GCSF Adjunct Therapy for Biliary Atresia

• Status: Completed
• Trial end date: 2020-01-31
• Target enrollment: 0
• Participant gender: All

Summary

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
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Holterman, Ai-Xuan, M.D.
• Collaborators: Big Leap Research, Vietnam; Children's National Health System; T. Rose Clinical, Inc., United States
• Treatments: Lenograstim; Sargramostim

Criteria

Inclusion Criteria:

1. Completed the preliminary work up for cholestasis with suspected or inconclusive diagnosis of BA

2. Gestational Age > 36wks

3. Weight > 2 Kg

4. Age >-2 weeks-<180 days at diagnosis

5. Serum Direct Bilirubin > 2 mg/dL GGT > 100 U/L

6. Kasai operated patients for Type 3 or 4 anatomy of BA

7. Cholangiogram/porta hepatis findings diagnostic of BA

8. Liver biopsy supporting BA diagnosis

Exclusion Criteria:

1. Having access to liver transplantation for immediate Kasai failure

2. Prior Kasai patients

3. Major cardiac, renal, CNS malformations with poor prognosis

4. Intracranial hemorrhage

5. History of recent TPN use within the last 2 weeks of surgery

6. GI tract obstruction

7. Laparoscopic Kasai repair