Clinical Trial (Sanfilippo B Syndrome)

Trial Summary

Official Title

Phase I/II Gene Transfer Clinical Trial of rAAV9.CMV.hNAGLU for Mucopolysaccharidosis (MPS) IIIB




Mucopolysaccharidosis Type 3 B

About the Trial

Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB

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. The vector will be delivered undiluted over 10 to 20 minutes, under light to moderate sedation as needed. Dosing volume will be approximately 2-5 mL/kg, depending on final vector product concentration and subject cohort. A tapering course of prophylactic enteral prednisolone will be administered.

Detailed Info

Brief Summary

Open-label, dose-escalation clinical trial of rAAV9.CMV.hNAGLU injected intravenously through a peripheral limb vein

Study Phase

Phase 1/Phase 2

Study Type


  • Biological: rAAV9.CMV.hNAGLU

Kevin Flanigan


For more information on enrollment into the current ABO-101 trial, please visit for location information.


The following cities have ABO-101 clinical trial sites. Please check back often as locations will be updated.

Nationwide Children's Hospital
Columbus, Ohio 43205
United States
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.