Takeda: Lysosomal Storage Diseases trials

The registry lists 10 Lysosomal Storage Diseases studies with Takeda as lead sponsor. 0 are open to participants now and 9 have results posted.

Phase 1 4Phase 2 7Phase 3 2Phase 4 1No phase 2(a study in two phases counts in both)

Studies

Open to participants first, then studies with results posted, then the rest; newest registration first within each.

• Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA DiseaseCompletedNCT02060526Phase 2Results posted
• Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive ImpairmentCompletedNCT02055118Phase 2/3Results posted
• An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive ImpairmentCompletedNCT01506141Phase 1/2Results posted
• Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA)TerminatedNCT01299727Phase 1/2Results posted
• Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA PatientsCompletedNCT01155778Phase 1/2Results posted
• 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®CompletedNCT00920647Phase 1/2Results posted
• An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome PatientsCompletedNCT00882921Results posted
• Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving IdursulfaseCompletedNCT00630747Phase 2/3Results posted
• Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase TherapyCompletedNCT00607386Phase 4Results posted
• Natural History Study of Children With Metachromatic LeukodystrophyTerminatedNCT01963650