A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy

NCT ID: NCT05160415

Last Updated: 2025-06-24

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-12-28
• Study Completion Date: 2024-03-01

Brief Summary

The ARCH study was an open-label, single-center, Phase 1b study of sevasemtem (EDG-5506) to assess the safety and pharmacokinetics (PK) of sevasemten in adults with Becker muscular dystrophy (BMD).

Sevasemten is an investigational product intended to protect and improve function of dystrophic muscle fibers.

Detailed Description

This open-label study evaluated the safety, tolerability, and pharmacokinetics (PK) of sevasemten in participants with BMD who completed the first-in-human study, EDG-5506-001, as well as additional (treatment-naïve) participants from outside the EDG-5506-001 study to meet the target sample size.

All participants received sevasemten. This study had a 24 month treatment period, followed by an optional 4 week follow-up period. On-site visits occurred approximately monthly for the first 12 months, followed by every 3 months to assess safety and measures of function.

Conditions

Becker Muscular Dystrophy

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treatment

Drug: Sevasemten

Group Type: EXPERIMENTAL

Sevasemten

Intervention Type: DRUG

Daily oral dose of 10 mg daily until Visit 8 (Day 57), followed by 15 mg daily until Visit 13 (Month 6), followed by 20 mg until Visit 21 (Month 15), followed by 10 mg daily to Visit 27 (Month 24).

Interventions

Sevasemten

Daily oral dose of 10 mg daily until Visit 8 (Day 57), followed by 15 mg daily until Visit 13 (Month 6), followed by 20 mg until Visit 21 (Month 15), followed by 10 mg daily to Visit 27 (Month 24).

Intervention Type: DRUG

Other Intervention Names

EDG-5506

Eligibility Criteria

Inclusion Criteria

1. Participants who have completed Study EDG-5506-001.

2. Participants who were not from Study EDG-5506-001 must meet the following:

1. Male sex at birth and aged 18 to 55 years inclusive at time of consent.

2. Documented dystrophin mutation with phenotype consistent with BMD.

3. Ambulatory at Screening (defined as ability to complete 100 meter [m] timed test, with or without assistance).

4. Body weight ≥ 50 kg at the Screening visit.

5. Body mass index (BMI) between 20 and 34 kg/m2 inclusive.

3. Female sexual partners of male participants must use highly effective contraception (<1% failure rate per year) through 6 months after last dose.

4. Capable of giving signed informed consent.

Exclusion Criteria

1. Any clinically significant changes during or following the completion of Study EDG 5506-001 that would affect the potential safety of the participant to receive EDG 5506.

2. Cardiac echocardiogram ejection fraction <45% or New York Heart Association (NYHA) Class III or Class IV.

3. Baseline 12-lead electrocardiogram (ECG) that demonstrates clinically relevant abnormalities that may affect participant safety or interpretation of study results.

4. Forced vital capacity (FVC) predicted <65% or using daytime (mechanical or noninvasive) ventilatory support.

5. Moderate or severe renal or hepatic impairment (eGFR <60 mL/min/1.73 m2).

6. Positive test for hepatitis C antibody (unless negative HCV PCR), hepatitis B surface antigen, or human immunodeficiency virus (HIV) antibody at screening.

7. History of substance abuse or dependency.

8. Receipt of oral corticosteroids for >5 days in the previous 6 months at a dose of >5 mg equivalent per day. Lower oral doses or inhaled/intranasal steroids are permitted.

9. Receiving moderate or strong cytochrome P450 CYP3A4 inhibitors or inducers.

10. Participation in any other investigational drug study or use of use of an investigational drug within 30 days or 5 half-lives (whichever is longer) of dosing in the present study.

11. Participants who are unlikely to comply with the study protocol or, in the opinion of the Investigator, would not be a suitable candidate for participation in the study.

12. Medical history or other medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior or laboratory result or abnormality that may increase the risk of study participation or, in the Investigator's judgment, make the participant inappropriate for the study. Includes venous access that would be too difficult to facilitate repeated blood sampling.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 55 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Medpace, Inc.

INDUSTRY

Sponsor Role: collaborator

Edgewise Therapeutics, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Rare Disease Research

Atlanta, Georgia, United States

Countries

United States

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

http://www.edgewisetx.com

Sponsor Website

Other Identifiers

EDG-5506-002

Identifier Type: -

Identifier Source: org_study_id