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

study id #: NCT04433234

condition: Duchenne Muscular Dystrophy

status: Enrolling by invitation

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.

intervention: DS-5141b

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT04433234

last updated: August 14, 2020

start date: July 8, 2020

estimated completion: March 31, 2022

phase of development: Phase 2

size / enrollment: 8

primary outcomes:

• 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 3 months (within approximately 2 years) ]
  
• Change in in Forced Vital Capacity (FVC) (percent predicted) [ Time Frame: Every 3 months (within approximately 2 years) ]
  
• Change in Muscle Strength Measured by Quantitative Muscle Strength Assessment [ Time Frame: Every 3 months (within approximately 2 years) ]

secondary outcomes:

• Concentration of DS-5141a in plasma [ Time Frame: Every 3 months (within approximately 2 years) ]

inclusion criteria:

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

sponsor: Daiichi Sankyo Co., Ltd.

• Japan
  Kobe University Hospital
  
  
• Japan
  National Center of Neurology and Psychiatry