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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03703882




Registration number
NCT03703882
Ethics application status
Date submitted
8/10/2018
Date registered
12/10/2018
Date last updated
21/06/2022

Titles & IDs
Public title
Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
Scientific title
A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy
Secondary ID [1] 0 0
CAT-1004-301
Universal Trial Number (UTN)
Trial acronym
PolarisDMD
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Muscular Dystrophy, Duchenne 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
Treatment: Drugs - Placebo

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

Placebo comparator: Placebo - Matching placebo


Treatment: Drugs: Edasalonexent
100 mg/kg/day

Treatment: Drugs: Placebo
Placebo
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in North Star Ambulatory Assessment (NSAA)
Timepoint [1] 0 0
Baseline (Day 1) to Week 52
Secondary outcome [1] 0 0
Change From Baseline in 10-meter Walk/Run Test
Timepoint [1] 0 0
Baseline (Day 1) to Week 52
Secondary outcome [2] 0 0
Change From Baseline in Time to Stand From Supine
Timepoint [2] 0 0
Baseline (Day 1) to Week 52
Secondary outcome [3] 0 0
Change From Baseline in 4-stair Climb
Timepoint [3] 0 0
Baseline (Day 1) to Week 52
Secondary outcome [4] 0 0
Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Timepoint [4] 0 0
Up to Week 52

Eligibility
Key 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
* 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
* Able to perform stand from supine without assistance in = 10 seconds
* Able to perform the 10MWT and 4-stair climb
* 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
Minimum age
4 Years
Maximum age
7 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
* Use of corticosteroids within 24 weeks prior to Day 1; 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 have received at least 24 weeks of a stable dose of eteplirsen prior to Day 1, and expected to continue treatment, will be eligible
* Use of the following within 4 weeks prior to Day 1: immunosuppressive therapy, warfarin, phenytoin, S mephenytoin, cyclosporine, dihydroergotamine, ergotamine, fentanyl, alfentanil, pimozide, quinidine, sirolimus, tacrolimus, or paclitaxel
* 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
Randomised controlled trial
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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
2/10/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
22/09/2020
Sample size
Target
Accrual to date
Final
131
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
The Children's Hospital at Westmead - Westmead
Recruitment hospital [2] 0 0
Children's Health Queensland Children's Hospital and Health Service - South Brisbane
Recruitment hospital [3] 0 0
Royal Children's Hospital - Parkville
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
4101 - South Brisbane
Recruitment postcode(s) [3] 0 0
3052 - Parkville
Recruitment outside Australia
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United States of America
State/province [1] 0 0
Arkansas
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United States of America
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California
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Florida
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Georgia
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Illinois
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Iowa
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Kansas
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United States of America
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Maryland
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Massachusetts
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Michigan
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Minnesota
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Nevada
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Ohio
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Oregon
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Pennsylvania
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Tennessee
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Texas
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Utah
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United States of America
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Virginia
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Canada
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Alberta
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Canada
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Ontario
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Canada
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Quebec
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Germany
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Hamburg
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Germany
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Munich
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Ireland
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Dublin
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Israel
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Jerusalem
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Sweden
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Gothenburg
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United Kingdom
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Bristol
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London
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United Kingdom
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Manchester

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Joanne M Donovan, Chief Medical Officer, 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

No information has been provided regarding IPD availability


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT03703882