Your session is about to expire
← Back to Search
AX 250 for Sanfilippo Syndrome
Study Summary
This trial is testing a new treatment for MPS IIIB, which is a disease that affects cognitive function. The goal is to see if the treatment is safe and tolerable long-term, and if it has an impact on cognitive function.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Trial Design
Find a Location
Who is running the clinical trial?
Media Library
- You have a condition that may cause problems if the drug is given directly into the ventricles of the brain, such as hydrocephalus or ventricular shunts.I will not have sex or will use effective birth control during and for 6 months after the study.You have a low score on tests that measure thinking and learning abilities.I cannot have brain surgery due to heart, lung, or clotting issues.I have a history of seizures that are hard to control.I have received treatments like stem cell, gene therapy, or ERT for MPS IIIB.I am not pregnant and agree to regular pregnancy tests during the study.
- Group 1: AX 250
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- 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
Is there an age limitation for participants in this clinical experiment?
"Qualified candidates for this trial must be between 0 and 18 years old. The study encompasses 27 studies geared towards children, as well as 16 trials specifically targeting individuals older than 65."
Is the enrollment process for this trial open at present?
"As observed on clinicaltrials.gov, this trial is no longer recruiting participants. Despite being initially posted in February 2018 and updated last October 2022, the study has stopped looking for applicants; however, 28 other research projects are actively seeking patients right now."
May I be eligible to partake in the research?
"Currently, 20 children between 0 and 18 years old with mucopolysaccharidoses are being recruited for this research. The key criteria they must meet include having completed 48 weeks of Study 250-201 within the past 8 weeks, obtaining informed consent from their legal guardians or parents (with assent from the subject), compliance with protocol requirements according to the investigator's judgement, contraceptive precautions if sexually active and not practicing true abstinence during & after study duration, as well as a negative pregnancy test prior to screening."
What potential risks should be taken into account when administering AX 250 to individuals?
"The safety of AX 250 has been evaluated by the Power team and given a rating of 2, as this is an experimental Phase 2 trial with evidence to support its security but no data proving efficacy."
Share this study with friends
Copy Link
Messenger