A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

Mucopolysaccharidosis Type IIIB Clinical Trial

Official title: A Multicenter, Multinational, Extension Study to Evaluate the Long Term Safety and Efficacy of Intracerebroventricular AX 250 in Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)

Status Active, not recruiting

Clinical Trial Summary

The primary objectives of this study are to evaluate the long-term safety and tolerability of AX 250 administered to subjects with MPS IIIB by an implanted ICV reservoir and catheter and to evaluate the impact of long-term AX 250 treatment on cognitive function in patients with MPS IIIB as assessed by developmental quotient (DQ).

Clinical Trial Description

n/a

Related Conditions & MeSH terms

• MPS III B
• Mucopolysaccharidoses
• Mucopolysaccharidosis III
• Mucopolysaccharidosis Type IIIB

• NCT number: NCT03784287
• Study type: Interventional
• Source: Allievex Corporation
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: February 19, 2018
• Completion date: February 2025