Part II: Granulocyte-Colony Stimulating Factor Adjunct Therapy for Biliary Atresia (BA_GCSF2b)

  • STATUS
    Recruiting
  • End date
    Oct 31, 2024
  • participants needed
    400
  • sponsor
    Holterman, Ai-Xuan, M.D.
Updated on 4 March 2022

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.

Description

This is a prospective, randomized, multi-institutional trial in KBA and NoK subjects to be conducted under a Food and Drug Administration approved Investigational New Drug application.

The KBA group is composed of just operated Kasai patients with intraoperative liver biopsy-confirmed BA. Their clinical characteristics have been described in the previously completed Phase 1 study under CR00005169 (ie. inclusion and exclusion criteria as described below)

The NoK group will be composed of newly diagnosed BA patients, including the following:

  • surgical patients in whom the Kasai was not performed for intraoperative technical reasons or due to advanced liver disease, who also have no option for rescue liver transplantation.
  • Unoperated patients whose family refuses surgery or who are not operative candidates

Having met the same inclusion and exclusion criteria as the Kasai KBS group,

  • eligible KBA subjects will be randomized to GCSF vs. no-GCSF at the 10 ug/kg/d dose to be given subcutaneously for 3 consecutive daily doses on the third day following the Kasai procedure.
  • eligible NoK subjects will be randomized to GCSF vs. no-GCSF at the 10 ug/kg/d dose to be given subcutaneously for 3 consecutive daily doses on the third day following diagnostic liver biopsy.

Details
Condition Biliary Atresia
Treatment filgrastim
Clinical Study IdentifierNCT04373941
SponsorHolterman, Ai-Xuan, M.D.
Last Modified on4 March 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

preliminary work up for cholestasis suspected or inconclusive diagnosis of BA
Serum Direct bilirubin > 2 mg/dl,GGT> 100 U/L
Male or female infants with a gestational age> 36 weeks
Admission weight > 2 kg
Age > 14 days - 180 days at diagnosis
For Kasai operated subjects, Type 3 or 4 anatomy of BA
For Kasai operated subjects, cholangiogram (if performed) diagnostic of BA
Liver biopsy supporting BA diagnosis

Exclusion Criteria

Patients having access to liver transplantation for immediate liver failure
Prior Kasai patients
Major cardiac, renal, central nervous system (CNS) malformations
Intracranial hemorrhage
History of recent total parenteral nutrition (TPN) use within the last 2 weeks
Gl tract obstruction
For Kasai-operated subjects: Type 1 or 2 biliary atresia anatomy
\. Current systemic infection
\. WBC > 20,000 cells/uL
\. Platelet count < 20,000 cells/uL or >1 million cells/uL
\. Concurrent respiratory, metabolic, neurological, cardiovascular
metabolic, and renal illness
\. Elevated serum creatinine > 1 mg/dL
\. Purpura fulminans or unexplained vascular thrombosis
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