AX 250 for Sanfilippo Syndrome
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests the safety and effectiveness of AX 250, a drug delivered directly to the brain through an implanted device, in improving cognitive function in patients with MPS IIIB.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but you cannot have received any investigational medication other than AX 250 within 30 days before starting the study.
Who Is on the Research Team?
Medical Monitor, MD
Principal Investigator
Allievex Corporation
Are You a Good Fit for This Trial?
This trial is for patients with Mucopolysaccharidosis Type IIIB who completed a previous study and can follow the new study's rules. They must not be pregnant, agree to use contraception or practice abstinence, and cannot have severe seizures, prior gene therapy/stem cell treatments (other than AX 250), or conditions making intraventricular drug delivery risky.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive AX 250 at the MTTD established in 250-201, 300mg administered weekly by ICV infusion
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- AX 250
Find a Clinic Near You
Who Is Running the Clinical Trial?
Allievex Corporation
Lead Sponsor