4 results
The primary objective of this study is to assess the efficacy of Ecopipam to reduce self-injurious behaviors (SIB) in adults and children with Lesch-Nyhan Disease (LND) in an outpatient setting. The secondary objectives of this study are to assess…
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…
The primary objective of this study is to evaluate the effects of intrathecal (IT) administration of SHP611 (also known as TAK-611) on the time to loss of locomotion, as indicated by category 5 or higher in the Gross Motor Function Classification in…
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…