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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03917719




Registration number
NCT03917719
Ethics application status
Date submitted
12/04/2019
Date registered
17/04/2019
Date last updated
23/11/2020

Titles & IDs
Public title
An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
Scientific title
An Open-Label Extension Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy
Secondary ID [1] 0 0
CAT-1004-302
Universal Trial Number (UTN)
Trial acronym
GalaxyDMD
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Duchenne Muscular Dystrophy 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Edasalonexent

Experimental: Dose 1 - Edasalonexent 100mg/kg/day. Capsules taken by mouth three times per day.


Treatment: Drugs: Edasalonexent
100 mg/kg/day
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety and tolerability of long-term treatment with edasalonexent measured by number of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Timepoint [1] 0 0
104 Weeks
Secondary outcome [1] 0 0
Durability of effects of edasalonexent on physical function as measured by the North Star Ambulatory Assessment (NSAA)
Timepoint [1] 0 0
104 Weeks
Secondary outcome [2] 0 0
Durability of effects of edasalonexent on physical function as measured by the 10-meter walk/run test
Timepoint [2] 0 0
104 Weeks
Secondary outcome [3] 0 0
Durability of effects of edasalonexent on physical function as measured by the time to stand from supine
Timepoint [3] 0 0
104 Weeks
Secondary outcome [4] 0 0
Durability of effects of edasalonexent on physical function as measured by the 4-stair climb
Timepoint [4] 0 0
104 Weeks

Eligibility
Key inclusion criteria
For 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
* Completion of either CAT-1004-201 or CAT-1004-301
Minimum age
4 Years
Maximum age
12 Years
Sex
Males
Can healthy volunteers participate?
No
Key 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



* 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 design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
14/03/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
26/10/2020
Sample size
Target
Accrual to date
Final
130
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Royal Children's Hospital - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Georgia
Country [3] 0 0
United States of America
State/province [3] 0 0
Iowa
Country [4] 0 0
United States of America
State/province [4] 0 0
Kansas
Country [5] 0 0
United States of America
State/province [5] 0 0
Maryland
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Michigan
Country [8] 0 0
United States of America
State/province [8] 0 0
Nevada
Country [9] 0 0
United States of America
State/province [9] 0 0
Oregon
Country [10] 0 0
United States of America
State/province [10] 0 0
Pennsylvania
Country [11] 0 0
United States of America
State/province [11] 0 0
Tennessee
Country [12] 0 0
United States of America
State/province [12] 0 0
Texas
Country [13] 0 0
United States of America
State/province [13] 0 0
Utah
Country [14] 0 0
Canada
State/province [14] 0 0
Ontario
Country [15] 0 0
Germany
State/province [15] 0 0
Hamburg
Country [16] 0 0
Germany
State/province [16] 0 0
Munich
Country [17] 0 0
Sweden
State/province [17] 0 0
Gothenburg
Country [18] 0 0
United Kingdom
State/province [18] 0 0
Bristol
Country [19] 0 0
United Kingdom
State/province [19] 0 0
London
Country [20] 0 0
United Kingdom
State/province [20] 0 0
Manchester

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Catabasis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT03917719
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Joanne M Donovan, MD, PhD
Address 0 0
Catabasis Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT03917719