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

Conditions

• MPS III B

Eligibility Criteria

Sex

ALL

Age

N/A - N/A

Healthy Volunteers

Not accepted

Interventions

• AX 250 — DRUG
  biweekly infusion by intracerebroventricular (ICV) infusion

Study Details

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.

Key Dates

Start date

Dec 2, 2022

Status verified

Aug 2023

Primary completion

Oct 31, 2027

Completion

Dec 31, 2027

Study Design

Enrollment

15 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: single arm
  AX 250 300 mg - open label

Primary Outcome Measure

Primary - neurocognition [ Time Frame: baseline to 144 weeks of treatment ]

Locations (2)

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