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Enzyme Replacement Therapy

AX 250 for Sanfilippo Syndrome Type B

Phase 4
Waitlist Available
Research Sponsored by Allievex Corporation
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
If female with childbearing potential, must have a negative pregnancy test at the Screening visit and be willing to have additional pregnancy tests during the study
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to 144 weeks of treatment
Awards & highlights

Study Summary

This trial will test a treatment for MPS IIIB that has been completed for up to 3 years in order to assess its effectiveness and safety.

Who is the study for?
This trial is for individuals with Mucopolysaccharidosis (MPS) IIIB, also known as Sanfilippo Syndrome, who have completed the Study 250-202. Participants must be able to follow the study plan and females of childbearing age should test negative for pregnancy. Those with very low cognitive scores or no improvement in previous studies, recent other treatments or investigational drugs, severe medical conditions, or risk from brain infusions cannot join.Check my eligibility
What is being tested?
The trial tests AX 250 delivered directly into the brain fluid every two weeks for up to three years. It aims to see how well it works on neurocognitive function and quality of life in MPS IIIB patients while monitoring safety through adverse events and regular health checks.See study design
What are the potential side effects?
While specific side effects are not listed here, participants will be monitored for any adverse reactions related to AX 250 treatment which could include issues at the infusion site in the brain or general drug-related side effects.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I am not pregnant and agree to regular pregnancy tests during the study.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to 144 weeks of treatment
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to 144 weeks of treatment for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Primary - neurocognition

Trial Design

1Treatment groups
Experimental Treatment
Group I: single armExperimental Treatment1 Intervention
AX 250 300 mg - open label
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AX 250
2016
Completed Phase 2
~30

Find a Location

Who is running the clinical trial?

Allievex CorporationLead Sponsor
4 Previous Clinical Trials
109 Total Patients Enrolled
Medical DirectorStudy DirectorAllievex Corporation
2,788 Previous Clinical Trials
8,066,037 Total Patients Enrolled
2 Trials studying Sanfilippo Syndrome
33 Patients Enrolled for Sanfilippo Syndrome

Media Library

AX 250 (Enzyme Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05492799 — Phase 4
Sanfilippo Syndrome Clinical Trial 2023: AX 250 Highlights & Side Effects. Trial Name: NCT05492799 — Phase 4
Sanfilippo Syndrome Research Study Groups: single arm
AX 250 (Enzyme Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05492799 — Phase 4
~10 spots leftby Oct 2027
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