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

NCT ID: NCT04585464

Last Updated: 2022-06-28

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 127 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-10-12
• Study Completion Date: 2021-12-27

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.

Conditions

Healthy Volunteer; Becker Muscular Dystrophy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Healthy Volunteer: Single Ascending Dose

Single oral ascending dose in healthy volunteers

Interventions:

Drug: EDG-5506 Drug: Placebo

Group Type: EXPERIMENTAL

EDG-5506

Intervention Type: DRUG

EDG-5506 is administered orally as a single dose or once daily

Placebo

Intervention Type: DRUG

Placebo is administered orally as a single dose or once daily

Healthy Volunteer: Multiple Ascending Dose

Multiple oral ascending doses in healthy volunteers

Interventions:

Drug: EDG-5506 Drug: Placebo

Group Type: EXPERIMENTAL

EDG-5506

Intervention Type: DRUG

EDG-5506 is administered orally as a single dose or once daily

Placebo

Intervention Type: DRUG

Placebo is administered orally as a single dose or once daily

Becker Muscular Dystrophy: Multiple Ascending Dose

Multiple oral ascending doses in adults with Becker muscular dystrophy

Interventions:

Drug: EDG-5506 Drug: Placebo

Group Type: EXPERIMENTAL

EDG-5506

Intervention Type: DRUG

EDG-5506 is administered orally as a single dose or once daily

Placebo

Intervention Type: DRUG

Placebo is administered orally as a single dose or once daily

Interventions

EDG-5506

EDG-5506 is administered orally as a single dose or once daily

Intervention Type: DRUG

Placebo

Placebo is administered orally as a single dose or once daily

Intervention Type: DRUG

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 55 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Worldwide Clinical Trials

OTHER

Sponsor Role: collaborator

Edgewise Therapeutics, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Director

Role: STUDY_DIRECTOR

Edgewise Therapeutics, Inc.

Locations

Worldwide Clinical Trials

San Antonio, Texas, United States

Countries

United States

References

Donovan J, Silverman JA, Barthel B, DuVall M, Madden M, MacDougall J, Kilburn NR, Bronson A, Evanchik M, Gordon G, Koch K, Russell AJ. A Phase 1, Double-Blind, Placebo-Controlled Trial of Sevasemten (EDG-5506), a Selective Modulator of Fast Skeletal Muscle Contraction, in Healthy Volunteers and Adults With Becker Muscular Dystrophy. Muscle Nerve. 2025 Sep;72(3):399-407. doi: 10.1002/mus.28444. Epub 2025 Jun 2.

Reference Type: DERIVED

PMID: 40452637 (View on PubMed)

Related Links

http://www.edgewisetx.com

Sponsor Website

Other Identifiers

EDG-5506-001

Identifier Type: -

Identifier Source: org_study_id