Edgewise Sevasemten (EDG-5506) Phase 2 Clinical Trial for Duchenne muscular dystrophy

Active, Not RecruitingPhase 2Improving Muscle Growth & Protection

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

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.

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.

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.

NCT ID

This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.

Study Requirements and Criteria