This is a natural history study for children up to 18 years of age who have been diagnosed with Mucopolysaccharidosis Type IIIB (MPS IIIB, also known as Sanfilippo Syndrome Type B). Mucopolysaccharidosis type IIIB is a severe neurodegenerative disorder. The information gathered from this trial may help inform the design and interpretation of subsequent interventional studies. No clinical intervention or study drug is provided by Allievex in this study.
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Neurocognitive function
Timeframe: Baseline + every 24 weeks for up to 240 weeks
Behavioral function
Timeframe: Baseline + every 24 weeks for up to 240 weeks
Quality of Life Tests
Timeframe: Baseline + every 24 weeks for up to 240 weeks
Sleep habits
Timeframe: Baseline + every 24 weeks for up to 240 weeks
Disease-specific Biomarkers
Timeframe: Baseline + every 24 weeks for up to 240 weeks
Biochemical, Molecular, Cellular and Genetic Markers of Disease Burden
Timeframe: Once (at baseline visit)