Yükleniyor... / Loading...
This study examines the natural course of the disease over time in patients with mucopolysaccharidosis type II. It enrolled patients with an enzyme-confirmed diagnosis of MPS II who had previously undergone certain neurocognitive assessments. This was a non-phase natural history study and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: 1. Documented diagnosis of MPS II confirmed by enzyme activity as measured in plasma, fibroblasts, or leukocytes 2. The subject has at least one of the neurocognitive assessments listed below, which occurred prior to age 6 and in or after 2006 in their medical records. 1. Bayley Scales of Infant and Toddler Development (BSID), any version 2. Differential Ability Scale (DAS), any version 3. Griffiths Mental Development Scale (GMDS), any version 4. Kaufman Assessment Battery for Children (KABC), any version 5. Kinder Infant Development Scale (KIDS) 6. Kyoto Scale of Psychological Development (KSPD), any version 7. Leiter International Performance Scale (LIPS), any version 8. Mullen Scales of Early Learning (MSEL), any version 9. Vineland Adaptive Behavior Scales (VABS), any version 10. Wechsler Intelligence Scale for Children (WISC), any version 11. Wechsler Preschool and Primary Scale of Intelligence (WPPSI), any version 3. If the subject has undergone hematopoietic stem cell transplantation (HSCT), they must have at least one neurocognitive assessment prior to HSCT.