Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)
NCT01047306 · View on ClinicalTrials.gov ↗
Study Summary
The purpose is to evaluate the course of disease progression in MPS IIIA patients who are untreated to identify potential surrogate endpoints that may be utilized in future ERT trials of MPS IIIA via defined assessments including standardized clinical, biochemical, neurocognitive, behavioral, developmental, and imaging measures.
Conditions Studied
Interventions
- OTHER assessment
Study Locations (1)
Minnesota
- University of Minnesota — Minneapolis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 25 participants |
| Start Date | 2010-02-15 |
| Est. Completion | 2013-07-10 |
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 NCT01047306
The ClinicalTrials.gov registry entry for NCT01047306 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 25 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 1 condition, with Sanfilippo Syndrome Type A appearing as the primary indexed condition, and to 1 intervention — of which assessment is the first listed. 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: NCT01047306 reports 1 study location spanning 1 distinct geographic area — top geographies include Minnesota. 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 NCT01047306 about?
NCT01047306 is a clinical study titled "A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)". The purpose is to evaluate the course of disease progression in MPS IIIA patients who are untreated to identify potential surrogate endpoints that may be utilized in future ERT trials of MPS IIIA via defined assessments including standardized clinical, biochemical, neurocognitive, behavioral, develo...
What is the current status of trial NCT01047306?
This trial is currently completed. The enrollment target is 25 participants. The study started on 2010-02-15. Estimated completion is 2013-07-10.
What conditions does trial NCT01047306 study?
This clinical trial studies the following conditions: Sanfilippo Syndrome Type A. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT01047306?
The interventions under investigation include: assessment (OTHER). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT01047306?
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 NCT01047306 being conducted?
This trial has 1 study location across Minnesota. 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.