An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (GalaxyDMD)
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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: NCT03917719 |
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Recruitment Status :
Terminated
(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)
First Posted : April 17, 2019
Last Update Posted : November 23, 2020
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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 |
| 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 |
| Arm | Intervention/treatment |
|---|---|
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Experimental: Dose 1
Edasalonexent 100mg/kg/day. Capsules taken by mouth three times per day.
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Drug: Edasalonexent
100 mg/kg/day
Other Names:
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- 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 ]
- 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 ]
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| Ages Eligible for Study: | 4 Years to 12 Years (Child) |
| Sexes Eligible for Study: | Male |
| Gender Based Eligibility: | Yes |
| Accepts Healthy Volunteers: | No |
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
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): NCT03917719
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| Study Chair: | Joanne M Donovan, MD, PhD | Catabasis Pharmaceuticals |
| Responsible Party: | Catabasis Pharmaceuticals |
| ClinicalTrials.gov Identifier: | NCT03917719 |
| Other Study ID Numbers: |
CAT-1004-302 |
| First Posted: | April 17, 2019 Key Record Dates |
| Last Update Posted: | November 23, 2020 |
| Last Verified: | November 2020 |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
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Muscular Dystrophies Musculoskeletal Diseases Neuromuscular Diseases DMD |
dystrophin dystrophy Duchenne |
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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 |