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

Study Status: Completed
Recruit Status: Completed
Study Type: Observational

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

Brief 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 Summary: 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.
Sponsor: Shire

Current Primary Outcome: evaluate the course of disease progression in patients with MPS IIIB who are untreated with any investigational products to inform possible future treatment studies [ Time Frame: 13 months ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Shire

Dates:
Date Received: January 10, 2012
Date Started: March 2012
Date Completion:
Last Updated: November 24, 2014
Last Verified: November 2014