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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
127
Worldwide Clinical Trials
San Antonio, Texas, United States
Incidence, frequency, severity and dose-relationship of adverse events
Time frame: Up to 42 days of monitoring
Incidence of abnormal laboratory test results (clinical chemistry, hematology, urinalysis, coagulation)
Time frame: Up to 42 days of monitoring
Incidence of treatment-emergent clinically abnormal electrocardiogram (ECG)
Time frame: Up to 42 days of monitoring
Incidence of abnormal vital signs
Time frame: Up to 42 days of monitoring
Incidence of abnormal physical exam findings
Time frame: Up to 42 days of monitoring
Plasma maximum measured drug concentration (Cmax)
Time frame: Up to 42 days of testing
Time of maximum concentration (Tmax)
Time frame: Up to 42 days of testing
Area under the concentration-time curve (AUC)
Time frame: Up to 42 days of testing
Plasma half-life (T½)
Time frame: Up to 42 days of testing
Renal clearance (CLR)
Time frame: Up to 42 days of testing
Drug excreted unchanged in urine (Amt0-24)
Time frame: Up to 42 days of testing
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Fraction excreted in urine (Fe)
Time frame: Up to 42 days of testing