Clinical trial NCT04585464: A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults
Official title: A Phase 1, Randomized, Placebo-Controlled, Double-blind, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and PK of EDG-5506 in Adult Healthy Volunteers and Adults With Becker Muscular Dystrophy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Healthy Volunteer
Becker Muscular Dystrophy
Keywords: Healthy Volunteers
Becker Muscular Dystrophy
Lead sponsor: Edgewise Therapeutics, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Worldwide Clinical Trials
First posted: 2020-10-14
Enrollment: 127
Sex: ALL
Minimum age (years): 18
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Incidence, frequency, severity and dose-relationship of adverse events
timeFrame: Up to 42 days of monitoring
measure: Incidence of abnormal laboratory test results (clinical chemistry, hematology, urinalysis, coagulation)
timeFrame: Up to 42 days of monitoring
measure: Incidence of treatment-emergent clinically abnormal electrocardiogram (ECG)
timeFrame: Up to 42 days of monitoring
measure: Incidence of abnormal vital signs
timeFrame: Up to 42 days of monitoring
measure: Incidence of abnormal physical exam findings
timeFrame: Up to 42 days of monitoring
Secondary outcomes:
measure: Plasma maximum measured drug concentration (Cmax)
timeFrame: Up to 42 days of testing
measure: Time of maximum concentration (Tmax)
timeFrame: Up to 42 days of testing
measure: Area under the concentration-time curve (AUC)
timeFrame: Up to 42 days of testing
measure: Plasma half-life (T½)
timeFrame: Up to 42 days of testing
measure: Renal clearance (CLR)
timeFrame: Up to 42 days of testing
measure: Drug excreted unchanged in urine (Amt0-24)
timeFrame: Up to 42 days of testing
measure: Fraction excreted in urine (Fe)
timeFrame: Up to 42 days of testing
Other outcomes:
Locations:
city: San Antonio
country: United States
facility: Worldwide Clinical Trials
state: Texas
Study officials:
affiliation: Edgewise Therapeutics, Inc.
name: Medical Director
role: STUDY_DIRECTOR
References: