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

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ClinicalTrials.gov Identifier: NCT03703882
Recruitment Status : Recruiting
First Posted : October 12, 2018
Last Update Posted : April 12, 2019
Sponsor:
Information provided by (Responsible Party):
Catabasis Pharmaceuticals

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.


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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


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




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 ]


Information from the National Library of Medicine

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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

Information from the National Library of Medicine

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


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

  Hide Study Locations
Locations
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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 Not yet 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 Not yet 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 Not yet recruiting
Cincinnati, Ohio, United States, 45229
Contact: Cuixia Tian, MD         
Principal Investigator: Cuixia Tian, MD         
MetroHealth Medical Center Not yet 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 Not yet recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Gihan Tennekoon, MD         
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 Not yet recruiting
Westmead, New South Wales, Australia, 2145
Contact: Manoj Menezes, MD, PhD         
Principal Investigator: Manoj Menezes, MD, PhD         
Australia, Queensland
Queensland Children's Hospital Not yet 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
Children's Hospital of Western Ontario- LHSC Not yet recruiting
London, Ontario, Canada, N6A 4G5
Contact: Jennifer Petzke, BS         
Principal Investigator: Craig Campbell, MD         
Children's Hospital of Eastern Ontario Not yet recruiting
Ottawa, Ontario, Canada, K1H 8L1
Contact: Hugh McMillan, MD         
Principal Investigator: Hugh McMillan, MD         
Canada, Quebec
CHU Sainte-Justine Not yet recruiting
Montréal, Quebec, Canada, H3T 1C5
Contact: Cam-Tu Nguyen, MD         
Principal Investigator: Cam-Tu Nguyen, MD         
Germany
University of Hamburg Not yet recruiting
Hamburg, Germany, 20246
Contact: Jessika Johannsen, MD         
Principal Investigator: Jessika Johannsen, MD         
University of Munich Not yet 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 Not yet 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 Not yet recruiting
London, United Kingdom, SE1 7EU
Contact: Vasantha Gowda, MBBS, FRCPCH         
Principal Investigator: Vasantha Gowda, MBBS, FRCPCH         
Great Ormond Street Hospital (GOSH) Not yet 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
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Study Chair: Joanne M Donovan, Chief Medical Officer, MD, PhD Catabasis Pharmaceuticals

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Responsible Party: Catabasis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03703882     History of Changes
Other Study ID Numbers: CAT-1004-301
First Posted: October 12, 2018    Key Record Dates
Last Update Posted: April 12, 2019
Last Verified: April 2019

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