Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy

Phase 1

Recruiting

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

• Registration Number: CTIS2022-500090-13-00
• Lead Sponsor: Edgewise Therapeutics Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2022-07-14
• Last Update Posted: 21 August 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: Male
• Target Recruitment: 136

Inclusion Criteria

1. Adults (aged 18 to 50 years, inclusive, at the time of Screening visit) with a documented dystrophin mutation and phenotype consistent with Becker Muscular Dystrophy (BMD), and history of being ambulatory beyond 16 years of age without steroids; history of being ambulatory beyond 18 years of age with steroids, OR adolescents (12 to 17 years, inclusive) with genetic confirmation of dystrophin mutation and a phenotype consistent with Becker Muscular Dystrophy (BMD) as determined by the Investigator AND EITHER: a. An in-frame mutation in the dystrophin gene (DMD) OR b.If an out-of-frame mutation in the dystrophin gene (DMD), as seen in approximately 10% of BMD individuals, the ability to Stand from Supine <10 seconds, as individuals with DMD are uniformly unable to Stand from Supine in <10 seconds after 10 years of age., 6. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol., 2. Male sex at birth., 3. Able to complete the 100-meter timed test in <200 seconds with or without use of mobility aid devices, 4. Able to perform the North Star Ambulatory Assessment (NSAA) and achieve a score of 5 to 32, inclusive for adults (aged 18-50 years, inclusive at the time of Screening visit) OR a score of =5 for adolescents (aged 12-17 years, inclusive) at the Screening visit, 5. Willing to comply with contraception requirements described in Section 5.3.1 of the protocol.

Exclusion Criteria

1. Medical history or clinically significant physical examination/laboratory result that, in the opinion of the investigator, would render the participant unsuitable for the study., 3. Echocardiogram ejection fraction <40%., 4. A 12-lead electrocardiogram (ECG) that demonstrates clinically relevant abnormalities that may affect participant safety or interpretation of study results., 5. Forced vital capacity (FVC) predicted <60% or using daytime (mechanical or noninvasive) ventilatory support., 13. Participants with a known allergy to sevasemten or its excipients., 14. 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., 6. Moderate or severe renal or hepatic impairment (estimated glomerular filtration rate [eGFR] <60 mL/min/1.73 m2). eGFR will be based on Cystatin C formula [eGFR = 133 x min(Scys/0.8, 1) -0.499 x max (Scys/0.8, 1)-1.328 x 0.996Age]., 7. Positive test for hepatitis C antibody (unless negative hepatitis C virus polymerase chain reaction), hepatitis B surface antigen, or human immunodeficiency virus antibody., 8. 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., 9. Receiving moderate or strong cytochrome P450 CYP3A4 inhibitors or inducers., 10. Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of the screening visit in the present study., 11. Participants who, 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, recent (within the past year) history of substance abuse or dependency 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., 2. History of malignant neoplastic disease (except for adequately treated non-melanomatous skin carcinoma).

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified