Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
- Registration Number
- NCT04433234
- Lead Sponsor
- Daiichi Sankyo Co., Ltd.
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- Male
- Target Recruitment
- 8
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description DS-5141b 2.0 mg/kg DS-5141b Participants who will receive DS-5141b 2.0 mg/kg once weekly. DS-5141b 6.0 mg/kg DS-5141b Participants who will receive DS-5141b 6.0 mg/kg once weekly.
- Primary Outcome Measures
Name Time Method 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
Name Time Method Concentration of DS-5141a in plasma Every 3 months (within approximately 2 years)
Trial Locations
- Locations (2)
National Center of Neurology and Psychiatry
🇯🇵Tokyo, Kodaira-Shi, Japan
Kobe University Hospital
🇯🇵Hyōgo, Japan