Evaluation of the Use of Granulocyte Colony Stimulating Factor (GCSF) in Post Kasai Type 3 Biliary Atresia

December 9, 2024 updated by: National Liver Institute, Egypt
The aim of the study is to evaluate the use of Granulocyte Colony Stimulating Factor (GCSF) on the clinical and biochemical outcome of type 3 biliary atresia post kasai.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Biliary atresia (BA) is a devastating disease manifest early in infancy characterized by bile duct injury and extrahepatic biliary obstruction, leading to cirrhosis in the majority of infants.

Although BA is a rare disease, occurring in ~1 in 5600 to 1 in 18,000 infants worldwide, it is considered the most common indication for liver transplantation in children.

However, despite a 50-60% rate of initial jaundice clearance, liver transplantation by 2 years of age is necessary for long term survival in many of the post-Kasai patients.

GCSF cytokine that stimulates neutrophil and hematopoietic stem cell (HSC) production and mobilization from the bone marrow, and has served as a complementary agent to bone marrow stem cell therapy for patients with congenital or acquired diseases of bone marrow suppression.

Granulocyte colony-stimulating factor (G-CSF) mobilizes CD34+(cluster of differentiation34) cells, these CD34+ cells increase hepatocyte growth factor inducing the proliferation of hepatic progenitor cells within 7 days.

In experimental liver diseases of toxin-induced or bile duct ligation-induced liver injury, GCSF-based stem cell therapy has the same effects as direct HSC transplantation on improving liver regeneration and suppressing the inflammatory and fibrotic responses to hepatic injury. The cellular and molecular mechanisms are unknown but are postulated to be derived from the many paracrine actions of GCSF.

Study Type

Interventional

Enrollment (Estimated)

40

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

Yes

Description

Inclusion Criteria:

  • Infants with initial diagnosis of biliary atresia with biliary atresia score > 23.927 will be allocated for Kasai porto-enterostomy with intra-operative cholangiogram reaching type 3 biliary atresia anatomy as a final diagnosis.

Exclusion Criteria:

  • Major cardiac, renal, pulmonary, neurological malformations or illnesses.
  • Hemoglobinopathies, such as sickle cell anemia
  • Active systemic infection.
  • White blood cell count > 20,000 cells/mm3.
  • Platelet count < 40,000 cells/mm3 or ≥ 800,000 cells/mm3.
  • Purpura fulminans or unexplained vascular thrombotic conditions.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
No Intervention: biliary atresia without GCSF
20 infants with the final diagnosis of biliary atresia type 3 (supported by the liver histology and intra-operative finding) for GCSF after Kasai operation will not receive GCSF.
Experimental: biliary atresia with GCSF
20 infants with the final diagnosis of biliary atresia type 3 (supported by the liver histology and intra-operative finding) for GCSF after Kasai operation will receive GCSF.
Drug: Granulocyte Colony-Stimulating Factor
20 patients will receive subcutaneous GCSF at a daily dose of 10ug/kg for 3 consecutive days. It is administered within 3-4 days after the Kasai procedure.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Proportion of subjects with serum TBi (total bilirubin) ≥ 2 mg/dL at 3 months.
Time Frame: baseline
Proportion of subjects with serum TBi (total bilirubin) ≥ 2 mg/dL at 3 months will be analyzed using generalized linear mixed effects model (GLMM) with a logistic link function for the covariates as fixed effects, with study sites as random effect to control for site variability in management practices and patient characteristics. The covariates are potential confounders for poor outcome, such as age at the time of Kasai, presence of ductal plate malformation, cholangitis and possibly CMV IgM(cytomegalovirus immune globulin M antibodies) positivity.
baseline

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Differences in the proportion of subjects with cholangitis at 6 months.
Time Frame: base line
Differences in the proportion of subjects with cholangitis at 6 months will be calculated using the generalized linear mixed effect model with logistic link function for the covariates described in the primary outcome, with study sites as random effect to control for site variability in management practices and patient characteristics.
base line

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

National Liver Institute, Egypt

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

December 1, 2024

Primary Completion (Estimated)

October 30, 2026

Study Completion (Estimated)

October 30, 2026

Study Registration Dates

First Submitted

October 14, 2024

First Submitted That Met QC Criteria

November 25, 2024

First Posted (Actual)

November 27, 2024

Study Record Updates

Last Update Posted (Estimated)

December 11, 2024

Last Update Submitted That Met QC Criteria

December 9, 2024

Last Verified

October 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GCSFin biliary atresia

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Biliary Atresia

Clinical Trials on Granulocyte Colony-Stimulating Factor

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Singapore

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe