Trial Information This study is the first in-human clinical trial to explore the safety, tolerability, and clinical efficacy of ex vivo gene therapy (autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene) in MPSIIIA patients. This...
BMN-250 Enzyme Replacement | Type IIIB | Phase I-II | Biomarin/Allievex
March 9, 2020
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 ClinicalTrials.gov, which includes enrollment criteria, trial locations, and study contact information.