Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
PolarisDMD
A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy
1 other identifier
interventional
131
8 countries
40
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Oct 2018
40 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
October 2, 2018
CompletedFirst Submitted
Initial submission to the registry
October 8, 2018
CompletedFirst Posted
Study publicly available on registry
October 12, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 22, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
September 22, 2020
CompletedResults Posted
Study results publicly available
June 21, 2022
CompletedJune 21, 2022
June 1, 2022
2 years
October 8, 2018
January 10, 2022
June 16, 2022
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change From Baseline in North Star Ambulatory Assessment (NSAA)
To assess change from baseline in North Star Ambulatory Assessment(NSAA) Total Score at Wk52. NSAA is clinician-reported outcome instrument designed to measure ambulatory function in males with Duchenne muscular dystrophy(DMD). Patients asked to perform 17 different functional activities,including 10MWT,rising from sit to stand,standing on one leg,climbing \& descending a step,stand from supine, lifting the head, standing on heels, \& jumping. Each function activity will be scored as0=(unable to achieve independently),scored as1=(modified method but achieves goal independent of physical assistance from another),or scored as2=(no obvious modification of activity)or "Not Scored". If NSAA test was performed \& any of the individual items are scored as "not scored"(i.e, for reasons unrelated to patients physical capabilities), corresponding total score will be set to missing. Sum of 17 scores will be used to form an ordinal total score(range 0-34).Higher scores imply better functional status
Baseline (Day 1) to Week 52
Secondary Outcomes (4)
Change From Baseline in 10-meter Walk/Run Test
Baseline (Day 1) to Week 52
Change From Baseline in Time to Stand From Supine
Baseline (Day 1) to Week 52
Change From Baseline in 4-stair Climb
Baseline (Day 1) to Week 52
Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Up to Week 52
Study Arms (2)
Dose 1
EXPERIMENTALEdasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day.
Placebo
PLACEBO COMPARATORMatching placebo
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (40)
Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
Children's Hospital of Los Angeles
Los Angeles, California, 90027, United States
UC Davis
Sacramento, California, 95817, United States
Nemours Children's Hospital
Orlando, Florida, 32827, United States
Rare Disease Research, LLC
Atlanta, Georgia, 30318, United States
Rush University Children's Hospital
Chicago, Illinois, 60612, United States
University of Iowa Children's Hospital
Iowa City, Iowa, 52242, United States
University of Kansas Medical Center
Fairway, Kansas, 66205, United States
Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
Johns Hopkins School of Medicine
Baltimore, Maryland, 21287, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
University of Michigan
Ann Arbor, Michigan, 48109, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Las Vegas Clinic
Las Vegas, Nevada, 89145, United States
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States
MetroHealth Medical Center
Cleveland, Ohio, 44109, United States
Shriners Hospitals for Children
Portland, Oregon, 97239, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37212, United States
Cook Children's Medical Center
Fort Worth, Texas, 76104, United States
University of Texas Health Science Center at San Antonio
San Antonio, Texas, 78229, United States
University of Utah
Salt Lake City, Utah, 84112, United States
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23510, United States
Children's Hospital of Richmond at VCU
Richmond, Virginia, 23298, United States
The Children's Hospital at Westmead
Westmead, New South Wales, 2145, Australia
Children's Health Queensland Children's Hospital and Health Service
South Brisbane, Queensland, 4101, Australia
Royal Children's Hospital
Parkville, Victoria, 3052, Australia
Alberta Children's Hospital
Calgary, Alberta, T3B 6A8, Canada
London Health Sciences Centre - Children's Hospital
London, Ontario, N6A 4G5, Canada
Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1, Canada
CHU Sainte-Justine
Montreal, Quebec, H3T 1C5, Canada
University of Hamburg
Hamburg, 20246, Germany
University of Munich
Munich, 80337, Germany
Children's University Hospital
Dublin, 1, Ireland
Hadassah Medical Center
Jerusalem, 9124001, Israel
Queen Silvia Children's Hospital
Gothenburg, 41685, Sweden
Bristol Children's Hospital
Bristol, BS2 8AE, United Kingdom
Evelina Children's Hospital
London, SE1 7EU, United Kingdom
Great Ormond Street Hospital (GOSH)
London, WC1N 3JH, United Kingdom
Royal Manchester Children's Hospital
Manchester, M13 9WL, United Kingdom
Related Publications (1)
Finkel RS, McDonald CM, Lee Sweeney H, Finanger E, Neil Knierbein E, Wagner KR, Mathews KD, Marks W, Statland J, Nance J, McMillan HJ, McCullagh G, Tian C, Ryan MM, O'Rourke D, Muller-Felber W, Tulinius M, Burnette WB, Nguyen CT, Vijayakumar K, Johannsen J, Phan HC, Eagle M, MacDougall J, Mancini M, Donovan JM; (For the PolarisDMD Study Group). A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial. J Neuromuscul Dis. 2021;8(5):769-784. doi: 10.3233/JND-210689.
PMID: 34120912DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Andrew Nichols, PhD - Chief Scientific Officer
- Organization
- Astria Therapeutics, Inc
Study Officials
- STUDY CHAIR
Joanne M Donovan, Chief Medical Officer, MD, PhD
Catabasis Pharmaceuticals
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 8, 2018
First Posted
October 12, 2018
Study Start
October 2, 2018
Primary Completion
September 22, 2020
Study Completion
September 22, 2020
Last Updated
June 21, 2022
Results First Posted
June 21, 2022
Record last verified: 2022-06