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

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

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).

Study Overview

• Status: Completed
• Conditions: Becker Muscular Dystrophy; Healthy Volunteer
• Intervention / Treatment: Drug: EDG-5506; Drug: Placebo

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.

• Study Type: Interventional
• Enrollment (Actual): 127
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Texas
    • San Antonio, Texas, United States, 78217
      • Worldwide Clinical Trials

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 55 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Healthy Volunteer: Single Ascending Dose; Single oral ascending dose in healthy volunteers Interventions: Drug: EDG-5506 Drug: Placebo	Drug: EDG-5506; EDG-5506 is administered orally as a single dose or once daily; Drug: Placebo; Placebo is administered orally as a single dose or once daily
Experimental: Healthy Volunteer: Multiple Ascending Dose; Multiple oral ascending doses in healthy volunteers Interventions: Drug: EDG-5506 Drug: Placebo	Drug: EDG-5506; EDG-5506 is administered orally as a single dose or once daily; Drug: Placebo; Placebo is administered orally as a single dose or once daily
Experimental: Becker Muscular Dystrophy: Multiple Ascending Dose; Multiple oral ascending doses in adults with Becker muscular dystrophy Interventions: Drug: EDG-5506 Drug: Placebo	Drug: EDG-5506; EDG-5506 is administered orally as a single dose or once daily; Drug: Placebo; Placebo is administered orally as a single dose or once daily

What is the study measuring?

Primary Outcome Measures

Outcome 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 Outcome Measures

Outcome Measure	Time Frame
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
Renal clearance (CLR)	Up to 42 days of testing
Drug excreted unchanged in urine (Amt0-24)	Up to 42 days of testing
Fraction excreted in urine (Fe)	Up to 42 days of testing

Collaborators and Investigators

• Sponsor: Edgewise Therapeutics, Inc.
• Collaborators: Worldwide Clinical Trials
• Investigators: Study Director: Medical Director, Edgewise Therapeutics, Inc.

Publications and helpful links

Helpful Links

• Sponsor Website

Study record dates

Study Major Dates

• Study Start (Actual): October 12, 2020
• Primary Completion (Actual): November 15, 2021
• Study Completion (Actual): December 27, 2021

Study Registration Dates

• First Submitted: October 2, 2020
• First Submitted That Met QC Criteria: October 7, 2020
• First Posted (Actual): October 14, 2020

Study Record Updates

• Last Update Posted (Actual): June 28, 2022
• Last Update Submitted That Met QC Criteria: June 27, 2022
• Last Verified: June 1, 2022

More Information

Terms related to this study

• Keywords: Healthy Volunteers; Becker Muscular Dystrophy
• Additional Relevant MeSH Terms: Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Muscular Disorders, Atrophic; Muscular Dystrophies; Muscular Dystrophy, Duchenne
• Other Study ID Numbers: EDG-5506-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No