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

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.

Timeline

Start

2022-12-02

Primary completion

2027-10

Completion

2027-12

Drugs

Indications