Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (PolarisDMD)
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT03703882 |
Recruitment Status :
Completed
First Posted : October 12, 2018
Last Update Posted : November 20, 2020
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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 |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Triple (Participant, Care Provider, Investigator) |
Primary Purpose: | Treatment |
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 |
Actual Primary Completion Date : | September 22, 2020 |
Actual Study Completion Date : | September 22, 2020 |

Arm | Intervention/treatment |
---|---|
Experimental: Dose 1
Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day.
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Drug: Edasalonexent
100 mg/kg/day
Other Names:
|
Placebo Comparator: Placebo
Matching placebo
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Drug: Placebo
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 ]

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 4 Years to 7 Years (Child) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
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
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

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

United States, Arkansas | |
Arkansas Children's Hospital | |
Little Rock, Arkansas, United States, 72202 | |
United States, California | |
Children's Hospital of Los Angeles | |
Los Angeles, California, United States, 90027 | |
UC Davis | |
Sacramento, California, United States, 95817 | |
United States, Florida | |
Nemours Children's Hospital | |
Orlando, Florida, United States, 32827 | |
United States, Georgia | |
Rare Disease Research, LLC | |
Atlanta, Georgia, United States, 30318 | |
United States, Illinois | |
Rush University Children's Hospital | |
Chicago, Illinois, United States, 60612 | |
United States, Iowa | |
University of Iowa Children's Hospital | |
Iowa City, Iowa, United States, 52242 | |
United States, Kansas | |
University of Kansas Medical Center | |
Fairway, Kansas, United States, 66205 | |
United States, Maryland | |
Kennedy Krieger Institute | |
Baltimore, Maryland, United States, 21205 | |
Johns Hopkins School of Medicine | |
Baltimore, Maryland, United States, 21287 | |
United States, Massachusetts | |
Boston Children's Hospital | |
Boston, Massachusetts, United States, 02115 | |
United States, Michigan | |
University of Michigan | |
Ann Arbor, Michigan, United States, 48109 | |
United States, Minnesota | |
University of Minnesota | |
Minneapolis, Minnesota, United States, 55455 | |
United States, Nevada | |
Las Vegas Clinic | |
Las Vegas, Nevada, United States, 89145 | |
United States, Ohio | |
Cincinnati Children's Hospital | |
Cincinnati, Ohio, United States, 45229 | |
MetroHealth Medical Center | |
Cleveland, Ohio, United States, 44109 | |
United States, Oregon | |
Shriners Hospitals for Children | |
Portland, Oregon, United States, 97239 | |
United States, Pennsylvania | |
Children's Hospital of Philadelphia | |
Philadelphia, Pennsylvania, United States, 19104 | |
United States, Tennessee | |
Vanderbilt University Medical Center | |
Nashville, Tennessee, United States, 37212 | |
United States, Texas | |
Cook Children's Medical Center | |
Fort Worth, Texas, United States, 76104 | |
University of Texas Health Science Center at San Antonio | |
San Antonio, Texas, United States, 78229 | |
United States, Utah | |
University of Utah | |
Salt Lake City, Utah, United States, 84112 | |
United States, Virginia | |
Children's Hospital of the King's Daughters | |
Norfolk, Virginia, United States, 23510 | |
Children's Hospital of Richmond at VCU | |
Richmond, Virginia, United States, 23298 | |
Australia, New South Wales | |
The Children's Hospital at Westmead | |
Westmead, New South Wales, Australia, 2145 | |
Australia, Queensland | |
Children's Health Queensland Children's Hospital and Health Service | |
South Brisbane, Queensland, Australia, 4101 | |
Australia, Victoria | |
Royal Children's Hospital | |
Parkville, Victoria, Australia, 3052 | |
Canada, Alberta | |
Alberta Children's Hospital | |
Calgary, Alberta, Canada, T3B 6A8 | |
Canada, Ontario | |
London Health Sciences Centre - Children's Hospital | |
London, Ontario, Canada, N6A 4G5 | |
Children's Hospital of Eastern Ontario | |
Ottawa, Ontario, Canada, K1H 8L1 | |
Canada, Quebec | |
CHU Sainte-Justine | |
Montréal, Quebec, Canada, H3T 1C5 | |
Germany | |
University of Hamburg | |
Hamburg, Germany, 20246 | |
University of Munich | |
Munich, Germany, 80337 | |
Ireland | |
Children's University Hospital | |
Dublin, Ireland, 1 | |
Israel | |
Hadassah Medical Center | |
Jerusalem, Israel, 9124001 | |
Sweden | |
Queen Silvia Children's Hospital | |
Gothenburg, Sweden, 41685 | |
United Kingdom | |
Bristol Children's Hospital | |
Bristol, United Kingdom, BS2 8AE | |
Evelina Children's Hospital | |
London, United Kingdom, SE1 7EU | |
Great Ormond Street Hospital (GOSH) | |
London, United Kingdom, WC1N 3JH | |
Royal Manchester Children's Hospital | |
Manchester, United Kingdom, M13 9WL |
Study Chair: | Joanne M Donovan, Chief Medical Officer, MD, PhD | Catabasis Pharmaceuticals |
Responsible Party: | Catabasis Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT03703882 |
Other Study ID Numbers: |
CAT-1004-301 |
First Posted: | October 12, 2018 Key Record Dates |
Last Update Posted: | November 20, 2020 |
Last Verified: | November 2020 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Muscular Dystrophies Musculoskeletal Diseases Neuromuscular Diseases Duchenne muscular dystrophy |
DMD dystrophin dystrophy Duchenne |
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |