NCT05492799

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

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
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 15 (estimated)
• Sponsor: Allievex Corporation · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Conditions

• MPS III B

Interventions

Type	Name	Description
DRUG	AX 250	biweekly infusion by intracerebroventricular (ICV) infusion

Timeline

• Start date: 2022-12-02
• Primary completion: 2027-10-01
• Completion: 2027-12-01
• First posted: 2022-08-09
• Last updated: 2023-08-09

Locations

6 sites across 5 countries: United States, Colombia, Germany, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study