About Hunter Syndrome
Hunter syndrome (MPS II) is an X-linked lysosomal storage disorder caused by deficiency of iduronate-2-sulfatase (IDS), leading to accumulation of dermatan sulfate and heparan sulfate. It affects almost exclusively males. The severe form includes progressive neurodegeneration with behavioral problems and intellectual decline, while the attenuated form spares cognitive function. Idursulfase (Elaprase) is approved as intravenous ERT; intrathecal idursulfase beta is approved in Japan for the neuronopathic form.
Common Clinical Features
Clinical Trial Eligibility Tips
What to know before applying to Hunter Syndrome trials.
Distinguish severe (neuronopathic) from attenuated Hunter syndrome — CNS trials target severe form; somatic ERT trials may accept both
IDS enzyme activity in plasma or leukocytes and urinary heparan/dermatan sulfate are required eligibility biomarkers
Idursulfase (Elaprase) IV ERT is standard — intrathecal delivery trials require no prior intrathecal therapy
CNS biomarkers including CSF heparan sulfate and brain MRI findings are key eligibility and outcome measures for neuronopathic trials
Patient Resources
Find recruiting Hunter Syndrome trials
Search 500,000+ studies from ClinicalTrials.gov, filtered for Hunter Syndrome. Updated daily.