Clinical Trial (Sanfilippo A Syndrome)

Trial Summary

Official Title

Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA




Mucopolysaccharidosis Type 3 A Sanfilippo Syndrome

About the Trial

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

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. The vector will be delivered undiluted over 5 to 50 minutes, under light to moderate sedation as needed. Dosing volume will be approximately 0.5 to 3 mL/kg, depending on final vector product concentration and subject cohort. A tapering course of prophylactic enteral prednisone or prednisolone will be administered.

Detailed Info

Brief Summary

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

Study Phase

Phase 1/Phase 2

Study Type


  • Biological: scAAV9.U1a.hSGSH

Abeona Therapeutics, Inc


For more information on enrollment into the current ABO-102 trial, please visit for location information.


The following cities have ABO-102 clinical trial sites. Please check back often as locations will be updated.

Nationwide Children's Hospital
Columbus, Ohio 43205
United States
Status: Recruiting

Women's and Children's Hospital
North Adelaide, South Australia 5006
Status: Recruiting

Hospital Clínico Universitario de Santiago
Santiago De Compostela, 15706
Status: Recruiting

The safety and efficacy of the investigational use of this product has not been determined. There is no guarantee that the investigational use listed will be filed with and/or approved for marketing by a regulatory agency.