Last updated on July 2020

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. A tapering course of prophylactic enteral prednisone or prednisolone will be administered for a period of at least two months.

Clinical Study Identifier: NCT03315182

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Recruitment Status: Open


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