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RNAi Therapeutics

Patisiran for Amyloidosis

Q: Is there currently an enrollment opportunity for this experiment?

Clinicaltrials.gov reports that this clinical trial, originally posted on August 3rd 2022, is presently accepting participants. The information was most recently updated on September 11th 2023.

Q: Is this experiment a pioneering endeavor in the medical field?

At present, there are 3 ongoing open label studies spread across 21 cities and 27 countries. The first of these trials was conducted in 2019 by Alnylam Pharmaceuticals with 164 participants. Since then, 8 more similar investigations have been completed, including the Phase 3 drug approval stage.

Q: How many research participants are participating in this clinical trial?

Affirmative. Per the data available on clinicaltrials.gov, this medical trial is presently enrolling patients. The original post was made on August 3rd 2022 and it has been modified as recently as September 11th 2023. A total of 10 participants are needed from one site in particular.

Q: What can we learn from the open label studies conducted previously?

Presently, 3 clinical trials are studying open label with 2 of those in Phase 3. While Austin, <a href="https://www.withpower.com/clinical-trials/texas">Texas</a> is one of the cities housing these experiments, there are a total number of 122 sites conducting research regarding this particular topic.

Q: What objectives is the research striving to accomplish?

This trial will assess patient COMPASS 31score over the span of 24 months. Secondary objectives are to evaluate PND Polyneuropathy disability (PND) Score, Karnofsky performance status score, and an optional exploratory nerve/muscle biopsy in order to detect amyloid deposits within skeletal muscle and peripheral nerves. The PND scale is composed of five stages; 0 meaning no impairment through V which indicates confinement to a wheelchair or bedridden state with higher scores indicating worse outcomes. Meanwhile, the Karnofsky Performance Status Scale ranges from 0 (death) up to 100 (no evidence of disease).

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up start of study, and study end at 2 years

Awards & highlights

Study 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.Check my eligibility

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.See study design

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

Select...

I have been diagnosed with nerve damage that causes pain and weakness.

Select...

I do not have any other conditions that cause nerve damage.

Select...

My tests for hATTR are negative.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ start of study, and study end at 2 years

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

COMPASS 31score

Change in Neurological Impairment Score

Norfolk QOL-DN

Secondary outcome measures

EMG -Electromyography

EuroQOL

Karnofsky, performance status score

+3 more

Other outcome measures

Cardiac MRI

Echo with strain

NFL blood draw

+2 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: open labelExperimental Treatment1 Intervention

single open arm label

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

Austin Neuromuscular CenterLead Sponsor

Alnylam PharmaceuticalsIndustry Sponsor

72 Previous Clinical Trials

14,808 Total Patients Enrolled

29 Trials studying Amyloidosis

11,168 Patients Enrolled for Amyloidosis

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is there currently an enrollment opportunity for this experiment?

"Clinicaltrials.gov reports that this clinical trial, originally posted on August 3rd 2022, is presently accepting participants. The information was most recently updated on September 11th 2023."

Answered by AI

Is this experiment a pioneering endeavor in the medical field?

"At present, there are 3 ongoing open label studies spread across 21 cities and 27 countries. The first of these trials was conducted in 2019 by Alnylam Pharmaceuticals with 164 participants. Since then, 8 more similar investigations have been completed, including the Phase 3 drug approval stage."

Answered by AI

How many research participants are participating in this clinical trial?

"Affirmative. Per the data available on clinicaltrials.gov, this medical trial is presently enrolling patients. The original post was made on August 3rd 2022 and it has been modified as recently as September 11th 2023. A total of 10 participants are needed from one site in particular."

Answered by AI

What can we learn from the open label studies conducted previously?

"Presently, 3 clinical trials are studying open label with 2 of those in Phase 3. While Austin, Texas is one of the cities housing these experiments, there are a total number of 122 sites conducting research regarding this particular topic."

Answered by AI

What objectives is the research striving to accomplish?

"This trial will assess patient COMPASS 31score over the span of 24 months. Secondary objectives are to evaluate PND Polyneuropathy disability (PND) Score, Karnofsky performance status score, and an optional exploratory nerve/muscle biopsy in order to detect amyloid deposits within skeletal muscle and peripheral nerves. The PND scale is composed of five stages; 0 meaning no impairment through V which indicates confinement to a wheelchair or bedridden state with higher scores indicating worse outcomes. Meanwhile, the Karnofsky Performance Status Scale ranges from 0 (death) up to 100 (no evidence of disease)."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy

2022. "Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05023889.

All > Amyloidosis