Open-Label Extension of EDG-5506 in Participants with Becker Muscular Dystrophy

Phase 1

Recruiting

Conditions: Becker Muscular Dystrophy
MedDRA version: 20.0Level: PTClassification code: 10059117Term: Becker's muscular dystrophy Class: 100000004850
Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: CTIS2023-506231-15-00

Lead Sponsor: Edgewise Therapeutics Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: Male

Target Recruitment: 200

Inclusion Criteria

1. Males with a diagnosis of BMD and participation in EDG-5506-002, EDG-5506-201, or EDG-5506-202 (Note: Adolescents will be enrolled in countries with regulatory approval to enroll individuals in that age range). Participants are eligible if they complete the respective prior study visits as follows: - EDG-5506-002: Complete the final study Visit 27 [Month 24]; or, completion of the ET visit prior to Visit 27 [Month 24] - EDG-5506-201: Complete the final study visit (Cohorts 1, 2, 4, and 5: Visit 12 [Month 12]; Cohort 6: Visit 11 [Month 18]) - EDG-5506-202: Complete at least 36 weeks of open-label treatment (Visit 14 [Week 52]), 2.Willing to comply with contraception requirements described in the protocol (Section 5.3.1.), 3. Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.

Exclusion Criteria

1. Medical Conditions: 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. Prior/Concomitant Therapy: Receiving moderate or strong cytochrome P450 CYP3A4 inhibitors or inducers., 3. Prior/Concomitant Therapy: Receipt of an investigational drug other than sevasemten within 30 days or 5 half-lives (whichever is longer) of dosing in the present study., 4. Prior/Concomitant Therapy: Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months. - Receipt of low dose (=5 mg equivalent per day) oral corticosteroids for indications other than BMD or receipt for a short duration (=10 days in previous 6 months), along with receipt of chronic inhaled/intranasal steroids, is permitted., 5. Other Exclusions: 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., 6. Other Exclusions: Participants with a known allergy to sevasemten or its excipients., 7. Other Exclusions: Participants who are submitted to an institution by virtue of an order of a court or government authority to comply with Section 40a No. 2 of the German Medical Products Act.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the safety and tolerability of long-term treatment of sevasemten;Secondary Objective: To assess the change in individual safety parameters;Primary end point(s): 1. Incidence, frequency, and severity of AEs and SAEs in those treated with sevasemten

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s):1. Incidence of treatment-emergent abnormal laboratory test results (clinical chemistry, hematology);Secondary end point(s):2. Change from Baseline in: - Safety labs - Vital Signs - Physical and neurological examination - ECG parameters - Cardiac function as assessed by an echocardiogram - Pulmonary function as assessed by FEV1, FVC - C-SSRS