Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With Cardiomyopathy

Sponsor

Alnylam Pharmaceuticals (Industry)

Overall Status

Available

CT.gov ID

NCT05505838

Collaborator

(none)

Study Details

Study Description

Brief Summary

The objective of the study is to assess the long-term safety of patisiran in patients with ATTR amyloidosis with cardiomyopathy as assessed by a review of adverse events (AEs).

Condition or Disease	Intervention/Treatment	Phase
Transthyretin-mediated Amyloidosis With Cardiomyopathy ATTR Amyloidosis With Cardiomyopathy	Drug: Patisiran

Detailed Description

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Study Design

Study Type:

Expanded Access

Official Title:

Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis (ATTR Amyloidosis) With Cardiomyopathy

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 85 Years

Sexes Eligible for Study:

All

Inclusion Criteria:

Documented diagnosis of ATTR amyloidosis with cardiomyopathy, classified as either hATTR amyloidosis with cardiomyopathy or wtATTR amyloidosis with cardiomyopathy; AND
Had an inadequate response to or could not tolerate standard of care, in the opinion of the investigator.
Is not eligible for on-label use of commercial patisiran in the opinion of the investigator.

Exclusion Criteria:

New York Heart Association (NYHA) Class IV
NYHA Class III AND ATTR amyloidosis disease Stage 3 (defined as both N-terminal prohormone B-type natriuretic peptide (NT-proBNP) >3000 ng/L and estimated glomerular filtration rate [eGFR] <45 ml/min/1.73 m^2). [Gillmore 2018]
Current or future participation in another investigational device or drug study, scheduled to occur during this study, or has received an investigational agent or device within 30 days (or 5 half-lives of the investigational drug, whichever is longer) prior to dosing (Day 1). Patients who have previously participated in a gene therapy trial for hATTR amyloidosis.
Patients currently enrolled in, eligible for inclusion in, or who have dropped out of an ongoing interventional (therapeutic) clinical trial related to ATTR amyloidosis.