NCT03784287

A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

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: Unknown
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: Allievex Corporation · Industry
• Sex: All
• Age: 0 Years – 18 Years
• Healthy volunteers: Not accepted

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).

Conditions

• Mucopolysaccharidosis Type IIIB
• MPS III B

Interventions

Type	Name	Description
DRUG	AX 250	Chimeric fusion of recombinant human alpha-N-acetylglucosaminidase and truncated human insulin-like growth factor 2 (rhNAGLU-IGF2)

Timeline

• Start date: 2018-02-19
• Primary completion: 2025-01-01
• Completion: 2025-02-01
• First posted: 2018-12-21
• Last updated: 2023-01-18

Locations

8 sites across 7 countries: United States, Colombia, Germany, Spain, Taiwan, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study