Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

Study Overview
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Brief Summary
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This is a multicenter, open-label, long-term, extension, phase 2 study to evaluate the safety and efficacy of long-term treatment with DS-5141b in patients with DMD who have completed DS5141-A-J101.

Official Title
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A Phase II, Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

Conditions
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Duchenne Muscular Dystrophy

Intervention / Treatment
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* Drug: DS-5141b


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Has competed a study of DS5141-A-J101 Exclusion Criteria: Significant safety issues in a study of DS5141-A-J101 Patient who does not consent to use appropriate contraception Patient not appropriate to participant in the study as determined by the Investigator

Ages Eligible for Study
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Minimum Age: 5 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
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: DS-5141b 2.0 mg/kg<br>Participants who will receive DS-5141b 2.0 mg/kg once weekly. | Drug: DS-5141b<br>* Administered via subcutaneous injection once weekly<br>* Other names: Renadirsen;|
| Experimental: DS-5141b 6.0 mg/kg<br>Participants who will receive DS-5141b 6.0 mg/kg once weekly. | Drug: DS-5141b<br>* Administered via subcutaneous injection once weekly<br>* Other names: Renadirsen;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Adverse Events (AEs) |  | From first injection to after the last injection of DS-5141b in this study (within approximately 2 years) | 
| Change in distance walked during 6-minute walk test (6MWT) |  | Every 3 months (within approximately 2 years) | 
| Change in time to stand (TTSTAND) |  | Every 3 months (within approximately 2 years) | 
| Change in time in Timed up and go test |  | Every 3 months (within approximately 2 years) | 
| Change in time in 10-meter Run/Walk test |  | Every 3 months (within approximately 2 years) | 
| Change in score in the North Star Ambulatory Assessment (NSAA) |  | Every 3 months (within approximately 2 years) | 
| Change in score in the Performance of Upper Limb (PUL) |  | Every 3 months (within approximately 2 years) | 
| Change in Left Ventricular Ejection Fraction percentage (LVEF %) |  | Every 6 months (within approximately 2 years) | 
| Change in in Forced Vital Capacity (FVC) (percent predicted) |  | Every 6 months (within approximately 2 years) | 
| Change in Muscle Strength Measured by Quantitative Muscle Strength Assessment |  | Every 3 months (within approximately 2 years) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Concentration of DS-5141a in plasma |  | Every 3 months (within approximately 2 years) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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DMD, Duchenne muscular dystrophy, Oligonucleotides, antisense, Exon skipping, DS-5141b