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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
Provides written informed consent from parent or legal guardian and assent from subject, if required
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 belowSelect...
My parent or guardian and I have both agreed to participate.
Select...
I am not pregnant and agree to regular pregnancy tests during the study.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline to 144 weeks of treatment
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to 144 weeks of treatment
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
2,766 Previous Clinical Trials
8,060,778 Total Patients Enrolled
2 Trials studying Sanfilippo Syndrome
33 Patients Enrolled for Sanfilippo Syndrome
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My parent or guardian and I have both agreed to participate.You have a condition that may cause problems with receiving the drug through a particular method of administration.I am not pregnant and agree to regular pregnancy tests during the study.You have a very low score on cognitive and developmental tests and did not show improvement in a previous study.I have received treatments like stem cell, gene therapy, or enzyme replacement for MPS IIIB, but not AX 250.I have a history of seizures that are hard to control.
Awards:
This trial has 3 awards, including:- Drug Has Already Been Approved - The FDA has already approved this drug, and is just seeking more data.
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Research Study Groups:
This trial has the following groups:- Group 1: single arm
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Is participation in this research endeavor still open to those interested?
"Unfortunately, according to clinicaltrials.gov this investigation is no longer recruiting participants; the trial was initially published on November 1st 2022 and its last update occurred August 8th 2022. Despite this, there are currently 6 other trials actively seeking people for their studies."
Answered by AI
Has the Food and Drug Administration given its seal of approval to AX 250?
"There is significant evidence indicating AX 250's safety, so it recieved a rating of 3."
Answered by AI
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