Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE

MPS III B Clinical Trial

Official title: Open-Label Multicenter Extension Study to Further Evaluate Safety, Tolerability and Efficacy of Intracerebroventricular AX 250 Treatment in Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) Patients

Status Enrolling by invitation

Clinical Trial Summary

This is a Phase 3B/4, multicenter, multinational, open label study to further evaluate intracerebroventricular (ICV) delivered AX 250 treatment in MPS IIIB subjects that complete Study 250-202 for up to an additional 3 years (144 weeks) of treatment with AX 250 administered by ICV infusion every other week. Subjects will be evaluated for neurocognitive function, communication, adaptive behavior, quality of life, imaging characteristics and biochemical markers of disease burden. Safety will be assessed by adverse events, clinical labs, and physical exams.

Clinical Trial Description

n/a

Related Conditions & MeSH terms

• MPS III B
• Mucopolysaccharidosis III

• NCT number: NCT05492799
• Study type: Interventional
• Source: Allievex Corporation
• Status: Enrolling by invitation
• Phase: Phase 4
• Start date: December 2, 2022
• Completion date: December 2027