Patisiran for Amyloidosis
Recruiting in Palo Alto (17 mi)
Age: 18+
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase < 1
Waitlist Available
Sponsor: Austin Neuromuscular Center
No Placebo Group
Breakthrough Therapy
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.
Research Team
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
I am over 18 years old.
You do not have a specific type of protein buildup in your heart, as determined by a biopsy or a specific imaging test.
You do not have hepatitis B or hepatitis C.
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Exclusion Criteria
I haven't had cancer in the last 5 years, except for certain skin cancers or cervical cancer that was treated.
My heart condition is not caused by TTR amyloidosis.
I need medication for my heart or blood pressure, like verapamil or diltiazem.
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Treatment Details
Interventions
- Patisiran (RNAi Therapeutics)
Trial OverviewThe trial is testing the effectiveness and safety of patisiran for patients with wtATTR amyloidosis who are experiencing nerve damage symptoms. It aims to see if patisiran can improve neurological functions and life quality.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: open labelExperimental Treatment1 Intervention
single open arm label
Find a Clinic Near You
Who Is Running the Clinical Trial?
Austin Neuromuscular Center
Lead Sponsor
Trials
2
Recruited
20+
Alnylam Pharmaceuticals
Industry Sponsor
Trials
81
Recruited
16,100+
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