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Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)

NCT01509768 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 19

Last updated 2021-03-17

No results posted yet for this study

Summary

The purpose of this study is to evaluate the natural course of disease progression in Mucopolysaccharidosis Type III (MPS IIIB) patients who are untreated to identify potential surrogate endpoints that may be utilized in future treatment trials of MPS IIIB using predefined assessments including standardized clinical, biochemical, neurocognitive, developmental, and imaging measures.

Conditions

  • Sanfilippo Syndrome Type B

Sponsors & Collaborators

  • Shire

    lead INDUSTRY

Principal Investigators

  • Study Director · Takeda

Eligibility

Min Age
1 Year
Max Age
10 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-04-09
Primary Completion
2013-10-10
Completion
2013-10-10

Countries

  • United States
  • Germany
  • United Kingdom

Study Locations

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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 NCT01509768 on ClinicalTrials.gov