NCT02493998

A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

A Prospective, Observational Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 22 (actual)
• Sponsor: Allievex Corporation · Industry
• Sex: All
• Age: 1 Year – 10 Years
• Healthy volunteers: Not accepted

Summary

Mucopolysaccharidosis type IIIB (MPS IIIB, also known as Sanfilippo Syndrome Type B) is a severe neurodegenerative disorder. The purpose of this study is to learn more about the health problems in patients with MPS IIIB and how to measure these problems over time. It will particularly look at how the disease develops in young children. This is an observational study, so no experimental drug will be given. The results from this study will help us design future studies to measure whether these health problems get better when we give experimental drug for MPS IIIB.

Detailed description

This is a multicenter, multinational, longitudinal, observational study in subjects 1 through 10 years of age who have been diagnosed with MPS IIIB. Data will be prospectively collected from 20 to 30 subjects to understand the clinical progression of MPS IIIB in terms of neurocognitive function, behavior, quality of life, imaging characteristics, genotype, and biochemical markers of disease burden. This information may help inform the design and interpretation of subsequent interventional studies.

Conditions

• Mucopolysaccharidosis Type IIIB
• Mucopolysaccharidosis Type 3 B
• MPS III B
• MPS 3 B

Timeline

• Start date: 2015-11-01
• Primary completion: 2019-04-01
• Completion: 2019-04-01
• First posted: 2015-07-10
• Last updated: 2020-03-06

Locations

8 sites across 8 countries: United States, Australia, Colombia, Germany, Spain, Taiwan, Turkey (Türkiye), United Kingdom