Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome (NCT01449240) | Clinical Trial Compass
CompletedNot Applicable
Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome
United States10 participantsStarted 2012-11-12
Plain-language summary
The purpose of the study is to collect data on CSF biomarkers in patients with Hunter Syndrome that would serve as reference data for comparison with cognitively impaired patients with Hunter syndrome, patients with other lysosomal storage diseases, or other diseases with CNS involvement.
Who can participate
Age range70 Years
SexMALE
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Inclusion Criteria:
* The patient is male and has a documented diagnosis of Hunter syndrome (MPSII).
* The adult patient has completed a cognitive assessment at screening/baseline or within the previous 3 months and has been determined to have an intelligence quotient (IQ) ≥78. Note: cognitive evaluation of pediatric patients is not required.
* The adult patient or the adult patient's legally authorized representative(s) has voluntarily signed an Institutional Review Board/Independent Ethics Committee-approved informed consent form after all relevant aspects of the study have been explained and discussed.
* The pediatric patient must be scheduled to undergo a non-study related lumbar puncture or other medical or diagnostic procedure that requires the administration of general anesthesia. The pediatric patient's parent(s) or legally authorized representative(s) must have provided written informed consent (with patient assent as relevant), after all relevant aspects of the study have been explained and discussed, to allow CSF sample collection for this study in conjunction with performance of the non-study related procedure requiring general anesthesia.
Exclusion Criteria:
* The patient has a history of complications from a previous lumbar puncture(s) or technical challenges in conducting lumbar puncture.
* The patient has received a hematopoietic stem cell transplant.
* The patient has taken aspirin, non-steroidal anti-inflammatory drugs (NSAIDs), or other over-the-counter …