Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (PolarisDMD)

• ClinicalTrials.gov Identifier: NCT03703882
• Recruitment Status: Completed
• First Posted: October 12, 2018
• Last Update Posted: November 20, 2020
• Sponsor: Catabasis Pharmaceuticals
• Information provided by (Responsible Party): Catabasis Pharmaceuticals

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Muscular Dystrophy, Duchenne	Drug: Edasalonexent; Drug: Placebo	Phase 3

Detailed Description:

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 Design

• 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

Arms and Interventions

Arm	Intervention/treatment
Experimental: Dose 1; Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day.	Drug: Edasalonexent; 100 mg/kg/day; Other Names: Edasa; CAT-1004
Placebo Comparator: Placebo; Matching placebo	Drug: Placebo; Placebo

Outcome Measures

Primary Outcome Measures :

1. Change from baseline in North Star Ambulatory Assessment (NSAA) [ Time Frame: 52 Weeks ]

Secondary Outcome Measures :

1. Safety and tolerability measured by number of treatment- emergent adverse events (TEAEs) and serious adverse events (SAEs) [ Time Frame: 52 Weeks ]
2. Change from baseline in 10-meter walk/run test [ Time Frame: 52 Weeks ]
3. Change from baseline in time to stand from supine [ Time Frame: 52 Weeks ]
4. Change from baseline in 4-stair climb [ Time Frame: 52 Weeks ]

Other Outcome Measures:

1. Change from baseline in muscle strength testing assessed by knee extension and elbow flexion [ Time Frame: 52 Weeks ]
2. Change from baseline in the Performance of Upper Limb (PUL) Scale to assess upper limb function [ Time Frame: 52 Weeks ]
3. 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 ]

Eligibility Criteria

• Ages Eligible for Study: 4 Years to 7 Years (Child)
• Sexes Eligible for Study: Male
• Accepts Healthy Volunteers: No

Criteria

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

Contacts and Locations

Locations

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

Sponsors and Collaborators

Catabasis Pharmaceuticals

Investigators

• Study Chair: Joanne M Donovan, Chief Medical Officer, MD, PhD; Catabasis Pharmaceuticals

More Information

• Responsible Party: Catabasis Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT03703882
• Other Study ID Numbers: CAT-1004-301
• First Posted: October 12, 2018
• 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

Keywords provided by Catabasis Pharmaceuticals:

Muscular Dystrophies; Musculoskeletal Diseases; Neuromuscular Diseases; Duchenne muscular dystrophy; DMD; dystrophin; dystrophy; Duchenne

Additional relevant MeSH terms:

Muscular Dystrophies; Muscular Dystrophy, Duchenne; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked