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

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

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

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See also
Status	Clinical Trial	Phase
Completed	`NCT02493998` - A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
Active, not recruiting	`NCT03227042` - A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
Completed	`NCT02037880` - Natural History Studies of Mucopolysaccharidosis III	N/A
Completed	`NCT02754076` - A Treatment Study of Mucopolysaccharidosis Type IIIB	Phase 1/Phase 2