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)
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)
The primary objectives of this study are to evaluate the effects of Tralesinidase Alfa (TA) on cognition
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.
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