Becker Muscular Dystrophy, Healthy Volunteer
Conditions
Keywords
Healthy Volunteers, Becker Muscular Dystrophy
Brief summary
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).
Detailed description
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.
Interventions
DRUGEDG-5506
EDG-5506 is administered orally as a single dose or once daily
DRUGPlacebo
Placebo is administered orally as a single dose or once daily
Sponsors
Worldwide Clinical Trials
Edgewise Therapeutics, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 55 Years
Healthy volunteers
Yes
Inclusion criteria
* For all potential participants (Healthy volunteers and BMD): Male or for HV: female. For all: adults aged 18 to 55 years at time of consent. * For HVs: Good general health, with no significant medical history, no clinically significant abnormalities on physical exam * For BMD: Diagnosis of BMD based on documentation of mutation(s) in the dystrophin gene and BMD phenotype * For BMD: Ability to ambulate * For all: Weight greater than or equal to 50 kg and BMI less than 33 kg/m2 * For HV: Females must be of non-childbearing potential. * For all: Males with female partners must use a medically accepted contraceptive regimen from first dose through 90 days after the last dose * For all: Non-smoker and must not have used any tobacco products within 3 months prior to the Screening visit. * For all: Able and willing to attend the necessary visits at the study center.
Exclusion criteria
* For all: History of, or physical exam findings indicating clinically significant endocrine, neurological, gastrointestinal, cardiovascular, hematological, hepatic, immunological, renal, respiratory, or genitourinary abnormalities or diseases that, in the opinion of the Investigator, would render the subject being unsuitable for the study. * For all: Unable to refrain from strenuous exercise for 3 days prior to check-in and during study. * For all: Participation in any other investigational drug study within 30 days or 5 half-lives (whichever is longer) of dosing in the present study.
Design outcomes
Primary
| Measure | 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 |
Secondary
| Measure | Time frame |
|---|---|
| Renal clearance (CLR) | Up to 42 days of testing |
| Fraction excreted in urine (Fe) | Up to 42 days of testing |
| Drug excreted unchanged in urine (Amt0-24) | Up to 42 days of testing |
| 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) | Up to 42 days of testing |
| Plasma half-life (T½) | Up to 42 days of testing |
Countries
United States
Outcome results
None listed