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出境医 / 临床实验 / An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (GalaxyDMD)

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

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.


Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: Edasalonexent Phase 3

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.
Study Design
Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 130 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Extension Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy
Actual Study Start Date : March 14, 2019
Actual Primary Completion Date : October 26, 2020
Actual Study Completion Date : October 26, 2020
Arms and Interventions
Arm Intervention/treatment
Experimental: Dose 1
Edasalonexent 100mg/kg/day. Capsules taken by mouth three times per day.
 Drug: Edasalonexent
100 mg/kg/day
Other Names:
  • Edasa
  • CAT-1004
Outcome Measures
Primary Outcome Measures :
  1. 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 Measures :
  1. Durability of effects of edasalonexent on physical function as measured by the North Star Ambulatory Assessment (NSAA) [ Time Frame: 104 Weeks ]
  2. Durability of effects of edasalonexent on physical function as measured by the 10-meter walk/run test [ Time Frame: 104 Weeks ]
  3. Durability of effects of edasalonexent on physical function as measured by the time to stand from supine [ Time Frame: 104 Weeks ]
  4. Durability of effects of edasalonexent on physical function as measured by the 4-stair climb [ Time Frame: 104 Weeks ]

Eligibility Criteria
Layout table for eligibility information
Ages Eligible for Study:   4 Years to 12 Years   (Child)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
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
Contacts and Locations

Locations
Show Show 23 study locations
Sponsors and Collaborators
Catabasis Pharmaceuticals
Investigators
Layout table for investigator information
Study Chair: Joanne M Donovan, MD, PhD Catabasis Pharmaceuticals
Tracking Information
First Submitted Date  ICMJE April 12, 2019
First Posted Date  ICMJE April 17, 2019
Last Update Posted Date November 23, 2020
Actual Study Start Date  ICMJE March 14, 2019
Actual Primary Completion Date October 26, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 16, 2019) 		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 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 16, 2019)
  • Durability of effects of edasalonexent on physical function as measured by the North Star Ambulatory Assessment (NSAA) [ Time Frame: 104 Weeks ]
  • Durability of effects of edasalonexent on physical function as measured by the 10-meter walk/run test [ Time Frame: 104 Weeks ]
  • Durability of effects of edasalonexent on physical function as measured by the time to stand from supine [ Time Frame: 104 Weeks ]
  • Durability of effects of edasalonexent on physical function as measured by the 4-stair climb [ Time Frame: 104 Weeks ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
Official Title  ICMJE An Open-Label Extension Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE Drug: Edasalonexent
100 mg/kg/day
Other Names:
  • Edasa
  • CAT-1004
Study Arms  ICMJE Experimental: Dose 1
Edasalonexent 100mg/kg/day. Capsules taken by mouth three times per day.
Intervention: Drug: Edasalonexent
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: November 19, 2020) 		130
Original Estimated Enrollment  ICMJE
 (submitted: April 16, 2019) 		100
Actual Study Completion Date  ICMJE October 26, 2020
Actual Primary Completion Date October 26, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Ages  ICMJE 4 Years to 12 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Canada,   Germany,   Sweden,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03917719
Other Study ID Numbers  ICMJE CAT-1004-302
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Catabasis Pharmaceuticals
Study Sponsor  ICMJE Catabasis Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Chair: Joanne M Donovan, MD, PhD Catabasis Pharmaceuticals
PRS Account Catabasis Pharmaceuticals
Verification Date November 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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