Transthyretin-Mediated (ATTR) Amyloidosis with Cardiomyopathy
Conditions
Sponsors
Alnylam Pharmaceuticals, Inc
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Has documented diagnosis of ATTR amyloidosis with cardiomyopathy including those with hereditary ATTR (hATTR) or wild-type ATTR (wATTR) amyloidosis. - Has medical history of heart failure (HF) with at least 1 prior hospitalization for HF or signs and symptoms that require treatment with a diuretic. - Has screening N-terminal prohormone B-type natriuretic peptide (NT-proBNP) >300 ng/L and 600 ng/L and <8500 ng/L. - Patients may be receiving approved TTR stabilizers for ATTR amyloidosis (eg, tafamidis, acoramidis) and may be receiving background therapy for HF at the discretion of the Investigator.
Exclusion criteria
Exclusion criteria: - Has New York Heart Association (NYHA) Class IV HF; or NYHA Class III heart failure AND ATTR Amyloidosis Disease Stage 3. - Has a polyneuropathy disability (PND) Score IIIa, IIIb, or IV. - Has an estimated glomerular filtration rate eGFR of <30 mL/min/1.73m^2 at screening. - Has received prior or currently receiving TTR-lowering therapy.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Composite outcome of all-cause mortality and recurrent CV events (CV hospitalizations and urgent HF visits) | — |
Countries
8 other countries, Argentina, Australia, Austria, Belgium, Brazil, Canada, Chile, China, Colombia, Denmark, France, Germany, Greece, Hong Kong, Hungary, Ireland, Israel, Italy, Japan, Malaysia, Mexico, New Zealand, Norway, Poland, Portugal, Romania, Saudi Arabia, South Korea, The Czech Republic, The Netherlands
Contacts
Public ContactMasanori Ichida
PPD-SNBL K.K.
Outcome results
None listed