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.
Open-label, dose-escalation clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein
Phase 1/Phase 2
- Biological: scAAV9.U1a.hSGSH
Abeona Therapeutics, Inc
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
Women's and Children's Hospital
North Adelaide, South Australia 5006
Hospital Clínico Universitario de Santiago
Santiago De Compostela, 15706
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.