10
Participants
Start Date
August 3, 2022
Primary Completion Date
February 19, 2026
Study Completion Date
February 25, 2026
patisiran
"Patients will receive 0.3 mg/kg patisiran once every 21 days administered as an IV infusion over 70 minutes (approximately 1 mL/minute for the first 15 minutes followed by approximately 3 mL/minute for the remainder of the infusion) by a controlled infusion device.~All patients in this study will be premedicated prior to dosing with patisiran. Study drug supplied for this study must not be used for any purpose other than the present study and must not be administered to any person not enrolled in the study.~The first dose of study drug (week 1) will be administered under the supervision of site personnel. After the first dose of patisiran, patients should return to the site for patisiran dosing once every 21 days or receive the patisiran infusions at a local infusion center by a healthcare professional trained on the Protocol, administration of premedication, and patisiran infusion. Patient must receive a dose of interventional drug within the dosing window (±3 days)."
Austin Neuromuscler Center/National Neuromuscular Research Institute, Austin
Collaborators (1)
Alnylam Pharmaceuticals
INDUSTRY
Austin Neuromuscular Center
OTHER