Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)
NCT01822184 · View on ClinicalTrials.gov ↗
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?
Always speak with your doctor before enrolling in a clinical 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. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 100 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Shire, which has 56 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 2 conditions, with Hunter Syndrome appearing as the primary indexed condition, and to 0 interventions. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT01822184 reports 7 study locations spanning 6 distinct geographic areas — top geographies include Other, California, Illinois. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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). These conditions were identified from the trial registry and reflect the primary focus areas of the research.
Who is sponsoring clinical trial NCT01822184?
This trial is sponsored by Shire, which has 56 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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.
Learn More About Clinical Trials
How Clinical Trials Work
Understand phases 1-4, trial design, randomization, and the informed consent process.
Patient Rights in Clinical Trials
Your rights as a participant: consent, withdrawal, privacy, and who to contact.
Finding the Right Clinical Trial
A practical guide to searching trials, understanding eligibility, and evaluating options.
All Guides
Browse our complete library of clinical trial educational resources.
Read our methodology — how this data is sourced, computed, and verified.