1
Participants
Start Date
March 11, 2022
Primary Completion Date
March 31, 2027
Study Completion Date
March 31, 2027
MELPIDA
A single intrathecal infusion of 10 mL at 1E14 vg/mL for a total dose of 1E15 vg
The Hospital for Sick Children, Toronto
The Hospital for Sick Children
OTHER