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 patient’s peripheral limb vein. A tapering course of prophylactic enteral prednisone or prednisolone will be administered for a period of at least two months.
As of July 2020, patients are still being recruited. There are six trial locations globally, including two in the United States (Ohio and Pennsylvania). The others are in Australia, France, Germany, and Spain.
Read this clinical trial’s information on ClinicalTrials.gov, which includes enrollment criteria, trial locations, and study contact information.