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NCT01873911 · ClinicalTrials.gov registry record
Neurobehavioral Phenotypes in MPS III
A clinical trial of Sanfilippo Syndrome Type A and Hurler Syndrome, sponsored by University of Minnesota.
- Completed
- Registry status
- 30
- Enrollment target
- 1
- Study location
NCT01873911 is a study of Sanfilippo Syndrome Type A and Hurler Syndrome that has completed, run by University of Minnesota. The registered enrollment target is 30 participants. The trial reports 1 study location across 1 state.
The verdict
NCT01873911, a study of Sanfilippo Syndrome Type A and Hurler Syndrome, has completed, sponsored by University of Minnesota.
- COMPLETED
- Registry status
- 30 participants
- Enrollment target
- 1
- Study location
Study 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 different factors that are at least partially independent. Hypothesis #2a: Children with MPS III will show more hyperlocomotion, fearlessness, asociality and noncompliance than children of similar cognitive ability with MPS I. Hypothesis #2b: These behaviors will become more frequent and/or intensify over time, consistent with the Cleary and Wraith (1993) model. Quantifying them will provide a more empirical framework for staging disease progression. Hypothesis #3: Brain volumetric analysis and diffusion-tensor imaging will reveal abnormalities of frontal and temporal lobe structures that will correlate with externalizing and Klüver-Bucy syndrome-like behaviors, respectively. Hypothesis #4. Loss of cognitive and language function as measures of neurologic decline will directly precede or co-vary with behavioral decline. The primary objective of this study is to identify the behavioral phenotype and its neural basis in MPS III (Sanfilippo syndrome). Is the behavioral phenotype similar to that of Klüver-Bucy syndrome, and is there evidence for amygdala abnormality? The secondary objective of this research study is to develop easily administered, sensitive and specific neurobehavioral and neuroimaging markers to characterize the behavioral phenotype(s) of MPS III; to track their progression; and to delineate their neural substrates. Such markers are critical for identifying the stage of disease for each patient, and to measure treatment outcome. Although we know that severe cognitive decline is one essential characteristic of MPS III, the other highly salient characteristic is a range of abnormal and disruptive behaviors that can include, but go well beyond, childhood noncompliance and oppositionality. These behaviors set Sanfilippo syndrome apart from the other MPS disorder
Conditions Studied
Study Locations (1)
Minnesota
- University of Minnesota - Minneapolis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 30 participants |
| Start Date | 2010-12 |
| Est. Completion | 2014-08 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01873911
The ClinicalTrials.gov registry entry for NCT01873911 describes a study currently listed as completed, categorized as an unspecified phase. The registered enrollment target is 30 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is University of Minnesota, which has 966 total studies on file at ClinicalTrials.gov.
The record links to 3 conditions, with Sanfilippo Syndrome Type A appearing as the primary indexed condition, and to 0 interventions.
NCT01873911 reports 1 study location spanning 1 distinct geographic area - top geographies include Minnesota.
Frequently Asked Questions
What is clinical trial NCT01873911 about?
NCT01873911 is a clinical study titled "Neurobehavioral Phenotypes in MPS III". 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 different factors that are at least partially independent. Hypothesis #2a: Children with MPS III will show...
What is the current status of trial NCT01873911?
This trial is currently completed. The enrollment target is 30 participants. The study started on 2010-12. Estimated completion is 2014-08.
What conditions does trial NCT01873911 study?
This clinical trial studies the following conditions: Sanfilippo Syndrome Type A, Hurler Syndrome, Sanfilippo Syndrome Type B.
Who is sponsoring clinical trial NCT01873911?
This trial is sponsored by University of Minnesota, which has 966 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01873911 being conducted?
This trial has 1 study location across Minnesota. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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