The Investigators propose to test the hypothesis that GCSF therapy enhances the clinical
outcome of Kasai operated Biliary Atresia (BA) patients. In this study, Investigators will
conduct a dose determination for GCSF use in post Kasai subjects to support a future phase 2
efficacy study. The first 3 post Kasai BA subjects with liver biopsy-confirmed BA will be
given 5 ug/kg/d of GCSF in 3 daily subcutaneous doses starting on post Kasai day 3. A second
group of 3 subjects will be assigned to the 10 ug/Kg/d dose after the 5ug/kg/d dose has been
proven to be safe. The levels of circulating hematopoietic stem cells and a 1-month safety
profile will be analyzed.
Phase:
Early Phase 1
Details
Lead Sponsor:
Holterman, Ai-Xuan, M.D.
Collaborators:
Big Leap Research, Vietnam Children's National Health System T. Rose Clinical, Inc., United States