Edgewise Sevasemten (EDG-5506) Phase 2 Clinical Trial for Duchenne muscular dystrophy
Study Overview
- Age
- 4–9 years
- Phase
- Phase 2
- Sponsor
- Edgewise Therapeutics
- Therapeutic Approach
- Improving Muscle Growth & Protection
- Variant Requirement
- A documented mutation on the DMD gene and phenotype consistent with Duchenne muscular dystrophy.
- Eligible Sex
- Male
- Ambulation
- Ambulatory
- Study Start (Actual)
- 2022-10-24
- Primary Completion (Estimated)
- 2027-01
- Study Completion (Estimated)
- 2027-01
- Enrollment (Estimated)
- 76
- Countries
- United States
Study Requirements and Criteria
Steroid Use
- Aged 4-9 years on a stable dose of corticosteroids for a minimum of 6 months prior to the Baseline visit.
- Aged 4-7 years not on corticosteroids within 6 months prior to the Baseline visit.
Inclusion Criteria
- Able to complete the stand from supine in ≤ 10 seconds and able to perform the 4-stair climb in < 10 seconds at the Screening visit.
- Body weight greater than or equal to 15 kg at the Screening visit.
Exclusion Criteria
- Medical history or clinically significant physical exam/laboratory result that, in the opinion of the investigator, would render the participant unsuitable for the study. This includes venous access that would be too difficult to facilitate repeated blood testing.
- A forced vital capacity < 60% predicted at the Screening visit for those participants who are > 8 years old at Screening.
- A cardiac echocardiography showing left ventricular ejection < 45% at the Screening visit.
- Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of the Screening visit in the present study.
- Receipt of a stable dose of an approved exon-skipping therapy with a treatment duration of less than 1 year prior to the Screening visit.
Contacts and Locations
Contacts and Locations
Contact
Not yet provided
Locations
This study has 14 locations
United States
Arkansas Locations
Little Rock, Arkansas, United States, 72202
Arkansas Children's Hospital
California Locations
Los Angeles, California, United States, 90095
UCLA Medical Center
Sacramento, California, United States, 95817
UC Davis Medical Center
Colorado Locations
Aurora, Colorado, United States, 80045
Children's Hospital Colorado
Florida Locations
Gainesville, Florida, United States, 32610
University of Florida
Georgia Locations
Atlanta, Georgia, United States, 30329
Rare Disease Research
Iowa Locations
Iowa City, Iowa, United States, 52242
University of Iowa
Kansas Locations
Kansas City, Kansas, United States, 66160
University of Kansas Medical Center
Maryland Locations
Baltimore, Maryland, United States, 21205
Kennedy Krieger Institute
Massachusetts Locations
Worcester, Massachusetts, United States, 01605
University of Massachusetts Memorial Medical Center
Missouri Locations
St Louis, Missouri, United States, 63110
Washington University School of Medicine
Ohio Locations
Cincinnati, Ohio, United States, 45229
Cincinnati Children's Hospital
Columbus, Ohio, United States, 43205
Nationwide Children's Hospital
Texas Locations
Fort Worth, Texas, United States, 76104
Cook Children's Medical Center
Clinical Trial Registry
NCT ID
NCT05540860This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.