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

Alnylam Pharmaceuticals

Status

Conditions: ATTR Amyloidosis With Cardiomyopathy; Transthyretin-mediated Amyloidosis With Cardiomyopathy

Treatments: Drug: Patisiran

Study type: Expanded Access

Funder types: Industry

Identifiers

NCT05505838

ALN-TTR02-014

Details and patient eligibility

About

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).

Full description

Choosing to participate in an expanded access program is an important personal decision. Talk with your doctor and family members or friends about deciding to join a research study. To learn more about this study, please have your doctor contact the study research staff using the Contacts provided.

Sex: All

Ages: 18 to 85 years old

Volunteers: No Healthy Volunteers

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.