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Low Enrollment
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The purpose of this study is to investigate how participant's body's immune system responds to idursulfase, an enzyme replacement therapy (ERT) and find out which types of immune cells are involved in causing untoward responses to the ERT so that the investigators can relate the level of immune response to the treatment.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment Naive | Subjects who are naïve to ERT and start treatment with idursulfase | ||
| Treatment less than 3 years | Subjects who have received ERT for <3 years | ||
| Treatment over 3 years | Subjects who have received ERT for 3 or more years |
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| Measure | Description | Time Frame |
|---|---|---|
| Changes in the frequency of natural killer cells measured by flow cytometry | baseline, 1, 3, 6 months | |
| Changes in level of circulation memory B cells measured by flow cytometry | baseline, 1, 3, 6 months | |
| Changes in level of T follicular helper cells measured by flow cytometry | baseline, 1, 3, 6 months | |
| Changes in distribution of helper T cells measured by flow cytometry | baseline, 1, 3, 6 months |
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Inclusion Criteria:
Exclusion Criteria:
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Metabolic or Genetics Clinic patients
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| Name | Affiliation | Role |
|---|---|---|
| Priya Kishnani, MD | Duke University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Duke University | Durham | North Carolina | 27710 | United States |
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| ID | Term |
|---|---|
| D013398 | Sudden Infant Death |
| D016532 | Mucopolysaccharidosis II |
| ID | Term |
|---|---|
| D003645 | Death, Sudden |
| D003643 | Death |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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blood samples
| D066088 |
| Infant Death |
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |