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A 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 : October 17, 2018
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.


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 ]


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

Locations
United States, Georgia
Rare Disease Research, LLC Recruiting
Atlanta, Georgia, United States, 30318
Contact: Tu Tran, BS         
Principal Investigator: Han Phan, MD         
United States, Maryland
Kennedy Krieger Institute Recruiting
Baltimore, Maryland, United States, 21205
Contact: Genila Bibat, MD         
Principal Investigator: Katherine R Wagner, MD, PhD         
Sponsors and Collaborators
Catabasis Pharmaceuticals
Investigators
Study Chair: Joanne M Donovan, Chief Medical Officer, MD, PhD Catabasis Pharmaceuticals

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: October 17, 2018
Last Verified: October 2018

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