A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults

Healthy Volunteer Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04585464
• Other study ID #: EDG-5506-001
• Status: Completed
• Phase: Phase 1
• Start date: October 12, 2020
• Est. completion date: December 27, 2021

Study information

• Verified date: June 2022
• Source: Edgewise Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 127
• Est. completion date: December 27, 2021
• Est. primary completion date: November 15, 2021
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 55 Years

Eligibility

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.

Related Conditions & MeSH terms

• Becker Muscular Dystrophy
• Healthy Volunteer
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• EDG-5506
EDG-5506 is administered orally as a single dose or once daily
• Placebo
Placebo is administered orally as a single dose or once daily

Locations

Country	Name	City	State
United States	Worldwide Clinical Trials	San Antonio	Texas

Sponsors (2)

Lead Sponsor	Collaborator
Edgewise Therapeutics, Inc.	Worldwide Clinical Trials

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence, frequency, severity and dose-relationship of adverse events		Up to 42 days of monitoring
Primary	Incidence of abnormal laboratory test results (clinical chemistry, hematology, urinalysis, coagulation)		Up to 42 days of monitoring
Primary	Incidence of treatment-emergent clinically abnormal electrocardiogram (ECG)		Up to 42 days of monitoring
Primary	Incidence of abnormal vital signs		Up to 42 days of monitoring
Primary	Incidence of abnormal physical exam findings		Up to 42 days of monitoring
Secondary	Plasma maximum measured drug concentration (Cmax)		Up to 42 days of testing
Secondary	Time of maximum concentration (Tmax)		Up to 42 days of testing
Secondary	Area under the concentration-time curve (AUC)		Up to 42 days of testing
Secondary	Plasma half-life (T½)		Up to 42 days of testing
Secondary	Renal clearance (CLR)		Up to 42 days of testing
Secondary	Drug excreted unchanged in urine (Amt0-24)		Up to 42 days of testing
Secondary	Fraction excreted in urine (Fe)		Up to 42 days of testing

External Links

•   Sponsor Website