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

• ClinicalTrials.gov Identifier: NCT03703882
• Recruitment Status: Recruiting
• First Posted: October 12, 2018
• Last Update Posted: July 16, 2019
• 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)
• Estimated Enrollment: 125 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
• Estimated Primary Completion Date: June 2020
• Estimated Study Completion Date: June 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

Contacts

Contact: Maria C Mancini, Vice President, Clinical Operations; 617-349-1971; DMDtrials@catabasis.com

  Hide Study Locations

Locations

United States, Arkansas

Arkansas Children's Hospital; Recruiting

Little Rock, Arkansas, United States, 72202

Contact: Vikki A Stefans, MD

Principal Investigator: Vikki A Stefans, MD

United States, California

Children's Hospital of Los Angeles; Recruiting

Los Angeles, California, United States, 90027

Contact: Leigh Ramos-Platt, MD

Principal Investigator: Leigh Ramos-Platt, MD

UC Davis; Recruiting

Sacramento, California, United States, 95817

Contact: David Derkacs, BS

Principal Investigator: Craig McDonald, MD

United States, Florida

Nemours Children's Hospital; Recruiting

Orlando, Florida, United States, 32827

Contact: Kristine Moore, RN, CCRP

Principal Investigator: Richard Finkel, MD

United States, Georgia

Rare Disease Research, LLC; Recruiting

Atlanta, Georgia, United States, 30318

Contact: Tu Tran, BS

Principal Investigator: Han Phan, MD

United States, Illinois

Rush University Children's Hospital; Recruiting

Chicago, Illinois, United States, 60612

Contact: Peter Heydemann, MD

Principal Investigator: Peter Heydemann, MD

United States, Iowa

University of Iowa Children's Hospital; Recruiting

Iowa City, Iowa, United States, 52242

Contact: Evgenia Folts, BS

Principal Investigator: Kathy Mathews, MD

United States, Kansas

University of Kansas Medical Center; Recruiting

Fairway, Kansas, United States, 66205

Contact: Katherine Roath, BS

Principal Investigator: Jeffrey Statland, MD

United States, Maryland

Kennedy Krieger Institute; Recruiting

Baltimore, Maryland, United States, 21205

Contact: Genila Bibat, MD

Principal Investigator: Katherine R Wagner, MD, PhD

Johns Hopkins School of Medicine; Recruiting

Baltimore, Maryland, United States, 21287

Contact: Robin Worrell- Thorne

Principal Investigator: Jessica Nance, MD

United States, Massachusetts

Boston Children's Hospital; Recruiting

Boston, Massachusetts, United States, 02115

Contact: Deborah Cesar, BS

Principal Investigator: Partha Ghosh, MD

United States, Michigan

University of Michigan; Recruiting

Ann Arbor, Michigan, United States, 48109

Contact: Amy Hurst, CCRC

Principal Investigator: Erin Neil, DO

United States, Minnesota

University of Minnesota; Recruiting

Minneapolis, Minnesota, United States, 55455

Contact: MD Center

Principal Investigator: Peter Karachunski, MD

United States, Nevada

Las Vegas Clinic; Recruiting

Las Vegas, Nevada, United States, 89145

Contact: Kaitlyn Fell

Principal Investigator: Jonathan McKinnon, MD

United States, Ohio

Cincinnati Children's Hospital; Recruiting

Cincinnati, Ohio, United States, 45229

Contact: Cuixia Tian, MD

Principal Investigator: Cuixia Tian, MD

MetroHealth Medical Center; Recruiting

Cleveland, Ohio, United States, 44109

Contact: Andre Prochoroff, MD

Principal Investigator: Andre Prochoroff, MD

United States, Oregon

Shriners Hospitals for Children; Recruiting

Portland, Oregon, United States, 97239

Contact: Paige Lemhouse, BS

Principal Investigator: Erika Finanger, MD

United States, Pennsylvania

Children's Hospital of Philadelphia; Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contact: Rosetta Chiavacci

Principal Investigator: Gihan Tennekoon, MD

United States, Tennessee

Vanderbilt University Medical Center; Recruiting

Nashville, Tennessee, United States, 37212

Contact: Sarah N McKeel

Principal Investigator: W. Bryan Burnette, MD

United States, Texas

Cook Children's Medical Center; Recruiting

Fort Worth, Texas, United States, 76104

Contact: Kayla Blough, BS

Principal Investigator: Warren Marks, MD

University of Texas Health Science Center at San Antonio; Recruiting

San Antonio, Texas, United States, 78229

Contact: Yogeet Kaur, BS

Principal Investigator: Ratna Bhavaraju-Sanka, MD

United States, Utah

University of Utah; Recruiting

Salt Lake City, Utah, United States, 84112

Contact: Sterling Meisner, BS

Principal Investigator: Russell Butterfield, MD

United States, Virginia

Children's Hospital of the King's Daughters; Recruiting

Norfolk, Virginia, United States, 23510

Contact: Terrie Conklin, RN, CCRC

Principal Investigator: Crystal Proud, MD

Children's Hospital of Richmond at VCU; Recruiting

Richmond, Virginia, United States, 23298

Contact: Sarah Valkovci, BS

Principal Investigator: Amy Harper, MD

Australia, New South Wales

The Children's Hospital at Westmead; Recruiting

Westmead, New South Wales, Australia, 2145

Contact: Manoj Menezes, MD, PhD

Principal Investigator: Manoj Menezes, MD, PhD

Australia, Queensland

Children's Health Queensland Children's Hospital and Health Service; Recruiting

South Brisbane, Queensland, Australia, 4101

Contact: Kate Munro, BSN

Principal Investigator: Anita Cairns, MBBS, FRACP

Australia, Victoria

Royal Children's Hospital; Recruiting

Parkville, Victoria, Australia, 3052

Contact: Jemima Mitchell, BS

Principal Investigator: Monique M Ryan, MD

Canada, Alberta

Alberta Children's Hospital; Recruiting

Calgary, Alberta, Canada, T3B 6A8

Contact: Tiffany Haig, BS

Principal Investigator: Jean Mah, MD

Canada, Ontario

London Health Sciences Centre - Children's Hospital; Recruiting

London, Ontario, Canada, N6A 4G5

Contact: Jennifer Petzke, BS

Principal Investigator: Craig Campbell, MD

Children's Hospital of Eastern Ontario; Recruiting

Ottawa, Ontario, Canada, K1H 8L1

Contact: Hugh McMillan, MD

Principal Investigator: Hugh McMillan, MD

Canada, Quebec

CHU Sainte-Justine; Recruiting

Montréal, Quebec, Canada, H3T 1C5

Contact: Cam-Tu Nguyen, MD

Principal Investigator: Cam-Tu Nguyen, MD

Germany

University of Hamburg; Recruiting

Hamburg, Germany, 20246

Contact: Jessika Johannsen, MD

Principal Investigator: Jessika Johannsen, MD

University of Munich; Recruiting

Munich, Germany, 80337

Contact: Wolfgang Müller-Felber, MD

Contact: Astrid Blaschek, MD

Principal Investigator: Wolfgang Müller-Felber, MD

Ireland

Children's University Hospital; Recruiting

Dublin, Ireland, 1

Contact: Declan O'Rourke, MD

Principal Investigator: Declan O'Rourke, MD

Israel

Hadassah Medical Center; Recruiting

Jerusalem, Israel, 9124001

Contact: Talya Dor-Wollman, MD

Principal Investigator: Talya Dor-Wollman, MD

Sweden

Queen Silvia Children's Hospital; Recruiting

Gothenburg, Sweden, 41685

Contact: Mar Tulinius, MD, PhD

Principal Investigator: Mar Tulinius, MD, PhD

United Kingdom

Bristol Children's Hospital; Recruiting

Bristol, United Kingdom, BS2 8AE

Contact: Kayal Vijayakumar, MD

Principal Investigator: Kayal Vijayakumar, MD

Evelina Children's Hospital; Recruiting

London, United Kingdom, SE1 7EU

Contact: Vasantha Gowda, MBBS, FRCPCH

Principal Investigator: Vasantha Gowda, MBBS, FRCPCH

Great Ormond Street Hospital (GOSH); Recruiting

London, United Kingdom, WC1N 3JH

Contact: Mariacristina Scoto, MD, PhD

Principal Investigator: Mariacristina Scoto, MD, PhD

Royal Manchester Children's Hospital; Recruiting

Manchester, United Kingdom, M13 9WL

Contact: Jill Wilson, RN

Contact: Zainab Suleman, RN

Principal Investigator: Gary McCullagh, MB BCH BAO

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 History of Changes
• Other Study ID Numbers: CAT-1004-301
• First Posted: October 12, 2018
• Last Update Posted: July 16, 2019
• Last Verified: July 2019

• 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