Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE
Status:
Not yet recruiting
Trial end date:
2027-12-01
Target enrollment:
Participant gender:
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.