Last updated on July 2019

Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB

Brief description of study

Open-label, dose-escalation clinical trial of rAAV9.CMV.hNAGLU injected intravenously through a peripheral limb vein

Detailed Study Description

Adeno-associated virus serotype 9 carrying the human NAGLU gene under the control of a CMV enhancer/promoter (rAAV9.CMV.hNAGLU) will be delivered one-time through a venous catheter inserted into a peripheral limb vein.

Clinical Study Identifier: NCT03315182

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Hospital Clinico Universitario de Santiago

Santiago De Compostela, Spain
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