Intracerebroventricular Tralesinidase Alfa in Children With Mucopolysaccharidosis Type IIIB

Phase 3Not Yet RecruitingNCT07579910

Spruce Biosciences14 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

SINGLE

Enrollment

Conditions

MPS IIIB

Interventions

Tralesinidase alfa (TA)DRUG

TA study drug is a sterile solution for ICV infusion.

Eligibility

Sex: ALLMin age: 1 YearMax age: 5 Years

Medical Language ↔ Plain English

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.

Outcomes

Primary Outcomes

Change from Baseline in Bayley Scales of Infant and Toddler Development, Third Edition, Cognition Domain (BSID-III-C) Raw Score

The BSID-III-C is a standardized tool that evaluates cognitive development in young children, capturing skills such as processing speed, problem solving, and play. Raw scores are used to sensitively measure individual change over time, independent of age. Assessments are administered by a trained, blinded rater and reviewed by a central blinded rater. The endpoint is the difference in mean change from baseline in BSID-III-C raw score between the tralesinidase alfa and control arms at Week 260

Time frame: Baseline to Week 260 (approximately 5 years)

Data from ClinicalTrials.gov

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