NCT01509768

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

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

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 19 (actual)
• Sponsor: Shire · Industry
• Sex: All
• Age: 1 Year – 10 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This is a multicenter longitudinal, prospective, natural history study of patients with MPSIIIB designed to identify potential surrogate endpoints that may be utilized in future Enzyme replacement therapy (ERT) trials of MPS IIIB via defined assessments including standardized clinical, biochemical, neurocognitive, development, and imaging measures.

Conditions

• Sanfilippo Syndrome Type B

Timeline

• Start date: 2012-04-09
• Primary completion: 2013-10-10
• Completion: 2013-10-10
• First posted: 2012-01-13
• Last updated: 2021-03-17

Locations

4 sites across 3 countries: United States, Germany, United Kingdom