Trials / Completed
CompletedNCT01047306
A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)
A Longitudinal, Prospective, Natural History Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)
- Status
- Completed
- Phase
- —
- Study type
- Observational
- Enrollment
- 25 (actual)
- Sponsor
- Shire · Industry
- Sex
- All
- Age
- 1 Year
- Healthy volunteers
- Not accepted
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
Interventions
| Type | Name | Description |
|---|---|---|
| OTHER | assessment | Physical, developmental, neurological, behavioral, and neurocognitive assessments |
Timeline
- Start date
- 2010-02-15
- Primary completion
- 2013-07-10
- Completion
- 2013-07-10
- First posted
- 2010-01-12
- Last updated
- 2021-06-11
- Results posted
- 2016-03-30
Locations
1 site across 1 country: United States
Source: ClinicalTrials.gov record NCT01047306. Inclusion in this directory is not an endorsement.