Intracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Typ… (NCT07579910) | Clinical Trial Compass
Not Yet RecruitingPhase 3
Intracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Type B)
14 participantsStarted 2026-12
Plain-language summary
The primary objectives of this study are to evaluate the effects of Tralesinidase Alfa (TA) on cognition
Who can participate
Age range
1 Year – 5 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.
Inclusion Criteria:
Has a diagnosis of MPS IIIB confirmed by deficient NAGLU enzyme activity during screening.
Has nonattenuated severe MPS IIIB by the Genotype and Clinical Assessment Committee.
Is ≥1 and ≤5 years of age with BSID-III-C raw score of \<70. Is male or female as identified at birth.
Exclusion Criteria:
Genotyped for a known MPS IIIB variant associated with an attenuated phenotype or has a sibling with a known attenuated phenotype.
Has another neurological illness that may have caused cognitive decline (e.g., trauma, meningitis, or hemorrhage) before study entry.
Has received stem cell, gene therapy, or enzyme replacement therapy for MPS IIIB.
Questions worth asking your doctor
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.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
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.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change from Baseline in Bayley Scales of Infant and Toddler Development, Third Edition, Cognition Domain (BSID-III-C) Raw Score
Timeframe: Baseline to Week 260 (approximately 5 years)