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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

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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: 18 Mar 2026

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At a glance

  • Drugs Tralesinidase alfa (Primary)
  • Indications Mucopolysaccharidosis III
  • Focus Registrational; Therapeutic Use
  • Sponsors Allievex Corporation

Most Recent Events

  • 09 Mar 2026 According to Spruce Biosciences media release, the company announced successful completion of type B meeting, The FDA confirmed that the integrated study data from interventional clinical studies of TA-ERT and the available natural history data could potentially serve as an adequate and well-controlled study, effects of TA-ERT on cerebral spinal fluid heparan sulfate non-reducing end could be surrogate end point supporting accelerated approval. BLA submission anticipated in Q4 2026.
  • 10 Nov 2025 According to Spruce Biosciences media release, biologics license application (BLA) submission expected in the first quarter of 2026.
  • 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.

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