Trial Profile

Open-Label Multicenter Extension Study to Further Evaluate Safety, Tolerability and Efficacy of Intracerebroventricular AX 250 Treatment in Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) Patients

Status: Recruiting

Phase of Trial: Phase III/IV

Latest Information Update: 25 May 2025

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At a glance

06 May 2025 According to Spruce Biosciences media release, the company intends to submit the Biologics License Application (BLA) of TA-ERT for the treatment of MPS IIIB in the first half of 2026.
16 Dec 2023 This trial has been suspended in Germany according to European Clinical Trials Database record.
16 Jan 2023 Status changed from not yet recruiting to recruiting.

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