Last updated on February 2020

Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Brief description of study

Open-label, dose-escalation clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein

Detailed Study Description

Self-complementary adeno-associated virus serotype 9 carrying the human SGSH gene under the control of a U1a promoter (scAAV9.U1a.hSGSH) 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: NCT02716246

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