Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy, McArdle Disease
Conditions
Brief summary
Incidence, frequency, and severity of adverse events and serious adverse events in those treated with EDG-5506 or placebo., Change in serum creatine kinase (CK) from baseline to week 16.
Detailed description
Change in serum CK from pre-exercise baseline to week 52., Incidence of treatment-emergent abnormal laboratory test results (clinical chemistry, hematology, and urinalysis). Change from Baseline in: - Safety laboratory parameters - Vital signs - Physical and neurological examination - Electrocardiogram (ECG) parameters - Cardiac function as assessed by echocardiogram - Pulmonary function as assessed by spirometry (FEV1,FVC) - C-SSRS (Columbia Suicide Severity Rating Scale), Steady state plasma concentrations of sevasemten
Interventions
Sponsors
Edgewise Therapeutics Inc.
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence, frequency, and severity of adverse events and serious adverse events in those treated with EDG-5506 or placebo., Change in serum creatine kinase (CK) from baseline to week 16. | — |
Secondary
| Measure | Time frame |
|---|---|
| Change in serum CK from pre-exercise baseline to week 52., Incidence of treatment-emergent abnormal laboratory test results (clinical chemistry, hematology, and urinalysis). Change from Baseline in: - Safety laboratory parameters - Vital signs - Physical and neurological examination - Electrocardiogram (ECG) parameters - Cardiac function as assessed by echocardiogram - Pulmonary function as assessed by spirometry (FEV1,FVC) - C-SSRS (Columbia Suicide Severity Rating Scale), Steady state plasma concentrations of sevasemten | — |
Countries
Denmark
Outcome results
None listed