Duchenne Muscular DystrophyPhase 3GalaxyDMDNCT03917719
An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
A Phase 3 study testing Edasalonexent in people with Duchenne Muscular Dystrophy, run by Catabasis Pharmaceuticals at 23 sites in 6 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 4 to 12 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Edasalonexent
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 130 participants
- Where
- 23 sitesin 6 countries
- Study dates
- 14 Mar 2019to 26 Oct 2020
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 12 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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
- 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
Who cannot join
- 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:
- 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
Trial locations and contacts
23 sites in 6 countries.
23 sites in 6 countries
United States15 sites
University of Michigan
Ann Arbor, Michigan, 48109
Rare Disease Research, LLC
Atlanta, Georgia, 30318
Kennedy Krieger Institute
Baltimore, Maryland, 21205
Johns Hopkins School of Medicine
Baltimore, Maryland, 21287
Boston Children's Hospital
Boston, Massachusetts, 02115
University of Kansas Medical Center
Fairway, Kansas, 66205
Cook Children's Medical Center
Fort Worth, Texas, 76104
University of Iowa Children's Hospital
Iowa City, Iowa, 52242
Las Vegas Clinic
Las Vegas, Nevada, 89145
Vanderbilt University Medical Center
Nashville, Tennessee, 37212
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Shriners Hospital for Children
Portland, Oregon, 97239
UC Davis
Sacramento, California, 95817
University of Utah
Salt Lake City, Utah, 84112
University of Texas Health Science Center at San Antonio
San Antonio, Texas, 78229
United Kingdom3 sites
Bristol Children's Hospital
Bristol, BS2 8AE
Great Ormond Street Hospital (GOSH)
London, WC1N 3JH
Royal Manchester Children's Hospital
Manchester, M13 9WL
Germany2 sites
University of Hamburg
Hamburg, 20246
University of Munich
Munich, 80337
About this study
Official title: An Open-Label Extension Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy
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.
Read the 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.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Edasalonexent
- Keywords
- Muscular Dystrophies, Musculoskeletal Diseases, Neuromuscular Diseases, DMD, dystrophin, dystrophy, Duchenne
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Musculoskeletal Diseases, Neuromuscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EdasalonexentDrug
Also called Edasa, CAT-1004
100 mg/kg/day
| Study group | Role | What they receive |
|---|---|---|
| Dose 1 | Experimental | Edasalonexent 100mg/kg/day. Capsules taken by mouth three times per day. Receives: Drug: Edasalonexent |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of long-term treatment with edasalonexent measured by number of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Measured: 104 Weeks
Secondary outcomes (4)
Durability of effects of edasalonexent on physical function as measured by the North Star Ambulatory Assessment (NSAA)
Measured: 104 Weeks
Durability of effects of edasalonexent on physical function as measured by the 10-meter walk/run test
Measured: 104 Weeks
Durability of effects of edasalonexent on physical function as measured by the time to stand from supine
Measured: 104 Weeks
Durability of effects of edasalonexent on physical function as measured by the 4-stair climb
Measured: 104 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2019
- Study start14 Mar 2019
- Primary completion26 Oct 2020
- Study completion26 Oct 2020
Why it 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
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 130 participants (actual)
Sponsor and investigators
- Lead sponsor
- Catabasis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Joanne M Donovan, MD, PhD, Study chair, Catabasis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03917719
- Sponsor's protocol ID
- CAT-1004-302
- Acronym
- GalaxyDMD
- Status last verified
- Nov 2020
- First submitted
- 12 Apr 2019
- Last updated
- 23 Nov 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Extension Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT03917719
- Acronym / sponsor ID
- GalaxyDMD / CAT-1004-302
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Muscular Dystrophies, Musculoskeletal Diseases, Neuromuscular Diseases, DMD, dystrophin, dystrophy, Duchenne
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason 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
- Enrolment
- 130 participants (actual)
- Lead sponsor
- Catabasis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 14 Mar 2019 (actual)
- Primary completion
- 26 Oct 2020 (actual)
- Study completion
- 26 Oct 2020 (actual)
- First posted
- 17 Apr 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dose 1 | Experimental | Drug: EdasalonexentEdasalonexent 100mg/kg/day. Capsules taken by mouth three times per day. |
Who may qualify
- Ages
- 4 to 12 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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
- 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
Key exclusion criteria (7 listed)
- 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
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EdasalonexentAlso: Edasa, CAT-1004100 mg/kg/day | Drug | Dose 1 |
Primary outcome
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
4 secondary outcomes
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
Results and safety
The study was stopped early (reason given: 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). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Nov 2020.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Joanne M Donovan, MD, PhD study chair
Catabasis Pharmaceuticals
23 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Edasalonexent in people with Duchenne Muscular Dystrophy, run by Catabasis Pharmaceuticals at 23 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 23 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in United States, United Kingdom, Germany, Australia, Canada and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: 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). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Nov 2020.