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An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (GalaxyDMD)

Primary Purpose

Duchenne Muscular Dystrophy

Status
Terminated
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Edasalonexent
Sponsored by
Catabasis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring Muscular Dystrophies, Musculoskeletal Diseases, Neuromuscular Diseases, DMD, dystrophin, dystrophy, Duchenne

Eligibility Criteria

4 Years - 12 Years (Child)MaleDoes not accept healthy volunteers

For Patients who Completed CAT-1004-201 or CAT-1004-301:

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
  • Completion of either CAT-1004-201 or CAT-1004-301

Exclusion Criteria:

  • In the Investigator's opinion, unwilling or unable for any reason to complete all study assessments and laboratory tests and comply with scheduled visits, administration of drug, and all other study procedures

For Siblings of Patients who Completed CAT-1004-201 or CAT-1004-301:

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
  • A sibling of a patient who completed either CAT-1004-201 or CAT-1004-301
  • 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
  • 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 oral corticosteroids at screening; 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 are currently on or plan to initiate treatment with approved oligonucleotide exon-skipping therapies, and expected to continue treatment throughout the study, will be eligible
  • Use of the following within 4 weeks prior to Day 1: immunosuppressive therapy, anticoagulants, cyclosporine, dihydroergotamine, ergotamine, fentanyl, alfentanil, pimozide, quinidine, sirolimus or tacrolimus
  • Use of human growth hormone within 3 months prior to Day 1
  • Other prior or ongoing significant medical conditions

Sites / Locations

  • UC Davis
  • Rare Disease Research, LLC
  • University of Iowa Children's Hospital
  • University of Kansas Medical Center
  • Kennedy Krieger Institute
  • Johns Hopkins School of Medicine
  • Boston Children's Hospital
  • University of Michigan
  • Las Vegas Clinic
  • Shriners Hospital for Children
  • Children's Hospital of Philadelphia
  • Vanderbilt University Medical Center
  • Cook Children's Medical Center
  • University of Texas Health Science Center at San Antonio
  • University of Utah
  • Royal Children's Hospital
  • Children's Hospital of Eastern Ontario
  • University of Hamburg
  • University of Munich
  • Queen Silvia Children's Hospital
  • Bristol Children's Hospital
  • Great Ormond Street Hospital (GOSH)
  • Royal Manchester Children's Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Dose 1

Arm Description

Edasalonexent 100mg/kg/day. Capsules taken by mouth three times per day.

Outcomes

Primary Outcome Measures

Safety and tolerability of long-term treatment with edasalonexent measured by number of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)

Secondary Outcome Measures

Durability of effects of edasalonexent on physical function as measured by the North Star Ambulatory Assessment (NSAA)
Durability of effects of edasalonexent on physical function as measured by the 10-meter walk/run test
Durability of effects of edasalonexent on physical function as measured by the time to stand from supine
Durability of effects of edasalonexent on physical function as measured by the 4-stair climb

Full Information

First Posted
April 12, 2019
Last Updated
November 19, 2020
Sponsor
Catabasis Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT03917719
Brief Title
An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
Acronym
GalaxyDMD
Official Title
An Open-Label Extension Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy
Study Type
Interventional

2. Study Status

Record Verification Date
November 2020
Overall Recruitment Status
Terminated
Why Stopped
The Phase 3 PolarisDMD trial did not meet the primary endpoint. As a result, activities related to the development of edasalonexent have stopped including the CAT-1004-302 Open-Label Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
Study Start Date
March 14, 2019 (Actual)
Primary Completion Date
October 26, 2020 (Actual)
Study Completion Date
October 26, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Catabasis Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The GalaxyDMD study is a global Phase 3, open-label, treatment extension study to evaluate the safety, tolerability, and durability of effect in long-term dosing of edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD. Patients who completed CAT-1004-201 or CAT-1004-301 or siblings of these boys from 4-12 years of age (up to 13th birthday) will be enrolled. Edasalonexent is an orally administered small molecule that inhibits NF-kB, which is a 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.
Detailed Description
The study includes a 104-week open-label treatment period with edasalonexent. Patients who completed CAT-1004-201 or CAT-1004-301 and eligible siblings of these boys will be enrolled in this trial.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
Muscular Dystrophies, Musculoskeletal Diseases, Neuromuscular Diseases, DMD, dystrophin, dystrophy, Duchenne

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
130 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Dose 1
Arm Type
Experimental
Arm Description
Edasalonexent 100mg/kg/day. Capsules taken by mouth three times per day.
Intervention Type
Drug
Intervention Name(s)
Edasalonexent
Other Intervention Name(s)
Edasa, CAT-1004
Intervention Description
100 mg/kg/day
Primary Outcome Measure Information:
Title
Safety and tolerability of long-term treatment with edasalonexent measured by number of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Time Frame
104 Weeks
Secondary Outcome Measure Information:
Title
Durability of effects of edasalonexent on physical function as measured by the North Star Ambulatory Assessment (NSAA)
Time Frame
104 Weeks
Title
Durability of effects of edasalonexent on physical function as measured by the 10-meter walk/run test
Time Frame
104 Weeks
Title
Durability of effects of edasalonexent on physical function as measured by the time to stand from supine
Time Frame
104 Weeks
Title
Durability of effects of edasalonexent on physical function as measured by the 4-stair climb
Time Frame
104 Weeks

10. Eligibility

Sex
Male
Gender Based
Yes
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
For Patients who Completed CAT-1004-201 or CAT-1004-301: 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 Completion of either CAT-1004-201 or CAT-1004-301 Exclusion Criteria: In the Investigator's opinion, unwilling or unable for any reason to complete all study assessments and laboratory tests and comply with scheduled visits, administration of drug, and all other study procedures For Siblings of Patients who Completed CAT-1004-201 or CAT-1004-301: 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 A sibling of a patient who completed either CAT-1004-201 or CAT-1004-301 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 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 oral corticosteroids at screening; 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 are currently on or plan to initiate treatment with approved oligonucleotide exon-skipping therapies, and expected to continue treatment throughout the study, will be eligible Use of the following within 4 weeks prior to Day 1: immunosuppressive therapy, anticoagulants, cyclosporine, dihydroergotamine, ergotamine, fentanyl, alfentanil, pimozide, quinidine, sirolimus or tacrolimus Use of human growth hormone within 3 months prior to Day 1 Other prior or ongoing significant medical conditions
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Joanne M Donovan, MD, PhD
Organizational Affiliation
Catabasis Pharmaceuticals
Official's Role
Study Chair
Facility Information:
Facility Name
UC Davis
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
Rare Disease Research, LLC
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30318
Country
United States
Facility Name
University of Iowa Children's Hospital
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
University of Kansas Medical Center
City
Fairway
State/Province
Kansas
ZIP/Postal Code
66205
Country
United States
Facility Name
Kennedy Krieger Institute
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
Johns Hopkins School of Medicine
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
University of Michigan
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
Las Vegas Clinic
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89145
Country
United States
Facility Name
Shriners Hospital for Children
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37212
Country
United States
Facility Name
Cook Children's Medical Center
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Facility Name
University of Texas Health Science Center at San Antonio
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78229
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84112
Country
United States
Facility Name
Royal Children's Hospital
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
Facility Name
Children's Hospital of Eastern Ontario
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1H 8L1
Country
Canada
Facility Name
University of Hamburg
City
Hamburg
ZIP/Postal Code
20246
Country
Germany
Facility Name
University of Munich
City
Munich
ZIP/Postal Code
80337
Country
Germany
Facility Name
Queen Silvia Children's Hospital
City
Gothenburg
ZIP/Postal Code
41685
Country
Sweden
Facility Name
Bristol Children's Hospital
City
Bristol
ZIP/Postal Code
BS2 8AE
Country
United Kingdom
Facility Name
Great Ormond Street Hospital (GOSH)
City
London
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom
Facility Name
Royal Manchester Children's Hospital
City
Manchester
ZIP/Postal Code
M13 9WL
Country
United Kingdom

12. IPD Sharing Statement

Learn more about this trial

An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy

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