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A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation | Clinical Trials | Clareo Health

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A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

A Phase 4 Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of Hereditary Transthyretin-Mediated (ATTRv) Amyloidosis With a V122I or T60A Mutation

NCT04201418•Alnylam Pharmaceuticals

View on ClinicalTrials.gov

Summary

To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Diagnosed with ATTRv amyloidosis with polyneuropathy, with a documented V122I or T60A mutation
•PND score of I-IIIB at baseline.
•Exposure to commercial patisiran in one of the 3 cohorts:
•* Prospective Cohort: Naive to patisiran treatment at the time of enrollment with intention to initiate treatment with patisiran.
•* Mixed cohort: Currently on commercial patisiran therapy for less than 12 months at study enrollment.
•* Retrospective cohort: Exposed to commercial patisiran treatment for at least 12 months prior to study enrollment, regardless of current treatment status at enrollment.

Exclusion Criteria

•New York Heart Association (NYHA) heart failure classification ≥3
•Karnofsky Performance Status (KPS) \<60%
•Unstable congestive heart failure (CHF)
•Known primary amyloidosis (AL) or leptomeningeal amyloidosis
•Prior major organ transplant
•Previously received patisiran
•Previous treatment with a TTR silencing therapy

Locations (27)

Clinical Trial Site

Los Angeles, California, United States

Clinical Trial Site

San Diego, California, United States

Clinical Trial Site

Aurora, Colorado, United States

Clinical Trial Site

Farmington, Connecticut, United States

Clinical Trial Site

Jacksonville, Florida, United States

Clinical Trial Site

Tampa, Florida, United States

Clinical Trial Site

Braselton, Georgia, United States

Clinical Trial Site

Macon, Georgia, United States

Clinical Trial Site

Chicago, Illinois, United States

Clinical Trial Site

Chicago, Illinois, United States

Key Dates

Start Date

December 18, 2019

Primary Completion Date

May 24, 2022

Completion Date

May 24, 2022

Last Updated

June 6, 2022

Enrollment

ACTUAL participants

Conditions

Hereditary Transthyretin-mediated (ATTRv) AmyloidosisPolyneuropathy

Interventions

Patisiran

DRUG

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Chronic Inflammatory Demyelinating Polyneuropathy

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RECRUITINGPHASE3

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: June 6, 2022Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

Inclusion Criteria

Exclusion Criteria

A Study Investigating Intravenous Human Normal Immune Globulin (IGIV) 10% in Subjects With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

A Study to Assess Efficacy and Safety of Empasiprubart Versus IVIg in Adults With CIDP

TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy

IVIG in the Treatment of Autoimmune Small Fiber Neuropathy With TS-HDS, FGFR-3, or Plexin D1 Antibodies

Registry for Adults With Plasma Cell Disorders (PCD's)

Real-world Study of Treatment Outcomes in Chronic Inflammatory Demyelinating Polyneuropathy/Polyradiculoneuropathy (CIDP)