10 Participants Needed

Patisiran for Amyloidosis

LS
YH
EH
Overseen ByEmil Hussain
Age: 18+
Sex: Any
Trial Phase: Phase < 1
Sponsor: Austin Neuromuscular Center
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise

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

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.
See 5 more

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.
See 21 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

4 weeks
1 visit (in-person)

Treatment

Participants receive patisiran administered as an IV infusion once every 21 days for a 24-month period

24 months
Approximately 34 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • Patisiran
Trial Overview The 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

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