Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy (MESA)

Edgewise Therapeutics

Status and phase: Invitation-only; Phase 2

Conditions: Becker Muscular Dystrophy

Treatments: Drug: Sevasemten

Study type: Interventional

Funder types: Industry

Identifiers

NCT06066580

EDG-5506-203

Details and patient eligibility

About

EDG-5506-203 MESA is an open-label extension study to assess the long-term effect of sevasemten (EDG-5506) on safety, biomarkers, and functional measures in adults and adolescents with Becker muscular dystrophy

Full description

This is an open-label, treatment extension study to evaluate the safety, tolerability, and durability of effect in long-term dosing of sevasemten. EDG-5506-203 MESA will provide continued access to sevasemten treatment to participants with Becker muscular dystrophy who were previously enrolled in EDG-5506-002 ARCH, completed EDG-5506-201 CANYON and GRAND CANYON, or completed EDG-5506-202 DUNE.

Enrollment: 200 estimated patients

Sex: Male

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Participation in EDG-5506-002 ARCH, EDG-5506-201 CANYON and GRAND CANYON, or EDG-5506-202 DUNE. Participants are eligible if they complete the respective prior study visits as follows:

• EDG-5506-002 ARCH: Complete the final study Visit 27 [Month 24]; or, completion of the ET visit prior to Visit 27 [Month 24]
• EDG-5506-201 CANYON and GRAND CANYON: Complete the final study visit (Cohorts 1, 2, 4, and 5: Visit 12 [Month 12]; Cohort 6: Visit 11 [Month 18])
• EDG-5506-202 DUNE: Complete at least 36 weeks of open-label treatment

Exclusion criteria

1. Any clinically significant changes during or following participation in EDG-5506-002, EDG-5506-201, or EDG-5506-202 that would affect the potential safety of the participant to receive sevasemten.
2. Receipt of an investigational drug other than sevasemten within 30 days or 5 half-lives (whichever is longer) of dosing in the present study.
3. Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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200 participants in 1 patient group

Treatment

Experimental group

Description:

Drug: Sevasemten

Treatment:

Drug: Sevasemten