Table 1.

Demographics of notable transthyretin amyloidosis (ATTR) clinical trials

Drug name Trial name	Number of patients	Route	Main findings and primary outcomes	Percent women participants	Percent Black participants	Countries included
Transthyretin stabilizers
Tafamidis ATTR-ACT	441	Oral	Decreased mortality and cardiovascular-related hospitalizations.	10%	14%	Belgium, Brazil, Canada, Czech Republic, France, Germany, Italy, Japan, Netherlands, Spain, Sweden, UK, USA
AG10 ATTRibute-CM	510	Oral	Results pending: Primary outcomes will be change in 6-minute walk test and the composite of all-cause mortality and cardiovascular hospitalizations.	Recruitment ongoing	Recruitment ongoing	Australia, Belgium, Brazil, Canada, Greece, Hungary, Ireland, Israel, Italy, Korea, Netherlands, New Zealand, Poland, Portugal, Spain, USA
Transthyretin silencers
Patisiran APOLLO	225 (with neuropathy ± cardiomyopathy)	IV	Vastly improved neurological function vs. placebo. Subanalysis showed decreased LV wall thickness, decreased NT-ProBNP, and improved global longitudinal strain.	26%	2%	Argentina, Brazil, Canada, Bulgaria, Cyprus, France, Italy, Japan, Mexico, Netherlands, Portugal, South Korea, Spain, Sweden, Taiwan, Turkey, UK, USA
Inotersen NEURO-TTR	172 (with neuropathy ± cardiomyopathy)	SQ	Greatly improved neurological function vs. placebo. No significant difference in cardiac parameters.	31%	4%.	Argentina, Brazil, France, Germany, Italy, New Zealand, Portugal, UK, USA
Patisiran APOLLO-B	300	IV	Recruitment ongoing: primary outcome will be change from baseline at month 12 in Six-Minute Walk Test	Recruitment ongoing	Recruitment ongoing	Argentina, Australia, Brazil, Czechia, Denmark, France, Hong Kong, Italy, Japan, Republic of Korea, Mexico, Poland, Portugal, Sweden, Taiwan, UK, USA
Vutrisiran HELIOS-B	600	SQ	Recruitment ongoing: primary outcome will be the composite of all-cause mortality and cardiovascular-related hospitalizations.	Recruitment ongoing	Recruitment ongoing	Australia, France, Germany, Israel, Republic of Korea, Latvia, Lithuania, Norway, Portugal, Spain, Sweden, UK, USA
AKCEA-TTR-LRx CARDIO-TTRansform	750	SQ	Recruitment ongoing: The primary outcome will be the change in 6-minute walk test and the composite of cardiovascular mortality and clinical cardiovascular events.	Pending	Pending	Australia, Belgium, Brazil, Greece, Israel, Italy, Portugal, Spain, Sweden, USA
Amyloid clearance
PRX004	36	IV	Results pending: maximum tolerated dose and treatment-emergent adverse events	Pending	Phase I trial	Portugal, Spain, Sweden, USA

ATTR transthyretin amyloidosis, ATTRv variant ATTR, ATTRwt wild-type ATTR, IV intravenous, SQ subcutaneous, TTR transthyretin