Intracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Type B)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Tralesinidase alfa (TA).
- Who it may be relevant to
- Registry conditions: MPS IIIB. Basic parameters: 1 year — 5 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Center list to be confirmed — check the primary protocol.
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase 3, Randomized, Single-blind, Parallel Group, Controlled, Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of Intracerebroventricular Tralesinidase Alfa Treatment Compared to Standard of Care in Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)
Overview
The primary objectives of this study are to evaluate the effects of Tralesinidase Alfa (TA) on cognition
Interventions
- Drug Tralesinidase alfa (TA)
TA study drug is a sterile solution for ICV infusion.
Primary outcome measures
- Change from Baseline in Bayley Scales of Infant and Toddler Development, Third Edition, Cognition Domain (BSID-III-C) Raw Score [Time frame: Baseline to Week 260 (approximately 5 years)]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Single blind
- Primary purpose
- Treatment
Study locations
Center list to be confirmed — check the primary protocol.
Identifiers
NCT: NCT07579910 · 250-301