Takeda: Mucopolysaccharidosis II trials

The registry lists 19 Mucopolysaccharidosis II studies with Takeda as lead sponsor. 1 are open to participants now and 12 have results posted.

Phase 1 2Phase 2 7Phase 3 5Phase 4 4No phase 8(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.

• Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter SyndromeAvailableNCT05795361
• A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in IndiaCompletedNCT05058391Phase 4Results posted
• Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of AgeCompletedNCT02455622Phase 4Results posted
• Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094CompletedNCT02412787Phase 2/3Results 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
• Collection and Study of Cerebrospinal Fluid in Patients With Hunter SyndromeCompletedNCT01449240Results posted
• Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®CompletedNCT00937794Results 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
• Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)CompletedNCT00069641Phase 2/3Results posted
• Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive ImpairmentActive, not recruitingNCT06031259Phase 2/3
• A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)WithdrawnNCT05494593Phase 4
• A Study to Improve the Awareness of Mucopolysaccharidosis Type II in BrazilCompletedNCT05155488
• Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)CompletedNCT03582449
• Hunter Outcome Survey (HOS)CompletedNCT03292887
• Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)CompletedNCT01822184