Patisiran for Amyloidosis
Trial Summary
What is the purpose of this trial?
To evaluate the efficacy and safety of patisiran in patients with wtATTR amyloidosis and symptomatic polyneuropathy by evaluating the effect on neurologic impairment and quality of life.
Eligibility Criteria
Adults over 18 with wild-type ATTR amyloidosis and symptomatic polyneuropathy can join this trial. They must have a specific protein level, no hepatitis B or C, proper organ function, and no other neuropathy causes. Exclusions include recent cancer (except certain skin/cervical), uncontrolled thyroid issues or blood pressure, heart/liver transplants, drug abuse history, HIV infection, severe kidney disease, and certain medication use.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive patisiran administered as an IV infusion once every 21 days for a 24-month period
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Patisiran
Find a Clinic Near You
Who Is Running the Clinical Trial?
Austin Neuromuscular Center
Lead Sponsor
Alnylam Pharmaceuticals
Industry Sponsor
Dr. Yvonne Greenstreet
Alnylam Pharmaceuticals
Chief Executive Officer since 2021
MD from the University of Leeds, MBA from INSEAD
Dr. Pushkal Garg
Alnylam Pharmaceuticals
Chief Medical Officer since 2016
MD from Columbia University