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

Study Overview
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Brief Summary
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The ARCH study is an open-label, single-center, Phase 1b study of EDG-5506 to assess the safety and pharmacokinetics (PK) of EDG-5506 in adults with Becker muscular dystrophy (BMD). EDG-5506 is an investigational product intended to protect and improve function of dystrophic muscle fibers.

Detailed Description
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This open-label study will evaluate the safety, tolerability, and pharmacokinetics (PK) of EDG-5506 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 will receive EDG-5506. On-site visits will occur approximately monthly for the first 12 months, followed by every 3 months to assess safety and measures of function. This study will have a 24 month treatment period, followed by a 4 week follow-up period.

Official Title
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A Phase 1b, Open-label Study of the Safety and Pharmacokinetics of EDG-5506 in Adults 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: Participants who have completed Study EDG-5506-001. Participants who were not from Study EDG-5506-001 must meet the following: Male sex at birth and aged 18 to 55 years inclusive at time of consent. Documented dystrophin mutation with phenotype consistent with BMD. Ambulatory at Screening (defined as ability to complete 100 meter [m] timed test, with or without assistance). Body weight ≥ 50 kg at the Screening visit. Body mass index (BMI) between 20 and 34 kg/m2 inclusive. Exclusion Criteria: Receipt of oral corticosteroids for >5 days in the previous 6 months at a dose of >5 mg equivalent per day. Lower oral doses or inhaled/intranasal steroids are permitted. Receiving moderate or strong cytochrome P450 CYP3A4 inhibitors or inducers. Participation in any other investigational drug study or use of use of an investigational drug within 30 days or 5 half-lives (whichever is longer) of dosing in the present study. Medical history or other medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior 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. Includes venous access that would be too difficult to facilitate repeated blood sampling.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 55 Years

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>* Daily oral dose<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of AEs in those treated with EDG-5506 |  | 25 Months | 
| Frequency of AEs in those treated with EDG-5506 |  | 25 Months | 
| Severity of AEs in those treated with EDG-5506 |  | 25 Months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of treatment-emergent abnormal clinical chemistry test results |  | 24 Months | 
| Incidence of treatment-emergent abnormal hematology test results |  | 24 Months | 
| Incidence of treatment-emergent abnormal coagulation test results |  | 24 Months | 
| Incidence of treatment-emergent abnormal urinalysis test results |  | 24 Months | 
| Number of participants with changes in clinical chemistry |  | 24 Months | 
| Number of participants with changes in hematology |  | 24 Months | 
| Number of participants with changes in coagulation |  | 24 Months | 
| Number of participants with changes in urinalysis |  | 24 Months | 
| Number of participants with changes in vital signs |  | 24 Months | 
| Number of participants with changes in physical examination |  | 24 Months | 
| Number of participants with changes in ECG PR Interval |  | 24 Months | 
| Number of participants with changes in ECG QRS Interval |  | 24 Months | 
| Number of participants with changes in ECG QT Interval |  | 24 Months | 
| Number of participants with changes in ECG QTc Interval |  | 24 Months | 
| Number of participants with changes in FVC | Assessed by spirometry | 24 Months | 
| Number of participants with changes in FEV1 | As assessed by spirometry | 24 Months | 

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