Granulocyte-Colony Stimulating Factor (G-CSF) as Optimizing Therapy for Pediatric Liver Transplantation

NCT04113317 | Completed Phase 3 FDA Drug

• 1 other identifier: FakultasKUI
• Study Type: interventional
• Target: 52
• Locations: 1 country
• Sites: 1

Brief Summary

This study compares the effect of human recombinant Granulocyte-Colony Stimulating Factor (G-CSF) in pediatric patients with liver cirrhosis with a control group. The study aims to observe improvement of (Pediatric End-stage Liver Disease) PELD score and nutritional status prior to liver transplantation procedure. In addition to the intervention, standard treatments for liver cirrhosis are also given for both groups. G-CSF is administered for 12 times. Condition of disease: Pediatric patient aged 3 months to 12 years old Liver cirrhosis Undernourished / Severe malnutrition PELD score 10-25 Intervention: Drug: Recombinant Human G-CSF Phase: Phase 3

Conditions

Liver Cirrhosis; Severe Malnutrition; PELD; Undernutrition

Outcome Measures

Primary Outcomes (2)

• PELD Score

  The scoring determines the individual's need for liver transplant. Calculation is based on an equation that incorporates the values of bilirubin serum, international normalized ratio, albumin and body growth measurement. Score ranges between 6 (low illness level) to 40 (severely ill).

  90 days

• Anthropometric Changes

  Measurement of mid-arm circumference (MAC) will be obtained in centimeters and be plotted on the curve to observe changes in nutritional status

  90 days

Secondary Outcomes (3)

• Pro-inflammatory Marker

  30 days

• Anti-inflammatory Marker

  30 days

• Liver Regeneration Marker

  30 days

Study Arms (2)

Treatment Group

EXPERIMENTAL

Subcutaneous injection of G-CSF with dose of 5μg/kg/day for 5 days consecutively, in addition to a single dose every 3 days up to 12 times, as well as liver cirrhosis standard regimen

Drug: G-CSF (Filgrastim)

Control Group

NO INTERVENTION

Liver cirrhosis standard treatment only

Interventions

G-CSF (Filgrastim) DRUG

Contains 0,59 mg of acetate, 0,04 mg of polysorbate 80, 0,035 mg of sodium, 50 mg of sorbitol

Also known as: Neupogen

Treatment Group

Eligibility Criteria

• Age: 3 Months - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Age between 3 months to 12 years old
• Conscious state
• Without the presence of fever (temperature: \<37,5°C)
• Decompensated liver cirrhosis
• Liver cirrhosis due to various etiology
• Undernourished or severe malnutrition
• PELD score between 10-25

You may not qualify if:

• Malignancy (liver origin or other types)
• History of undergoing any organ transplantation procedure
• Acute liver failure
• Failure of any organ other than the liver
• Encephalopathy
• Severe infection such as bacterial peritonitis and pneumonia

Sponsors & Collaborators

• Fakultas Kedokteran Universitas Indonesia: lead
• Dr Cipto Mangunkusumo General Hospital: collaborator

Study Sites (1)

Fakultas Kedokteran Universitas Indonesia

Jakarta, Java, 16424, Indonesia

Location

Related Publications (5)

• Fioredda F, Lanza T, Gallicola F, Riccardi F, Lanciotti M, Mastrodicasa E, Signa S, Zanardi S, Calvillo M, Dufour C. Long-term use of pegfilgrastim in children with severe congenital neutropenia: clinical and pharmacokinetic data. Blood. 2016 Oct 27;128(17):2178-2181. doi: 10.1182/blood-2016-07-727891. Epub 2016 Sep 12. No abstract available.

  PMID: 27621310 BACKGROUND

• Dale DC, Crawford J, Klippel Z, Reiner M, Osslund T, Fan E, Morrow PK, Allcott K, Lyman GH. A systematic literature review of the efficacy, effectiveness, and safety of filgrastim. Support Care Cancer. 2018 Jan;26(1):7-20. doi: 10.1007/s00520-017-3854-x. Epub 2017 Sep 22.

  PMID: 28939926 BACKGROUND

• Yang Q, Yang Y, Shi Y, Lv F, He J, Chen Z. Effects of Granulocyte Colony-Stimulating Factor on Patients with Liver Failure: a Meta-Analysis. J Clin Transl Hepatol. 2016 Jun 28;4(2):90-6. doi: 10.14218/JCTH.2016.00012. Epub 2016 Jun 15.

  PMID: 27350939 BACKGROUND

• Garg V, Garg H, Khan A, Trehanpati N, Kumar A, Sharma BC, Sakhuja P, Sarin SK. Granulocyte colony-stimulating factor mobilizes CD34(+) cells and improves survival of patients with acute-on-chronic liver failure. Gastroenterology. 2012 Mar;142(3):505-512.e1. doi: 10.1053/j.gastro.2011.11.027. Epub 2011 Nov 23.

  PMID: 22119930 BACKGROUND

• Chavez-Tapia NC, Mendiola-Pastrana I, Ornelas-Arroyo VJ, Norena-Herrera C, Vidana-Perez D, Delgado-Sanchez G, Uribe M, Barrientos-Gutierrez T. Granulocyte-colony stimulating factor for acute-on-chronic liver failure: systematic review and meta-analysis. Ann Hepatol. 2015 Sep-Oct;14(5):631-41.

  PMID: 26256891 BACKGROUND

MeSH Terms

Conditions

Liver Cirrhosis; Malnutrition

Interventions

Granulocyte Colony-Stimulating Factor; Filgrastim

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Fibrosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Nutrition Disorders; Nutritional and Metabolic Diseases

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

Study Officials

• Tri Hening Rahayatri, MD

  Faculty of Medicine, Universitas Indonesia - Cipto Mangunkusumo Hospital

  STUDY CHAIR

• Akmal Taher

  Faculty of Medicine, Universitas Indonesia - Cipto Mangunkusumo Hospital

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: September 22, 2019
• First Posted: October 2, 2019
• Study Start: January 1, 2020
• Primary Completion: February 28, 2022
• Study Completion: February 28, 2022
• Last Updated: July 27, 2022

Record last verified: 2022-07

Locations

ID (1)