4 results
The objective of this study is to determine the efficacy, safety, and tolerability of tafamidis in subjects with transthyretin cardiomyopathy.The primary objective is to assess the efficacy, safety, and tolerability of an oral dose of 20 mg or 80 mg…
Primary:• To monitor participants for delayed AEs associated with administration of autologous cells that have been genetically modified by lentiviral vectorsSecondary:• To monitor Replication Competent Lentivirus (RCL).• To measure persistence of…
Primary:To assess the safety, tolerability and determine recommended phase 2 dose (RP2D) of NY-ESO-1 and LAGE-1a specific T cells, alone or in combination with other agents, in HLA-A*02-positive participants with NY-ESO-1 and/or LAGE-1a positive…
To obtain additional, long term, safety data for tafamidis in subjects with transthyretin amyloid cardiomyopathy (ATTR CM). To provide investigational product, tafamidis, to ATTR CM subjects who complete 30 months of blinded treatment on protocol…