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Also known as Tralesinidase alfa (TA)
Tralesinidase alfa · 1 trial · 1 indication
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
| Arm | Type | Description |
|---|---|---|
| Tralesinidase alfa (TA) | EXPERIMENTAL | - |
| Control | NO_INTERVENTION | - |
| Name | Type | Description |
|---|---|---|
| Tralesinidase alfa (TA) | DRUG | TA study drug is a sterile solution for ICV infusion. |
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...
Tralesinidase alfa is an investigational therapy being developed for MPS IIIB, also known as Sanfilippo syndrome type B. It is designed to address the underlying enzyme deficiency in this rare pediatric lysosomal storage disorder. The drug has not been approved by the FDA and remains in clinical development.
Tralesinidase alfa targets IGF2/NAGLU. It is designed to deliver a functional form of the NAGLU enzyme, which is deficient in MPS IIIB, by using the IGF2 receptor to enable transport across the blood-brain barrier. This targeted approach aims to address the central nervous system manifestations of the disease.
Tralesinidase alfa is being developed by Spruce Biosciences, Inc., which trades on the Nasdaq under the ticker SPRB. The company is responsible for the clinical development program of this investigational therapy for MPS IIIB.
Tralesinidase alfa is in Phase 3 clinical development. It is an investigational drug and has not been approved by the FDA for any indication. The Phase 3 program is ongoing, with one active trial currently listed.
Tralesinidase alfa is being evaluated in a Phase 3 trial, NCT07579910, titled Intracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Type B). The trial has a planned enrollment of 14 participants and is not yet recruiting. It is randomized and uses a no-treatment control.
Yes, tralesinidase alfa is also known as TA. Both names refer to the same investigational therapy being developed by Spruce Biosciences for MPS IIIB. The abbreviation TA is commonly used in reference to tralesinidase alfa.