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RNAi Therapeutics

Patisiran for Amyloidosis

Phase < 1
Recruiting
Research Sponsored by Austin Neuromuscular Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Diagnosis of symptomatic polyneuropathy
No history of other secondary causes of neuropathy
Must not have
Other non-TTR cardiomyopathy, hypertensive cardiomyopathy, cardiomyopathy due to valvular heart disease, or cardiomyopathy due to ischemic heart disease
Requires treatment with calcium channel blockers (eg, verapamil, diltiazem) or digitalis
Timeline
Screening 3 weeks
Treatment Varies
Follow Up start of study, and study end at 2 years
Awards & highlights
All Individual Drugs Already Approved
Approved for 5 Other Conditions
No Placebo-Only Group

Summary

This trial will study a drug to help patients with a rare disease affecting nerve function and quality of life.

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
While not specified here, common side effects of drugs like patisiran may include injection site reactions such as pain or swelling; potential liver issues which could be monitored by blood tests; fatigue; rash; nausea; or other infusion-related reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with nerve damage that causes pain and weakness.
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I do not have any other conditions that cause nerve damage.
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My tests for hATTR are negative.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My heart condition is not caused by TTR amyloidosis.
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I need medication for my heart or blood pressure, like verapamil or diltiazem.
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I am currently on diflunisal or stopped it at least 6 months ago.
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I have previously received treatment to lower TTR levels or participated in a gene therapy trial for amyloidosis.
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I am not currently on doxycycline or tauroursodeoxycholic acid, or I have completed a 30-day break from these medications.
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I am not willing to follow the birth control requirements of the study.
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I am HIV positive.
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I have had or am planning to have an organ transplant.
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I am not in another drug study and haven't been for at least 30 days or 5 half-lives of the last investigational drug I took, except for tafamidis.
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I have untreated thyroid issues.
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My kidney function is severely reduced.
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I have had a serious heart rhythm problem before.
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My condition is primary AL amyloidosis.
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My heart condition severely limits my physical activity.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~start of study, and study end at 2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and start of study, and study end at 2 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
COMPASS 31score
Change in Neurological Impairment Score
Norfolk QOL-DN
Secondary study objectives
EMG -Electromyography
EuroQOL
Karnofsky, performance status score
+3 more
Other study objectives
Cardiac MRI
Echo with strain
NFL blood draw
+2 more

Awards & Highlights

All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
Approved for 5 Other Conditions
This treatment demonstrated efficacy for 5 other conditions.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: open labelExperimental Treatment1 Intervention
single open arm label

Find a Location

Who is running the clinical trial?

Alnylam PharmaceuticalsIndustry Sponsor
79 Previous Clinical Trials
16,055 Total Patients Enrolled
Austin Neuromuscular CenterLead Sponsor
1 Previous Clinical Trials
10 Total Patients Enrolled
~3 spots leftby Nov 2025
Unbiased ResultsWe believe in providing patients with all the options.
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