Clinical trial NCT05505838: Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With Cardiomyopathy
Official title: Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis (ATTR Amyloidosis) With Cardiomyopathy
Study type: EXPANDED_ACCESS
Overall status: NO_LONGER_AVAILABLE
Conditions: Transthyretin-mediated Amyloidosis With Cardiomyopathy
ATTR Amyloidosis With Cardiomyopathy
Keywords: RNAi therapeutic
Amyloidosis
Cardiomyopathy
TTR
Transthyretin
Lead sponsor: Alnylam Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2022-08-18
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT

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).

Detailed 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.

Eligibility criteria: 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.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Mayo Clinic Hospital
state: Arizona
city: Los Angeles
country: United States
facility: Cedars-Sinai Medical Center
state: California
city: San Diego
country: United States
facility: University of California San Diego
state: California
city: Jacksonville
country: United States
facility: Mayo Clinic Florida
state: Florida
city: Chicago
country: United States
facility: University of Chicago
state: Illinois
city: Evanston
country: United States
facility: NorthShore University
state: Illinois
city: Indianapolis
country: United States
facility: Indiana University Health Hospital
state: Indiana
city: Kansas City
country: United States
facility: The University of Kansas Medical Center
state: Kansas
city: Baltimore
country: United States
facility: The Johns Hopkins University
state: Maryland
city: Boston
country: United States
facility: Boston University
state: Massachusetts
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University
state: Missouri
city: Omaha
country: United States
facility: University of Nebraska Medical Center
state: Nebraska
city: New York
country: United States
facility: Columbia University Irving Medical Center
state: New York
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Columbus
country: United States
facility: OhioHealth Research Institute
state: Ohio
city: Philadelphia
country: United States
facility: Penn Presbyerian Medical Center
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt University
state: Tennessee
city: Dallas
country: United States
facility: Baylor Scott & White Research Institute
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
Study officials:
affiliation: Alnylam Pharmaceuticals
name: Medical Director
role: STUDY_DIRECTOR
References: