Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (PolarisDMD)
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|ClinicalTrials.gov Identifier: NCT03703882|
Recruitment Status : Active, not recruiting
First Posted : October 12, 2018
Last Update Posted : October 14, 2019
The PolarisDMD study is a Phase 3, global study to evaluate the efficacy and safety of edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD. Male patients from 4-7 years of age (up to 8th birthday) will be enrolled.
Edasalonexent is an orally administered small molecule that inhibits NF-kB, which is the 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.
|Condition or disease||Intervention/treatment||Phase|
|Muscular Dystrophy, Duchenne||Drug: Edasalonexent Drug: Placebo||Phase 3|
The study includes a 52-week, randomized, double-blind, placebo-controlled period, followed by a 2-week follow- up. Approximately 125 boys with DMD will be enrolled in this trial, with 2 boys receiving edasalonexent for every 1 boy receiving placebo.
Following completion of the treatment period, patients may elect to continue in a separate open-label extension study.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||131 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|Official Title:||A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy|
|Actual Study Start Date :||October 2, 2018|
|Estimated Primary Completion Date :||June 2020|
|Estimated Study Completion Date :||June 2020|
Experimental: Dose 1
Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day.
Placebo Comparator: Placebo
- Change from baseline in North Star Ambulatory Assessment (NSAA) [ Time Frame: 52 Weeks ]
- Safety and tolerability measured by number of treatment- emergent adverse events (TEAEs) and serious adverse events (SAEs) [ Time Frame: 52 Weeks ]
- Change from baseline in 10-meter walk/run test [ Time Frame: 52 Weeks ]
- Change from baseline in time to stand from supine [ Time Frame: 52 Weeks ]
- Change from baseline in 4-stair climb [ Time Frame: 52 Weeks ]
- Change from baseline in muscle strength testing assessed by knee extension and elbow flexion [ Time Frame: 52 Weeks ]
- Change from baseline in the Performance of Upper Limb (PUL) Scale to assess upper limb function [ Time Frame: 52 Weeks ]
- Change from baseline in parent/proxy reported physical functioning/quality of life assessed by the Pediatric Outcome Data Collection Instrument (PODCI) Questionnaire [ Time Frame: 52 Weeks ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03703882
|Study Chair:||Joanne M Donovan, Chief Medical Officer, MD, PhD||Catabasis Pharmaceuticals|