A Phase 2 interventional study of Filgrastim in Biliary Atresia, sponsored by Holterman, Ai-Xuan, M.D.. Recruiting at 4 sites in 3 countries. Open to participants aged 14 Days to 180 Days. Per ClinicalTrials.gov, last updated 2023-09-08.
Sponsored by Holterman, Ai-Xuan, M.D. · Phase 2, Interventional, and Treatment
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.
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:
Having met the same inclusion and exclusion criteria as the Kasai KBS group,
75 studies on the registry are indexed under Biliary Atresia; 25 are open to participants now.
This study's planned enrollment of 400 is above the median of 43 across 42 interventional studies indexed under Biliary Atresia.
Browse Biliary Atresia studies →Holterman, Ai-Xuan, M.D. is the lead sponsor of 2 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion criteria
Gl tract obstruction
For Kasai-operated subjects: Type 1 or 2 biliary atresia anatomy
The Kasai GCSF group will receive the standard of care PLUS 3 consecutive daily doses of 10 ug/kg of GCSF to be administered subcutaneously by day 3 post Kasai surgery
Drug: Filgrastim
The no GCSF group will not receive GCSF and receives the standard of care
The No Kasai GCSF group will receive the standard of care PLUS 3 consecutive daily doses of 10 ug/kg of GCSF to be administered subcutaneously once the diagnosis of BA is established
Drug: Filgrastim
The No Kasai No GCSF group will receive the standard of care and will not receive GCSF
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.
Also known as: Neupogen, granulocyte colony stimulating factor
GCSF Response on Bile flow (KBA)
For KBA subjects: Bile flow as measured by the percentage of subjects with total bilirubin\< 2 mg/dL at 3 months post-Kasai.
Time frame: 3 months
GCSF Response on transplant-free survival (NoK)
For NoK subjects: Changes at 6, 12, 18 and 24 months-transplant free survival
Time frame: 24 months
GCSF response on liver function and outcome (KBA)
KBA subjects: Pediatric end-stage liver disease (PELD) score at 6, 12, 18, and 24 months after GCSF treatment.
Time frame: 24 months
GCSF response on liver function and outcome (KBA)
KBA subjects: Percentage of patients with transplant-free survival at 6, 12, 18 and 24 months
Time frame: 24 months
GCSF response on liver function and outcome (KBA)
KBA subjects: Percentage of patients with cholangitis-free transplant-free survival at 6, 12, 18 and 24 months
Time frame: 24 months
GCSF response on liver function (NoK)
NoK subjects: Changes in Pediatric end-stage liver disease (PELD) score at 6, 12, 18, and 24 months after GCSF treatment.
Time frame: 24 months
Plan to share: No
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Holterman, Ai-Xuan, M.D.