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

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Recruiting
participants needed

400
sponsor

Holterman, Ai-Xuan, M.D.

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Updated on 19 February 2024

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direct bilirubin

filgrastim

granulocyte colony stimulating factor

g-csf

colony stimulating factor

conjugated bilirubin

biliary atresia

cholangiography

cholestasis

gcsf

serum direct bilirubin

Summary

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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
Age	1years or below
Treatment	Filgrastim
Clinical Study Identifier	NCT04373941
Sponsor	Holterman, Ai-Xuan, M.D.
Last Modified on	19 February 2024

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