Studies Open for Recruitment
Duchenne Muscular Dystrophy
Research study
Study on “Risk of cognitive impairment and microstructural brain alteration, and its genetic regulation in Duchenne muscular dystrophy” -- Recruitment for DMD patients in progress
Clinical trial
“A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects with Duchenne Muscular Dystrophy (ENVISION)” -- Recruitment in Progress
The study will enroll individuals with following criteria:
- Has been diagnosed with Duchenne muscular dystrophy, for which he has been on a stable dose of oral corticosteroids for at least 3 months.
- Does not have elevated antibodies to rAAVrh74.
- Group 1: Non-ambulatory male individuals with Duchenne with continuous wheelchair use for at least 6 months. There is no age limit for this group.
- Group 2: Ambulatory male individuals with Duchenne muscular dystrophy, age 8 years to less than 18 years old.
Study site: Hong Kong Children’s Hospital
Principal Investigator: Prof. Chan Hoi Shan Sophelia
Clinical Associate Professor, Paediatric Neurology Division, Department of Paediatrics and Adolescent Medicine, LKS Faculty of Medicine, The University of Hong Kong
Hon. Consultant, Department of Paediatrics and Adolescent Medicine, The Hong Kong Children’s Hospital
For more detail, please refer to the Sarepta Therapeutics website https://clinicaltrials.sarepta.com/ENVISION.
If you want to learn more about the study, please contact Ms. Amanda Mok (Phone: 22554538).