At a glance
ClinicalIndex Comparison RecordN/ACompleted· 25 enrolled
Drug / intervention
assessmentother
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Longitudinal, Prospective, Natural History Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)
In Brief
An observational study evaluating assessment for Sanfilippo Syndrome Type A. Completed, enrolled 25 participants across 1 site.
Detailed 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.
Study Details
Study Typeobservational
Allocation--
Masking--
Primary Purpose--
ConditionsSanfilippo Syndrome Type A
CountriesUnited States
Collaborators--
Timeline
N/ACompletedFinished
20102011201220132014201520162017201820192020202120222023202420252026
First PostedJan 2010
Enrollment StartFeb 2010
Primary CompletionJul 2013
TodayJul 2026
First PostedJan 12, 2010
Enrollment StartFeb 15, 2010
Primary CompletionJul 10, 2013
TodayJul 2, 2026
Enrollment to primary: 3.4 yearsPosted 16.5 years ago
Interventions
assessmentother
Physical, developmental, neurological, behavioral, and neurocognitive assessments