MPS IIIB
Conditions
Brief summary
The primary objectives of this study are to evaluate the effects of Tralesinidase Alfa (TA) on cognition
Interventions
TA study drug is a sterile solution for ICV infusion.
Sponsors
Spruce Biosciences
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
1 Years to 5 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change from Baseline in Bayley Scales of Infant and Toddler Development, Third Edition, Cognition Domain (BSID-III-C) Raw Score | Baseline to Week 260 (approximately 5 years) | 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 |
Contacts
CONTACTApril Freeman
Outcome results
None listed