Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy



Status:Recruiting
Conditions:Neurology
Therapuetic Areas:Neurology
Healthy:No
Age Range:4 - 7
Updated:3/31/2019
Start Date:October 2, 2018
End Date:June 2020
Contact:Maria C Mancini, Vice President, Clinical Operations
Email:DMDtrials@catabasis.com
Phone:617-349-1971

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A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy

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.

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.

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
We found this trial at
25
sites
Portland, Oregon 97239
Principal Investigator: Erika Finanger, MD
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707 North Broadway
Baltimore, Maryland 21205
443-923-9200
Principal Investigator: Katherine R Wagner, MD, PhD
Kennedy Krieger Institute While not officially part of Johns Hopkins Medicine, Kennedy Krieger Institute is...
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4650 Sunset Blvd
Los Angeles, California 90027
 (323) 660-2450
Principal Investigator: Leigh Ramos-Platt, MD
Childrens Hospital Los Angeles Children's Hospital Los Angeles is a 501(c)(3) nonprofit hospital for pediatric...
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Los Angeles, CA
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Minneapolis, Minnesota 55455
(612) 625-5000
Principal Investigator: Peter Karachunski, MD
Univ of Minnesota With a flagship campus in the heart of the Twin Cities, and...
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1211 Medical Center Dr
Nashville, Tennessee 37232
(615) 322-5000
Principal Investigator: W. Bryan Burnette, MD
Vanderbilt Univ Med Ctr Vanderbilt University Medical Center (VUMC) is a comprehensive healthcare facility dedicated...
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601 Children's Lane
Norfolk, Virginia 23507
(757) 668-7000
Principal Investigator: Crystal Proud, MD
Children's Hospital of The King's Daughters Children
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Principal Investigator: Gihan Tennekoon, MD
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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Philadelphia, PA
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201 Presidents Circle
Salt Lake City, Utah 84108
801) 581-7200
Principal Investigator: Russell Butterfield, MD
University of Utah Research is a major component in the life of the U benefiting...
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Salt Lake City, UT
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4502 Medical Drive
San Antonio, Texas 78284
(210) 567-7000
Principal Investigator: Ratna Bhavaraju-Sanka, MD
University of Texas Health Science Center at San Antonio The University of Texas Health Science...
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500 S State St
Ann Arbor, Michigan 48109
(734) 764-1817
Principal Investigator: Erin Neil, DO
University of Michigan The University of Michigan was founded in 1817 as one of the...
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Atlanta, Georgia 30318
Principal Investigator: Han Phan, MD
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Baltimore, Maryland 21287
Principal Investigator: Jessica Nance, MD
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300 Longwood Ave
Boston, Massachusetts 02115
(617) 355-6000
Principal Investigator: Partha Ghosh, MD
Boston Children's Hospital Boston Children's Hospital is a 395-bed comprehensive center for pediatric health care....
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Chicago, Illinois 60612
Principal Investigator: Peter Heydemann, MD
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Cincinnati, Ohio 45229
Principal Investigator: Cuixia Tian, MD
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2500 Metrohealth Dr
Cleveland, Ohio 44109
(216) 778-7800
Principal Investigator: Andre Prochoroff, MD
MetroHealth Med Ctr The MetroHealth System is one of the largest, most comprehensive health care...
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Fairway, Kansas 66205
Principal Investigator: Jeffrey Statland, MD
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Fairway, KS
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801 7th Avenue
Fort Worth, Texas 76104
(682) 885-4000
Principal Investigator: Warren Marks, MD
Cook Children's Medical Center Cook Children's Health Care System is a not-for-profit, nationally recognized pediatric...
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Iowa City, Iowa 52242
Principal Investigator: Kathy Mathews, MD
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Las Vegas, Nevada 89145
Principal Investigator: Jonathan McKinnon, MD
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Las Vegas, NV
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1 Children's Way
Little Rock, Arkansas 72202
(501) 364-1100
Principal Investigator: Vikki A Stefans, MD
Arkansas Children's Hospital Arkansas Children's Hospital (ACH) is the only pediatric medical center in Arkansas...
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13535 Nemours Parkway
Orlando, Florida 32827
(407) 567-4000
Principal Investigator: Richard Finkel, MD
Nemours Children's Hospital Nemours Children's Hospital in Orlando brings pediatric specialty care never before offered...
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Richmond, Virginia 23298
Principal Investigator: Amy Harper, MD
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Sacramento, California 95817
Principal Investigator: Craig McDonald, MD
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Westmead, New South Wales 2145
Principal Investigator: Manoj Menezes, MD, PhD
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