2 results
Approved WMOWill not start
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…
Approved WMOCompleted
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…