Status:
COMPLETED
Neurobehavioral Phenotypes in MPS III
Lead Sponsor:
University of Minnesota
Collaborating Sponsors:
Rare Diseases Clinical Research Network
National Center for Advancing Translational Sciences (NCATS)
Conditions:
Sanfilippo Syndrome Type A
Sanfilippo Syndrome Type B
Eligibility:
All Genders
2-12 years
Brief Summary
Hypothesis #1: Factor analysis of the revised Sanfilippo Behavior Rating Scale (SBRS) will identify a group of externalizing behaviors and a group of Klüver-Bucy syndrome-like behaviors as two differe...
Detailed Description
Like some other mucopolysaccharidosis (MPS) syndromes, MPS III (Sanfilippo syndrome) is characterized by a severe cognitive decline ending in dementia and death. Unlike other MPS syndromes, earlier-st...
Eligibility Criteria
Inclusion
- Research Subjects: Verified diagnosis of MPS IIIA or MPS IIIB (having proof of either genetic mutation or enzymatic analysis prior to enrollment in this study); must be between the ages of 2 and 12 years; must be able to walk.
- Control group: Verified diagnosis of MPS IH; must have already undergone hematopoietic cell transplantation in the past; must be aged between 2 and 5 years; and must be able to walk without support.
Exclusion
- Participants will be excluded who are unable to cooperate or comply with this study's procedures; in the opinion of the principal investigator, participants who have other severely-limiting co-existing conditions such as severe hearing or visual impairment, will be excluded from this study.
Key Trial Info
Start Date :
December 1 2010
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
August 1 2014
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT01873911
Start Date
December 1 2010
End Date
August 1 2014
Last Update
November 1 2019
Active Locations (1)
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1
University of Minnesota
Minneapolis, Minnesota, United States, 55455