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

NCT06708572 | Recruiting DMC

• 1 other identifier: GCSFin biliary atresia
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Granulocyte Colony-stimulating Factor; Biliary Atresia

Outcome Measures

Primary Outcomes (1)

• Proportion of subjects with serum TBi (total bilirubin) ≥ 2 mg/dL at 3 months.

  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 Outcomes (1)

• Differences in the proportion of subjects with cholangitis at 6 months.

  base line

Study Arms (2)

biliary atresia without GCSF

NO INTERVENTION

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.

biliary atresia with GCSF

EXPERIMENTAL

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

Interventions

Granulocyte Colony-Stimulating Factor DRUG

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.

biliary atresia with GCSF

Eligibility Criteria

• Age: 20 Days - 1 Year
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• National Liver Institute, Egypt: lead

Study Sites (1)

National Liver Institute

Cairo, Menofia Governorate, 32511, Egypt

RECRUITING

Related Publications (3)

• Bezerra JA, Wells RG, Mack CL, Karpen SJ, Hoofnagle JH, Doo E, Sokol RJ. Biliary Atresia: Clinical and Research Challenges for the Twenty-First Century. Hepatology. 2018 Sep;68(3):1163-1173. doi: 10.1002/hep.29905.

  PMID: 29604222 BACKGROUND

• Overi D, Carpino G, Cardinale V, Franchitto A, Safarikia S, Onori P, Alvaro D, Gaudio E. Contribution of Resident Stem Cells to Liver and Biliary Tree Regeneration in Human Diseases. Int J Mol Sci. 2018 Sep 25;19(10):2917. doi: 10.3390/ijms19102917.

  PMID: 30257529 BACKGROUND

• Corrado MM, Mack CL. Diagnostic Tools for Early Detection of Biliary Atresia: Is a Newborn Screen Attainable? Clin Liver Dis (Hoboken). 2022 Jan 24;19(1):25-28. doi: 10.1002/cld.1165. eCollection 2022 Jan.

  PMID: 35106146 BACKGROUND

MeSH Terms

Conditions

Biliary Atresia

Interventions

Granulocyte Colony-Stimulating Factor

Condition Hierarchy (Ancestors)

Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Digestive System Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Central Study Contacts

Marwa Narwa Foad Asker, master

CONTACT

+201008913103; marwafoadali@gmail.com

Behairy Behairy El Sayed Behairy, MD

CONTACT

+201001847754; behairyelsayed@yahoo.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Protocol of cohort study will be conducted on 40 infants with the final diagnosis of biliary atresia type 3 (supported by the liver histology and intra-operative findings) divided into two groups one of them is for GCSF after Kasai operation, and the other group will be without GCSF therapy and their data will be collected retrospectively.

Study Record Dates

• First Submitted: October 14, 2024
• First Posted: November 27, 2024
• Study Start: December 1, 2024
• Primary Completion (Estimated): October 30, 2026
• Study Completion (Estimated): October 30, 2026
• Last Updated: December 11, 2024

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

EG (1)