NCT05160415

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

A Phase 1b, Open-label Study of the Safety and Pharmacokinetics of EDG-5506 in Adults With Becker Muscular Dystrophy

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Edgewise Therapeutics, Inc. · Industry
• Sex: Male
• Age: 18 Years – 55 Years
• Healthy volunteers: Not accepted

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

Interventions

Type	Name	Description
DRUG	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).

Timeline

• Start date: 2021-12-28
• Primary completion: 2024-03-01
• Completion: 2024-03-01
• First posted: 2021-12-16
• Last updated: 2025-06-24
• Results posted: 2025-06-24

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study