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NCT01822184 · ClinicalTrials.gov registry record
Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)
A clinical trial of Hunter Syndrome and Mucopolysaccharidosis (MPS), sponsored by Shire.
- Completed
- Registry status
- 100
- Enrollment target
- 7
- Study locations
NCT01822184 is a study of Hunter Syndrome and Mucopolysaccharidosis (MPS) that has completed, run by Shire. The registered enrollment target is 100 participants, below the 336-participant average among 5 other Hunter Syndrome trials with a reported enrollment target (70% lower). The trial reports 7 study locations across 6 states.
The verdict
NCT01822184, a study of Hunter Syndrome and Mucopolysaccharidosis (MPS), has completed, sponsored by Shire.
- COMPLETED
- Registry status
- 100 participants
- Enrollment target
- 7
- Study locations
Study 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 patients will present with some degree of neurodevelopmental involvement, ranging from severe cognitive impairment and behavioral problems to mildly impaired cognition. This is an observational study; no investigational treatment will be administered. The primary objective of this study is to evaluate the neurodevelopmental status of pediatric patients with MPS II over time and to gain information to guide future treatment studies in this patient population.
Conditions Studied
Study Locations (7)
Other
- Instituto Nacional De Pediatria - Mexico City
- Hospital Infantil Universitario - Madrid
California
- Childrens Hospital & Research Center Oakland - Oakland
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago - Chicago
North Carolina
- University of North Carolina Division of Genetics and Metabolism - Chapel Hill
Buenos Aires
- Hospital Universitario Austral - Pilar
M13 9WL
- Central Manchester University Hospitals NHS Foundation Trust Willink Biochemical Genetics Unit, St. Mary's Hospital - Manchester
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 100 participants |
| Start Date | 2013-01-18 |
| Est. Completion | 2016-10-05 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01822184
The ClinicalTrials.gov registry entry for NCT01822184 describes a study currently listed as completed, categorized as an unspecified phase. The registered enrollment target is 100 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 336-participant average among 5 other Hunter Syndrome trials with a reported enrollment target (70% lower). The listed sponsor is Shire, which has 245 total studies on file at ClinicalTrials.gov.
The record links to 2 conditions, with Hunter Syndrome appearing as the primary indexed condition, and to 0 interventions.
NCT01822184 reports 7 study locations spanning 6 distinct geographic areas - top geographies include Other, California, Illinois.
Frequently Asked Questions
What is clinical trial NCT01822184 about?
NCT01822184 is a clinical study titled "Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)". 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 patients will present with some degree of neurodevelopmental involvement, ranging from severe cognitive im...
What is the current status of trial NCT01822184?
This trial is currently completed. The enrollment target is 100 participants. The study started on 2013-01-18. Estimated completion is 2016-10-05.
What conditions does trial NCT01822184 study?
This clinical trial studies the following conditions: Hunter Syndrome, Mucopolysaccharidosis (MPS).
Who is sponsoring clinical trial NCT01822184?
This trial is sponsored by Shire, which has 245 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01822184 being conducted?
This trial has 7 study locations across California, Illinois, North Carolina, Buenos Aires, M13 9WL. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Related
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