Clinical trial NCT04113317: Granulocyte-Colony Stimulating Factor (G-CSF) as Optimizing Therapy for Pediatric Liver Transplantation
Official title: The Role of Granulocyte-Colony Stimulating Factor (G-CSF) for Improving PELD Score and Nutritional Status in Pediatric Liver Cirrhosis Prior to Liver Transplantation Through Immunomodulation of Neutrophil, CD34+ Cell, TNF-α and IL-10
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Liver Cirrhosis
Severe Malnutrition
PELD
Undernutrition
Lead sponsor: Fakultas Kedokteran Universitas Indonesia
Sponsor class: OTHER
Collaborators: name: Dr Cipto Mangunkusumo General Hospital
First posted: 2019-10-02
Enrollment: 52
Sex: ALL
Minimum age (years): 3
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: SUPPORTIVE_CARE

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

Detailed description: This open-label randomized control trial conducted in Cipto Mangunkusumo Hospital, Jakarta, Indonesia, aimed to determine the effect of G-CSF to improve PELD score and nutritional status of pediatric patients with liver cirrhosis prior to liver transplantation. A total of 52 pediatric patients with liver cirrhosis, malnutrition and PELD score between 10-25 will be collected and divided into intervention group and control group. The intervention group will be given a subcutaneous injection of G-CSF 5μg/kg/day for 5 days consecutively, followed by a single dose every 3 days up to 12 times throughout the trial. Additionally, both the intervention and control group receive standard liver cirrhosis treatment. Randomization is done using block randomization.

The patients will be clinically monitored and evaluated for anthropometric changes (body weight, mid-arm circumference, mid-arm muscle circumference, and triceps skinfold thickness), complete blood count, absolute neutrophil count, liver function tests, cytokines (IL-10, TNF-α), Procalcitonin, hepatocyte growth factor (HGF), CD34+ cell count and PELD score (albumin, bilirubin, prothrombin and international normalized ratio values).

Data analysis will be performed using IBM SPSS Statistics version 20.0.0.

Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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

Primary outcomes:
measure: PELD Score
timeFrame: 90 days
measure: Anthropometric Changes
timeFrame: 90 days
Secondary outcomes:
measure: Pro-inflammatory Marker
timeFrame: 30 days
measure: Anti-inflammatory Marker
timeFrame: 30 days
measure: Liver Regeneration Marker
timeFrame: 30 days
Other outcomes:
Locations:
city: Jakarta
country: Indonesia
facility: Fakultas Kedokteran Universitas Indonesia
state: Java
Study officials:
affiliation: Faculty of Medicine, Universitas Indonesia - Cipto Mangunkusumo Hospital
name: Tri Hening Rahayatri, MD
role: STUDY_CHAIR
affiliation: Faculty of Medicine, Universitas Indonesia - Cipto Mangunkusumo Hospital
name: Akmal Taher
role: STUDY_DIRECTOR
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