Study of AX 250 for MPS IIIB in children
Part of Genetic & congenital, Hormones & metabolism, Skin clinical trials.
This Phase 4 trial studies how safe, tolerable, and helpful ICV AX 250 is for people with MPS IIIB. It is for participants who already finished a prior AX 250 study and meet specific health and eligibility checks.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have already completed 240 weeks of the earlier Study 250-202 and start the next study within 8 weeks after stopping dosing
- A parent or legal guardian must sign consent, and your child must also assent if required
- Your study team must believe you can follow all study visits and instructions
- If the participant is a female who could become pregnant: you must have a negative pregnancy test at screening and agree to repeat tests during the study
- You must not have very low thinking/development scores and must have shown improvement on prior study outcomes (not just stable results)
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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