A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®
PHASE1
Elaprase (idursulfase), a large molecular protein, is not expected to cross...
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
PHASE2
This was an open-label, repeat-dose, intra-participant dose-escalation stud...
Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment
PHASE2
Study HGT-HIT-094 is a multicenter study designed to determine the effect o...
Status
Completed
Location
Children's Hospital and Research Center at Oakland