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

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

Detailed Description
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This is an open-label, treatment extension study to evaluate the safety, tolerability, and durability of effect in long-term dosing of EDG-5506. EDG-5506-203 MESA will provide continued access to EDG-5506 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.

Official Title
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An Open-Label Extension Study to Assess the Long-term Effect of EDG-5506 on Safety, Biomarkers, and Functional Measures in Adults and Adolescents With Becker Muscular Dystrophy

Conditions
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Becker Muscular Dystrophy

Intervention / Treatment
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* Drug: EDG-5506


Participation Criteria
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Eligibility Criteria
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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: 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 EDG-5506. Receipt of an investigational drug other than EDG-5506 within 30 days or 5 half-lives (whichever is longer) of dosing in the present study. Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months.
Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Treatment<br>Drug: EDG-5506 | Drug: EDG-5506<br>* EDG-5506 is administered orally once per day<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of adverse events in those treated with EDG-5506 |  | 19 Months | 
| Severity of adverse events in those treated with EDG-5506 |  | 19 Months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of treatment-emergent abnormal clinical chemistry laboratory test results |  | 18 Months | 
| Incidence of treatment-emergent abnormal hematology laboratory test results |  | 18 Months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Becker Muscular Dystrophy