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A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)

NCT01047306 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 25

Last updated 2021-06-11

Study results available
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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

OTHER

assessment

Physical, developmental, neurological, behavioral, and neurocognitive assessments

Sponsors & Collaborators

  • Shire

    lead INDUSTRY

Principal Investigators

  • Study Director · Takeda

Eligibility

Min Age
1 Year
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2010-02-15
Primary Completion
2013-07-10
Completion
2013-07-10

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01047306 on ClinicalTrials.gov