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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the use of patisiran, a type of RNA interference therapy, to treat individuals with wtATTR amyloidosis who experience symptoms of nerve damage, such as tingling or weakness. The primary goal is to determine if patisiran can improve nerve function and overall quality of life. It is open to individuals diagnosed with symptomatic polyneuropathy due to wtATTR amyloidosis, confirmed by specific tests. Participants should not have other causes of nerve issues or certain health conditions, such as hepatitis. As an Early Phase 1 trial, this research focuses on understanding how patisiran works in people, offering participants the opportunity to be among the first to receive this treatment.

Do I need to stop my current medications for the trial?

You may need to stop certain medications before joining the trial. If you are taking diflunisal, doxycycline, or tauroursodeoxycholic acid, you must stop them and wait for a specific period (6 months for diflunisal and 30 days for the others) before starting the trial. Also, you cannot take calcium channel blockers or digitalis during the trial.

Is there any evidence suggesting that patisiran is likely to be safe for humans?

Research has shown that patisiran has been tested for safety in treating different types of amyloidosis. In a 5-year study, patients using patisiran lived longer and managed their disease better, suggesting the treatment is generally safe over a long period.

Another study examined patisiran over 12 months and found that patients maintained their physical abilities, indicating the treatment is well-tolerated for at least a year.

Several studies support the safety of patisiran for amyloidosis patients, with no strong evidence of serious side effects. However, like any treatment, some minor side effects might occur. It's always important to discuss potential risks with the trial team. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Patisiran is unique because it targets the root cause of amyloidosis by using RNA interference (RNAi) technology to silence the production of the TTR protein that causes amyloid deposits. Unlike traditional treatments that manage symptoms, such as tafamidis or diflunisal, patisiran works at the genetic level to prevent further protein buildup. Researchers are excited about its potential to improve outcomes by directly addressing the underlying genetic issues, offering a novel approach that could enhance quality of life for patients with amyloidosis.

What evidence suggests that patisiran might be an effective treatment for amyloidosis?

Research has shown that patisiran offers promising results for treating amyloidosis, a condition where abnormal proteins accumulate in the body. In earlier studies, patients with hereditary amyloidosis who took patisiran experienced better health outcomes and lived longer than those on a placebo. Another study found that patisiran helped patients with heart-related amyloidosis maintain their physical abilities over 12 months. Long-term data also suggest that patisiran can slow the disease's progression and extend patients' lives. These findings support patisiran's potential to manage symptoms and improve the quality of life for people with amyloidosis. ^{1 2 3 6 7}

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

Citations

1. jamanetwork.com jamanetwork.com/journals/jamaneurology/fullarticle/2828606

Five-Year Results With Patisiran for Hereditary ... Patients who received patisiran in their parent study had better outcomes and survival than those receiving placebo.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11894486/

A Randomized Clinical Trial With Open-Label Extension Infusion-related reactions were the most common treatment-related AE (n = 34 [16.1%]). Overall, 41 patients (19.4%) died during the study.

3. nejm.org nejm.org/doi/full/10.1056/NEJMoa2300757

Patisiran Treatment in Patients with Transthyretin Cardiac ... In this trial, administration of patisiran over a period of 12 months resulted in preserved functional capacity in patients with ATTR cardiac amyloidosis.

4. jwatch.org jwatch.org/na58817/2025/08/01/long-term-results-with-patisiran-familial-amyloidosis-with

Long-Term Results with Patisiran in Familial Amyloidosis ... In a 5-year extension study, patisiran was associated with continued attenuation of disease progression, and with improved survival when ...

5. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39804640/

A Randomized Clinical Trial With Open-Label Extension This study presents the longest-term data to date on patisiran for hATTR-PN. Objective: To present the long-term efficacy and safety of ...

6. frontiersin.org frontiersin.org/journals/neurology/articles/10.3389/fneur.2024.1465747/full

Assessing the effectiveness and safety of Patisiran and ... This systematic review recommends using Patisiran and Vutrisiran to treat ATTRv amyloidosis. The findings suggest that these RNA interference therapies improve ...

7. arci.org arci.org/patisiran-attr-cm-crl/

FDA Denies Patisiran for Cardiomyopathy of ATTR ... “The data from the APOLLO-B study demonstrate the safety of ONPATTRO in patients with ATTR cardiac amyloidosis,” says Ahmad Masri, M.D. ...