Indication
Mucopolysaccharidosis II (MPS II)
10 clinical trials
16 products
Clinical trial
An Open-Label, Phase 1 and Extension Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients With MPS Ⅱ Who Have Central Nervous System Involvement and Are Receiving Treatment With Intravenous DrugStatus: Recruiting, Estimated PCD: 2026-06-01
Product
GC1123Clinical trial
Phase 3, Double-blind, Randomized, Active-controlled (Part 1) and Open-labeled, Historical Placebo Controlled (Part 2) Study to Evaluate the Efficacy of Hunterase (Idursulfase-beta) in Hunter Syndrome (Mucopolysaccharidosis II) PatientsStatus: Completed, Estimated PCD: 2022-02-15
Product
ComparatorProduct
GC1111Clinical trial
Post-trial Access Program: Idursulfase-IT (HGT-2310) in Conjunction With Intravenous Elaprase® in Pediatric and Adult Patients With Hunter Syndrome and Cognitive ImpairmentStatus:
Product
Idursulfase-ITProduct
ElapraseClinical trial
An Open-Label Extension for Subjects in Studies HGT-HIT-046 and SHP609-302 Evaluating Long-Term Safety of Intrathecal Idursulfase-IT Administered in Conjunction With Intravenous Elaprase® in Subjects With Hunter Syndrome and Cognitive ImpairmentStatus: Recruiting, Estimated PCD: 2034-01-01
Clinical trial
An Open-Label Extension of Study HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Intrathecal Idursulfase-IT Administered in Conjunction With Intravenous Elaprase® in Pediatric Patients With Hunter Syndrome and Cognitive ImpairmentStatus: Completed, Estimated PCD: 2024-04-30
Clinical trial
An Open Label Extension of Study HGT-HIT-094 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Idursulfase Administered in Conjunction With Elaprase® in Patients With Hunter Syndrome and Cognitive ImpairmentStatus: Completed, Estimated PCD: 2024-04-18
Product
ELAPRASEClinical trial
An Open-label, Multicenter, Phase 4 Study to Assess the Effects of a Prophylactic Immune Tolerizing Regimen in MPS II Treatment-Naïve Patients Planned to Receive ELAPRASE Who Are at Risk of Developing Persistent Neutralizing AntibodiesStatus: Recruiting, Estimated PCD: 2027-06-15
Product
MethotrexateProduct
Intravenous ImmunoglobulinProduct
RituximabClinical trial
A Prospective, Multicenter, Single-arm, Open-label, Interventional Phase IV Study to Evaluate the Safety and Efficacy of Idursulfase (r-DNA Origin) (Elaprase™) in Indian Pediatric and Adult Population With Hunter Syndrome (Mucopolysaccharidosis II)Status: Recruiting, Estimated PCD: 2024-08-31
Clinical trial
A Long-Term, Open-Label, Multicenter, Phase IV Study to Assess Longitudinal Changes on Height and Weight in Patients With MPS II Who Are Receiving Elaprase and Started Treatment With Elaprase at <6 Years of AgeStatus: Active (not recruiting), Estimated PCD: 2025-07-09
Clinical trial
MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATGStatus: Recruiting, Estimated PCD: 2025-07-14
Product
Stem Cell TransplantationProduct
IMD Preparative RegimenProduct
OsteopetrosisProduct
CALD SR-AProduct
cALD SR-BProduct
cALD HR-D