AX250 Enzyme Replacement | MPS IIIB | Phase I-II | Allievex

March 9, 2020

Trial Information

The study’s primary objectives are to evaluate the safety and tolerability of AX 250 administered to subjects with MPS IIIB via an ICV reservoir and catheter and to evaluate the impact of AX 250 on cognitive function in patients with MPS IIIB as assessed by the Development Quotient.

23 patients have been recruited and is not recruiting any longer. There are seven trial locations globally, including one in the United States (California). The others are in Columbia, Germany, Spain, Taiwan, Turkey, and the United Kingdom.

Read this clinical trial’s information on, which includes enrollment criteria, trial locations, and study contact information.

This trial was originally developed by BioMarin Pharmaceutical and is now owned by Allievex Corporation.

Trial Updates

November 2022

Researchers publish full results from the trial, which are generally consistent with the interim findings. “Tralesinidase Alfa ERT Found to Benefit Sanfilippo Type B Children” is published in The Journal of Clinical Investigation.

October 2019

Allievex obtains an exclusive worldwide license for tralesinidase alfa from BioMarin. It has assumed all financial obligations associated with the therapy’s development and commercialization and will manage all studies in the clinical program, which it declares will continue uninterrupted.

March 202

The clinical trial is now listed as “active, but not recruiting” on, meaning the trial is ongoing but no longer accepting participants.

May 2016

The experimental enzyme replacement therapy (ERT) tralesinidase alfa (AX 250) clinical trial is now listed as “recruiting” on

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