Stem Cell Applications in Biliary Atresia Patients
NCT Number: NCT06564740
Status: RECRUITING
Study Type: INTERVENTIONAL
Phases: NA
Conditions: Biliary Atresia|Fibrosis, Liver|Stem Cell
Interventions: DRUG: Stem Cell|OTHER: Control
Sponsor: Necmi Kadıoğlu Hospital
Sex: ALL
Age: CHILD, ADULT
Enrollment: 64
Study Design: Allocation: RANDOMIZED|Intervention Model: PARALLEL|Masking: SINGLE (OUTCOMES_ASSESSOR)|Primary Purpose: TREATMENT
Start Date: 2024-06-01
Completion Date: 2026-06-01
Locations: Esenyurt State Hospital, Istanbul, 34340, Turkey

Summary:
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.

Primary Outcome Measures: 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., 2 years
Secondary Outcome Measures: Cholestasis, The number of cholestasis for each cases will be closely monitored in both groups. The frequency of cholestasis experienced by each patient will be recorded and evaluated., 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)., 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., 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., 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., 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., 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., 2 years|Liver biopsy, Liver biopsy is a crucial clinical tool for assessing the progression and severity of cirrhosis, 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)., 2 years|Re-operation rate, The patients who need further surgical intervention will be noted., 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., 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., 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., 2 years