Patisiran for Amyloidosis

Overseen By Emil Hussain

Age: 18+

Sex: Any

Trial Phase: Phase < 1

Sponsor: Austin Neuromuscular Center

Must not be taking: Diflunisal, Doxycycline, Calcium channel blockers

Disqualifiers: HIV, NYHA Class IV, eGFR < 30, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: 35

Duration: 24 months

10 Participants Needed

What You Need to Know Before You Apply

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.

Are You a Good Fit for This Trial?

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.

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.

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

Group I: open label Experimental Treatment 1 Intervention

single open arm label

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