Hunter Syndrome
26
0
3
18
Key Insights
Highlights
Success Rate
90% trial completion (above average)
Published Results
12 trials with published results (46%)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 5/100
7.7%
2 terminated out of 26 trials
90.0%
+3.5% vs benchmark
27%
7 trials in Phase 3/4
67%
12 of 18 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 18 completed trials
Clinical Trials (26)
Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome
Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age
An Extension Study to Assess the Long-term Safety and Efficacy of Hunterase (Idursulfase Beta)
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)
Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment
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 Impairment
Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094
A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India
Hunter Outcome Survey (HOS)
A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ
MPS II Immunophenotyping
A Study of GC1111 in Hunter Syndrom Patients
Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)
Biomarkers for Hunter Syndrome
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®
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®
Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment
Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase
Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome