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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03703882
Recruitment Status : Completed
First Posted : October 12, 2018
Results First Posted : June 21, 2022
Last Update Posted : June 21, 2022
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)
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


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: Baseline (Day 1) to Week 52 ]
    To assess change from baseline in North Star Ambulatory Assessment(NSAA) Total Score at Wk52. NSAA is clinician-reported outcome instrument designed to measure ambulatory function in males with Duchenne muscular dystrophy(DMD). Patients asked to perform 17 different functional activities,including 10MWT,rising from sit to stand,standing on one leg,climbing & descending a step,stand from supine, lifting the head, standing on heels, & jumping. Each function activity will be scored as0=(unable to achieve independently),scored as1=(modified method but achieves goal independent of physical assistance from another),or scored as2=(no obvious modification of activity)or "Not Scored". If NSAA test was performed & any of the individual items are scored as "not scored"(i.e, for reasons unrelated to patients physical capabilities), corresponding total score will be set to missing. Sum of 17 scores will be used to form an ordinal total score(range 0-34).Higher scores imply better functional status


Secondary Outcome Measures :
  1. Change From Baseline in 10-meter Walk/Run Test [ Time Frame: Baseline (Day 1) to Week 52 ]
    To assess the changes from baseline to Week 52 on the 10-meter walk/run test (10MWT). For timed function tests (TFTs), the time will be set to 12 seconds and the speed to 0 if the TFT assessment meets the following TFT grading criteria. Grade of 1 or 2 (from a 6-point scale). 1=Unable to walk independently 2=Unable to walk independently but can walk with knee-ankle foot orthoses or support from a person 3=Highly adapted wide based lordotic gait. Cannot increase walking speed 4=Moderately adapted gait. Can pick up speed but cannot run 5=Able to pick up speed, but runs with a double stance phase, i.e. cannot achieve both feet off the ground 6=Runs and gets both feet off the ground (with no double stance phase)

  2. Change From Baseline in Time to Stand From Supine [ Time Frame: Baseline (Day 1) to Week 52 ]
    To assess the change from baseline in the stand from supine speed at Week 52. For timed function tests (TFTs) , the time will be set to 12 seconds and the speed to 0 if the TFT assessment meets the following TFT grading criteria. Grade of 1 or 2 (from a 6-point scale). 1 = Unable to stand from supine, even with use of a chair, 2 = Assisted Gowers - requires furniture for assist in arising from supine to full upright posture (no time to be recorded) 3=Rolls over, stands up with both hands "climbing up" the legs to achieve full upright posture 4=Rolls over, stands up with 1 hand support on leg 5=Rolls to the side and stands up with one or both hands on the floor to start to rise but does not touch legs 6=Stands up without rolling over or using hands on legs or floor

  3. Change From Baseline in 4-stair Climb [ Time Frame: Baseline (Day 1) to Week 52 ]
    To assess the change from baseline to Week 52 on the 4-Stair Climb. For timed function tests (TFTs) , the time will be set to 12 seconds and the speed to 0 if the TFT assessment meets the following TFT grading criteria. Grade of 1(from a 6-point scale)1=Unable to climb 4 standard stairs(no time recorded) 2=Climbs 4 standard stairs "marking time"(climbs one foot at a time, with both feet on a step before moving to next step), uses both arms on one or both handrails or uses 1 handrail and the other arm pushes on the leg 3=Climbs 4 standard stairs "marking time", using one arm on one handrail or one hand pushing on leg or body 4=Climbs 4 standard stairs "marking time", not needing handrail and not using hands to push on leg 5=Climbs 4 standard stairs alternating feet, needs handrail/s for support or uses arms to push on the leg or body 6=Climbs 4 standard stairs alternating feet, not needing handrail support or using arm to push on the leg

  4. Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [ Time Frame: Up to Week 52 ]
    Adverse events that occurred from the time of the administration of the first dose of investigational product (IP) through the end of the safety follow-up were considered treatment-emergent AEs (TEAEs). Serious adverse event (SAE).



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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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


Locations
Hide Hide 40 study locations
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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
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Study Chair: Joanne M Donovan, Chief Medical Officer, MD, PhD Catabasis Pharmaceuticals
  Study Documents (Full-Text)

Documents provided by Catabasis Pharmaceuticals:
Study Protocol  [PDF] April 16, 2020
Statistical Analysis Plan  [PDF] October 5, 2020

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Catabasis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03703882    
Other Study ID Numbers: CAT-1004-301
First Posted: October 12, 2018    Key Record Dates
Results First Posted: June 21, 2022
Last Update Posted: June 21, 2022
Last Verified: June 2022

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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