An interventional study of Stem Cell and Control in Biliary Atresia, Fibrosis, Liver and Stem Cell, sponsored by Necmi Kadıoğlu Hospital. Recruiting at 1 site in Turkey. Open to participants aged 2 Months to 18 Years. Per ClinicalTrials.gov, last updated 2024-09-19.
Sponsored by Necmi Kadıoğlu Hospital · Not applicable, Interventional, and Treatment
Recently, mesenchymal stem cell (MSC) transplantation has emerged as a promising treatment for liver cirrhosis in adults. Additionally, bone marrow-derived stem cell transplantation has shown success in treating children with biliary atresia (BA). This study aims to evaluate the efficacy of Umbilical Cord-Derived Mesenchymal Stem Cell (UC-MSC) therapy in BA through a multicentric randomized controlled trial.
Biliary atresia (BA) is the most common cause of chronic cholestasis in neonates and accounts for at least 50% of pediatric liver transplants. The incidence of BA is estimated to range from 1:5000 to 1:19000 live births. If the operation is not performed, all patients will die due to complications of liver cirrhosis. Recently, mesenchymal stem cell (MSC) transplantation has been found to be a promising treatment for liver cirrhosis in adults. Stem cell transplantation derived from bone marrow has also been successfully applied to children with BA. The aim of this study is to demonstrate the efficacy of Umbilical Cord-Derived Mesenchymal Stem Cell (UC-MSC) therapy in BA by planning a multicentric randomized controlled trial.
77 studies on the registry are indexed under Biliary Atresia; 25 are open to participants now.
This study's planned enrollment of 64 is above the median of 45 across 43 interventional studies indexed under Biliary Atresia.
Browse Biliary Atresia studies →Necmi Kadıoğlu Hospital is the lead sponsor of 4 studies on the registry; 4 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
In this group, UC-MSC transplantation will be administered twice to each patient in the study group via the hepatic artery: the first transplantation will be performed post-surgery at the beginning, and the second one will be performed 6 months later, with a dose of 1 million UC-MSC/kg. The applications will be applied after kasai portoenterostomy.
Drug: Stem Cell
In this group, UC-MSC will not be administered. This group serves as a passive control. The standard treatments that are routinely provided to Biliary Atresia patients will continue to be administered.
Other: Control
UC-MSC transplantation will be administered twice to each patient in the study group via the hepatic artery: the first transplantation will be performed post-surgery at the beginning, and the second one will be performed 6 months later, with a dose of 1 million UC-MSC/kg.
In this group, UC-MSC will not be administered. This group serves as a passive control. The standard treatments that are routinely provided to these patients will continue to be administered.
Adverse events
Adverse events will be assessed during the stem cell applications, as well as at 1 week, 1 month, 3 months, 6 months, 1 year, and 2 years after the application.
Time frame: 2 years
Pediatric End-Stage Liver Disease (PELD) score
Using PELD score (according to the suggestion of The Liver and Intestinal Organ Transplantation Committee in 2009). PELD is calculated based on three indicators: albumin (g / dL), bilirubin (units: mg / dL) and INR (international normalized ratio). Formula: PELD = 10 \* (0.48 \* ln(Serum Bilirubin) + 1.857 \* ln(INR) - 0.687 \* ln(Albumin) + (0.436 if patient is less than 1 year old) + (0.667 if patient has growth failure)). Evaluate the result: If PELD \<10: good results If 10 \<PELD \<15: average results If PELD\> 15: bad results Albumin (Unit: g / dL), bilirubin (units: mg / dL) and INR (international normalized ratio).
Time frame: 2 years
ALT levels (Alanine transaminase)
The levels of ALT will be closely monitored in both groups. The values for this parameter will be recorded and evaluated individually for each patient.
Time frame: 2 years
AST levels (Aspartate trasnaminase)
The levels of AST will be closely monitored in both groups. The values for each parameter will be recorded and evaluated individually for each patient.
Time frame: 2 years
Direct bilirubin levels
The levels of direct bilirubin will be closely monitored in both groups. The values for each parameter will be recorded and evaluated individually for each patient.
Time frame: 2 years
Total bilirubin levels
The levels of total bilirubin will be closely monitored in both groups. The values for each parameter will be recorded and evaluated individually for each patient.
Time frame: 2 years
GGT levels (Gama glutamil transferase)
The levels of GGT will be closely monitored in both groups. The values for each parameter will be recorded and evaluated individually for each patient.
Time frame: 2 years
Liver biopsy
Liver biopsy is a crucial clinical tool for assessing the progression and severity of cirrhosis
Time frame: 2 years
Level of cirrhosis
Level of cirrhosis will be assesed with PELD score. Using PELD score (according to the suggestion of The Liver and Intestinal Organ Transplantation Committee in 2009). PELD is calculated based on three indicators: albumin (g / dL), bilirubin (units: mg / dL) and INR (international normalized ratio). Formula: PELD = 10 \* (0.48 \* ln(Serum Bilirubin) + 1.857 \* ln(INR) - 0.687 \* ln(Albumin) + (0.436 if patient is less than 1 year old) + (0.667 if patient has growth failure)). Evaluate the result: If PELD \<10: good results If 10 \<PELD \<15: average results If PELD\> 15: bad results Albumin (Unit: g / dL), bilirubin (units: mg / dL) and INR (international normalized ratio).
Time frame: 2 years
Re-operation rate
The patients who need further surgical intervention will be noted.
Time frame: 2 years
Liver transplantation
Liver transplantation will be evaluated. The number of patients requiring a liver transplant, along with the timing of the need, will be recorded.
Time frame: 2 years
Albumin levels
The levels of albumin will be closely monitored in both groups. The values for each parameter will be recorded and evaluated individually for each patient.
Time frame: 2 years
INR (international normalized ratio)
The levels of INR (international normalized ratio) will be closely monitored in both groups. The values for each parameter will be recorded and evaluated individually for each patient.
Time frame: 2 years
Cholangitis
The number of cholangitis for each cases will be closely monitored in both groups. The frequency of cholangitis experienced by each patient will be recorded and evaluated.
Time frame: 2 years
Plan to share: No
No publications or documents are linked to this record.
Started Jun 2024 (actual). Primary completion is estimated for May 2026, 23 months after the start.
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Studies of the drug tested here: Stem Cell
Necmi Kadıoğlu Hospital