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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
8
Administered via subcutaneous injection once weekly
National Center of Neurology and Psychiatry
Tokyo, Kodaira-Shi, Japan
Kobe University Hospital
Hyōgo, Japan
Incidence of Adverse Events (AEs)
Time frame: 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)
Time frame: Every 3 months (within approximately 2 years)
Change in time to stand (TTSTAND)
Time frame: Every 3 months (within approximately 2 years)
Change in time in Timed up and go test
Time frame: Every 3 months (within approximately 2 years)
Change in time in 10-meter Run/Walk test
Time frame: Every 3 months (within approximately 2 years)
Change in score in the North Star Ambulatory Assessment (NSAA)
Time frame: Every 3 months (within approximately 2 years)
Change in score in the Performance of Upper Limb (PUL)
Time frame: Every 3 months (within approximately 2 years)
Change in Left Ventricular Ejection Fraction percentage (LVEF %)
Time frame: Every 6 months (within approximately 2 years)
Change in in Forced Vital Capacity (FVC) (percent predicted)
Time frame: Every 6 months (within approximately 2 years)
Change in Muscle Strength Measured by Quantitative Muscle Strength Assessment
Time frame: Every 3 months (within approximately 2 years)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Concentration of DS-5141a in plasma
Time frame: Every 3 months (within approximately 2 years)