NCT04373941

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

Granulocyte-Colony Stimulating Factor Adjunct Therapy for Biliary Atresia: Part II of a Prospective, Randomized Controlled, Multi-Institutional Trial

• Status: Unknown
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 400 (estimated)
• Sponsor: Holterman, Ai-Xuan, M.D. · Individual
• Sex: All
• Age: 14 Days – 180 Days
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Biliary Atresia

Interventions

Type	Name	Description
DRUG	Filgrastim	G-CSF is a glycoprotein produced by monocytes, fibroblasts, and endothelial cells. Filgrastim is a human granulocyte colony stimulating factor (G-CSF) produced by recombinant DNA technology with NEUPOGEN® as the Amgen Inc. trademark for filgrastim. G-CSF regulates the production, proliferation and differentiation of neutrophils and hematopoietic stem cell precursors within the bone marrow leading to dose-dependent increase in circulating neutrophils and hematopoietic stem cells in the blood. It is indicated to reduce the incidence of infection in patients with severe neutropenia, for neutrophil recovery in neutropenic patients with bone marrow depletion, to mobilize hematopoietic progenitor stem cell for collection by leukapheresis in hematopoietic stem cell transplantation.

Timeline

• Start date: 2023-09-01
• Primary completion: 2024-08-31
• Completion: 2025-10-31
• First posted: 2020-05-05
• Last updated: 2023-09-08

Locations

4 sites across 3 countries: United States, Pakistan, Vietnam

Regulatory

• FDA-regulated drug study