Part II: Granulocyte-Colony Stimulating Factor Adjunct Therapy for Biliary Atresia

• Status: Recruiting
• Trial end date: 2024-10-31
• Target enrollment: 0
• Participant gender: All

Summary

The Investigators propose to test the hypothesis that GCSF enhances the clinical outcome of biliary atresia in a multi-institutional Phase 2 trial to prospectively evaluate the safety and efficacy of GCSF in each of the 2 groups of newly diagnosed BA patients: KBA (i.e., Kasai-operated) or NoK (i.e., patients who did not undergo Kasai surgery). Subjects who participate in the trial will be followed for 2 years.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Holterman, Ai-Xuan, M.D.
• Collaborators: Big Leap Research; T Rose Clinical, Inc.
• Treatments: Lenograstim; Sargramostim

Criteria

Inclusion criteria

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

2. Serum Direct bilirubin > 2 mg/dl,GGT> 100 U/L

3. Male or female infants with a gestational age> 36 weeks

4. Admission weight > 2 kg

5. Age > 14 days - 180 days at diagnosis

6. For Kasai operated subjects, Type 3 or 4 anatomy of BA

7. For Kasai operated subjects, cholangiogram (if performed) diagnostic of BA

8. Liver biopsy supporting BA diagnosis

Exclusion criteria

1. Patients having access to liver transplantation for immediate liver failure

2. Prior Kasai patients

3. Major cardiac, renal, central nervous system (CNS) malformations

4. Intracranial hemorrhage

5. History of recent total parenteral nutrition (TPN) use within the last 2 weeks

6. Gl tract obstruction

For Kasai-operated subjects: Type 1 or 2 biliary atresia anatomy

7. Current systemic infection

8. WBC > 20,000 cells/uL

9. Platelet count < 20,000 cells/uL or >1 million cells/uL

10. Concurrent respiratory, metabolic, neurological, cardiovascular, metabolic, and renal illness

11. Elevated serum creatinine > 1 mg/dL

12. Purpura fulminans or unexplained vascular thrombosis