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| Name | Class |
|---|---|
| Worldwide Clinical Trials | OTHER |
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EDG-5506 is an investigational product intended to protect and improve function of dystrophic muscle fibers. This Phase 1 study of EDG-5506 will assess the safety, tolerability, and pharmacokinetics (PK) and of EDG-5506 in adult healthy volunteers and in adults with Becker muscular dystrophy (BMD).
Enrolled participants in this study will receive a single oral dose or multiple oral doses of EDG-5506 or a placebo. Blood and urine samples will be collected to measure how EDG-5506 is processed by the body and how the body responds when exposed to EDG-5506. Participants in the single ascending dose part of the study will remain in the clinic for 7 days with a 42-day follow-up period. Participants in the multiple ascending dose part of the study will remain in the clinic for 16 days with a 13-day follow-up period. Safety, tolerability, and pharmacokinetics of EDG-5506 will be assessed in healthy volunteers prior to enrolling participants with Becker muscular dystrophy.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Healthy Volunteer: Single Ascending Dose | Experimental | Single oral ascending dose in healthy volunteers Interventions: Drug: EDG-5506 Drug: Placebo |
|
| Healthy Volunteer: Multiple Ascending Dose | Experimental | Multiple oral ascending doses in healthy volunteers Interventions: Drug: EDG-5506 Drug: Placebo |
|
| Becker Muscular Dystrophy: Multiple Ascending Dose | Experimental | Multiple oral ascending doses in adults with Becker muscular dystrophy Interventions: Drug: EDG-5506 Drug: Placebo |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| EDG-5506 | Drug | EDG-5506 is administered orally as a single dose or once daily |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence, frequency, severity and dose-relationship of adverse events | Up to 42 days of monitoring | |
| Incidence of abnormal laboratory test results (clinical chemistry, hematology, urinalysis, coagulation) | Up to 42 days of monitoring | |
| Incidence of treatment-emergent clinically abnormal electrocardiogram (ECG) | Up to 42 days of monitoring | |
| Incidence of abnormal vital signs | Up to 42 days of monitoring | |
| Incidence of abnormal physical exam findings | Up to 42 days of monitoring |
| Measure | Description | Time Frame |
|---|---|---|
| Plasma maximum measured drug concentration (Cmax) | Up to 42 days of testing | |
| Time of maximum concentration (Tmax) | Up to 42 days of testing | |
| Area under the concentration-time curve (AUC) |
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Inclusion Criteria:
Exclusion Criteria:
Healthy volunteers: Male and non-childbearing potential females. Male Becker muscular dystrophy (BMD) participants
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Edgewise Therapeutics, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Worldwide Clinical Trials | San Antonio | Texas | 78217 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40452637 | Derived | Donovan J, Silverman JA, Barthel B, DuVall M, Madden M, MacDougall J, Kilburn NR, Bronson A, Evanchik M, Gordon G, Koch K, Russell AJ. A Phase 1, Double-Blind, Placebo-Controlled Trial of Sevasemten (EDG-5506), a Selective Modulator of Fast Skeletal Muscle Contraction, in Healthy Volunteers and Adults With Becker Muscular Dystrophy. Muscle Nerve. 2025 Sep;72(3):399-407. doi: 10.1002/mus.28444. Epub 2025 Jun 2. |
| Label | URL |
|---|---|
| Sponsor Website | View source |
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| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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| Placebo | Drug | Placebo is administered orally as a single dose or once daily |
|
| Up to 42 days of testing |
| Plasma half-life (T½) | Up to 42 days of testing |
| Renal clearance (CLR) | Up to 42 days of testing |
| Drug excreted unchanged in urine (Amt0-24) | Up to 42 days of testing |
| Fraction excreted in urine (Fe) | Up to 42 days of testing |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |