Status:
COMPLETED
Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)
Lead Sponsor:
Shire
Conditions:
Mucopolysaccharidosis (MPS)
Hunter Syndrome
Eligibility:
MALE
2-18 years
Brief Summary
Hunter syndrome (Mucopolysaccharidosis II, \[MPS II\]) is a rare, genetically linked lysosomal storage disease (LSD) caused by deficiency of the enzyme, iduronate-2-sulfatase (I2S). Most MPS II patien...
Eligibility Criteria
Inclusion
- Patients must meet all of the following criteria to be considered eligible for enrollment:
- a. The patient has a deficiency in iduronate-2-sulfatase enzyme activity AND b. The patient has a documented mutation in the iduronate-2-sulfatase gene. OR c. The patient has a normal enzyme activity level of one other sulfatase
- The patient is male, and is at least 2 years of age and less than 18 years of age at the time of informed consent.
- The patient must have sufficient auditory capacity at enrollment, with or without hearing aids, in the Investigator's judgment to complete the required protocol testing, and be compliant with wearing the aids on scheduled study visits.
- The patient, patient's parent(s), or legally authorized guardian(s) has voluntarily signed an Institutional Review Board / Independent Ethics Committee-approved informed consent and/or assent form(s), as applicable.
Exclusion
- Patients who meet any of the following criteria will be excluded from the study.
- The patient has clinically significant non-Hunter syndrome-related CNS involvement or medical or psychiatric comorbidity(ies) which, in the investigator's judgment, may interfere with the accurate administration and interpretation of protocol assessments, affect study data, or confound the integrity of study results.
- The patient has a general conceptual ability score (GCA) or a developmental quotient on the cognitive scale below 55 at Screening.
- The patient is participating in an interventional clinical trial or has participated in an interventional clinical trial within 30 days prior to enrollment; participation in non interventional observational studies is permitted.
Key Trial Info
Start Date :
January 18 2013
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
October 5 2016
Estimated Enrollment :
100 Patients enrolled
Trial Details
Trial ID
NCT01822184
Start Date
January 18 2013
End Date
October 5 2016
Last Update
March 17 2021
Active Locations (7)
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1
Childrens Hospital & Research Center Oakland
Oakland, California, United States, 94609
2
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
3
University of North Carolina Division of Genetics and Metabolism
Chapel Hill, North Carolina, United States, 27514
4
Hospital Universitario Austral
Pilar, Buenos Aires, Argentina, B1629ODT