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NCT01047306 · ClinicalTrials.gov registry record
A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)
A clinical trial of Sanfilippo Syndrome Type A, sponsored by Shire.
- Completed
- Registry status
- 25
- Enrollment target
- 1
- Study location
NCT01047306: Completed study of Sanfilippo Syndrome Type A, sponsored by Shire.
NCT01047306 is a study of Sanfilippo Syndrome Type A that has completed, run by Shire. The registered enrollment target is 25 participants. The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01047306, a study of Sanfilippo Syndrome Type A, has completed, sponsored by Shire.
- COMPLETED
- Registry status
- 25 participants
- Enrollment target
- 1
- Study location
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.
Primary Outcome
Children 1 year-42 months were assessed by the BSID-III; those \>42 months and with a developmental age of \>42 months by the Vineland Adaptive Behavior Scales-II (VABS-II) were evaluated with the KABC-II. For children \>42 months, but \<42 months in developmental age, and those unable to complete at least 3 cognitive KABC-II subtests, the BSID-III was used. The BSID-III is a series of measurements to assess the motor, language, and cognitive development of infants and toddlers and consists of a
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 |
What the finished NCT01047306 record still lists
NCT01047306 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 25 participants, a relatively small participant target.
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.
NCT01047306 reports a single indexed study location in Minnesota.
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.
What interventions are being tested in trial NCT01047306?
The interventions under investigation include: assessment (OTHER).
Who is sponsoring clinical trial NCT01047306?
This trial is sponsored by Shire, which has 245 total clinical trials registered on ClinicalTrials.gov.
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.
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