Ormond Street Hospital (GOSH)
London, , United Kingdom
Royal Manchester Children's Hospital
Manchester, , United Kingdom
Countries
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Other Identifiers
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CAT-1004-302
Identifier Type: -
Identifier Source: org_study_id