Granulocyte Colony-stimulating FactorNCT06708572
Evaluation of the Use of Granulocyte Colony Stimulating Factor (GCSF) in Post Kasai Type 3 Biliary Atresia
A study testing Granulocyte Colony-Stimulating Factor in people with Granulocyte Colony-stimulating Factor and Biliary Atresia, run by National Liver Institute, Egypt at 1 site in Egypt.
Trial 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. Read more about this study
- Who is it for?
- Ages 20 days to 1 yearHealthy volunteers can take part
- Conditions the study is for
- Granulocyte Colony-stimulating Factor, Biliary Atresia
- What is being tested?
- Granulocyte Colony-Stimulating Factor
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteEgypt
- Study dates
- Dec 2024to 30 Oct 2026
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 20 days to 1 year
- All sexes
- Healthy volunteers accepted
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
Egypt1 site, 1 recruiting
- Recruiting
National Liver Institute
Cairo, Menofia Governorate, 32511
Behairy El Sayed El Sayed Behairy, MD, +201001847754, behairtelsayed@yahoo.com
Gasser Gasser El-Azab, MD, g_elazab@liver.menofia.edu.eg
About this study
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.
Read the 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.
- Being tested
- Granulocyte Colony-Stimulating Factor
- Medical subject headings
- Biliary Atresia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Granulocyte Colony-Stimulating FactorDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Granulocyte Colony-Stimulating Factor |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: baseline
Secondary outcomes (1)
Differences in the proportion of subjects with cholangitis at 6 months.
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.
Measured: base line
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Nov 2024
- Study startDec 2024 (estimated)
- Primary completion30 Oct 2026 (estimated)
- Study completion30 Oct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Granulocyte Colony-stimulating FactorStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Prevention
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- 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.
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Liver Institute, Egypt
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06708572
- Sponsor's protocol ID
- GCSFin biliary atresia
- Status last verified
- Oct 2024
- First submitted
- 14 Oct 2024
- Last updated
- 11 Dec 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 29604222Overi 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.
PubMed 30257529Corrado 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.
PubMed 35106146
Study at a glance
From the registry record, last updated by the sponsor on 11 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Use of Granulocyte Colony Stimulating Factor (GCSF) in Post Kasai Type 3 Biliary Atresia
- ClinicalTrials.gov ID
- NCT06708572
- Acronym / sponsor ID
- GCSFin biliary atresia
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- National Liver Institute, EgyptUniversity, hospital or other
- Study start
- Dec 2024 (estimated)
- Primary completion
- 30 Oct 2026 (estimated)
- Study completion
- 30 Oct 2026 (estimated)
- First posted
- 27 Nov 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Prevention
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- 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.
| Arm | Type | Receives |
|---|---|---|
| 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 | Drug: Granulocyte Colony-Stimulating Factor20 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. |
Who may qualify
- Ages
- 20 days to 1 year
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- 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.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Granulocyte Colony-Stimulating Factor20 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. | Drug | biliary atresia with GCSF |
Primary outcome
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.
1 secondary outcome
Differences in the proportion of subjects with cholangitis at 6 months.
Time frame: base line
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Dec 2024.
- Background: PubMed 29604222Bezerra 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.
- Background: PubMed 30257529Overi 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.
- Background: PubMed 35106146Corrado 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Granulocyte Colony-Stimulating Factor in people with Granulocyte Colony-stimulating Factor and Biliary Atresia, run by National Liver Institute, Egypt at 1 site in Egypt.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 11 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Egypt. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Marwa Narwa Foad Asker, master (+201008913103, marwafoadali@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of subjects with serum TBi (total bilirubin) ≥ 2 mg/dL at 3 months. (time frame: baseline). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06708572, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Dec 2024.