Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy

EARLY_PHASE1Active, not recruitingINTERVENTIONAL
Enrollment

10

Participants

Timeline

Start Date

August 3, 2022

Primary Completion Date

February 19, 2026

Study Completion Date

February 25, 2026

Conditions
PolyneuropathiesWild Type ATTR AmyloidosisWild-Type Transthyretin-Related (ATTR)AmyloidosisWild-Type Transthyretin Cardiac AmyloidosisTransthyretin Amyloidosis
Interventions
DRUG

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)."

Trial Locations (1)

78759

Austin Neuromuscler Center/National Neuromuscular Research Institute, Austin

Sponsors

Collaborators (1)

All Listed Sponsors
collaborator

Alnylam Pharmaceuticals

INDUSTRY

lead

Austin Neuromuscular Center

OTHER