Biliary AtresiaNCT06564740
Stem Cell Applications in Biliary Atresia Patients
A study testing Stem Cell in people with Biliary Atresia and Fibrosis, Liver, run by Necmi Kadıoğlu Hospital at 1 site in Turkey (Türkiye).
Trial 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). Read more about this study
- Who is it for?
- Ages 2 months to 18 years
- Conditions the study is for
- Biliary Atresia, Fibrosis, Liver, Stem Cell
- What is being tested?
- Stem Cell
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 64 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- 1 Jun 2024to 1 Jun 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 2 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Infants were diagnosed with liver cirrhosis due to biliary atresia following Kasai's operation.
- The patients two months old or older and exhibited signs of cirrhosis after the procedure, including hepatomegaly, congestive splenomegaly, elevated liver enzymes, esophageal varices (confirmed by endoscopy), and cirrhosis (confirmed by liver biopsy).
Who cannot join
- Epilepsy
- Neurological disorders
- Coagulation disorders
- Diabetes
- Syndromic type biliary atresia
- Allergies to anesthetic agents
- Severe health conditions such as cancer or failure of the heart, lungs, liver, or kidneys, active infections, and severe psychiatric disorders.
Trial locations and contacts
1 site in 1 country.
Turkey (Türkiye)1 site, 1 recruiting
- Recruiting
Esenyurt State Hospital
Istanbul, Istanbul, 34340
Mustafa Azizoglu, MD, PhD, +905447448244, mdmazizoglu@gmail.com
About this study
Official title: Umbilical Cord Derived Mesenchymal Stem Cell (UC-MSC) Transplantation in Infants with Biliary Atresia: a Prospective Randomized Controlled Trial
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.
Read the detailed description
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.
- Conditions
- Biliary Atresia, Fibrosis, Liver, Stem Cell
- Being tested
- Stem Cell, Control
- Keywords
- biliary atresia, liver fibrosis, stem cell
- Medical subject headings
- Biliary Atresia, Liver Cirrhosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Stem CellDrug
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.
ControlOther
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.
| Study group | Role | What they receive |
|---|---|---|
| Stem Cell Application Group | Experimental | 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. Receives: Drug: Stem Cell |
| Control | Sham comparator | 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. Receives: Other: Control |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 2 years
Secondary outcomes (13)
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).
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 2 years
Liver biopsy
Liver biopsy is a crucial clinical tool for assessing the progression and severity of cirrhosis
Measured: 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).
Measured: 2 years
Re-operation rate
The patients who need further surgical intervention will be noted.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Aug 2024
- Study start1 Jun 2024
- Primary completion30 May 2026 (estimated)
- Study completion1 Jun 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Biliary AtresiaStudies 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
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This study is designed as a multicentric randomized controlled trial (RCT). Patients will be randomly divided into two groups. Treatment Group: 30 Kasai-operated BA patients who will receive UC-MSC transplantation, and Control Group: 30 BA patients who will only undergo Kasai operation. 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.
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 64 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Necmi Kadıoğlu Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Mustafa Azizoğlu, Necmi Kadıoğlu Hospital
- Investigators
- Mustafa Azizoglu, MD, PhD, Principal investigator, Esenyurt State Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06564740
- Sponsor's protocol ID
- Biliar Atresia Stem Cell
- Status last verified
- Aug 2024
- First submitted
- 17 Aug 2024
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Umbilical Cord Derived Mesenchymal Stem Cell (UC-MSC) Transplantation in Infants with Biliary Atresia: a Prospective Randomized Controlled Trial
- ClinicalTrials.gov ID
- NCT06564740
- Acronym / sponsor ID
- Biliar Atresia Stem Cell
- Conditions
- Biliary Atresia, Fibrosis, Liver, Stem Cell
- Keywords
- biliary atresia, liver fibrosis, stem cell
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 64 participants (estimated)
- Lead sponsor
- Necmi Kadıoğlu HospitalUniversity, hospital or other
- Study start
- 1 Jun 2024 (actual)
- Primary completion
- 30 May 2026 (estimated)
- Study completion
- 1 Jun 2026 (estimated)
- First posted
- 21 Aug 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.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
- Model description
- This study is designed as a multicentric randomized controlled trial (RCT). Patients will be randomly divided into two groups. Treatment Group: 30 Kasai-operated BA patients who will receive UC-MSC transplantation, and Control Group: 30 BA patients who will only undergo Kasai operation. 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.
| Arm | Type | Receives |
|---|---|---|
| Stem Cell Application Group | Experimental | Drug: Stem CellIn 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. |
| Control | Sham comparator | Other: ControlIn 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. |
Who may qualify
- Ages
- 2 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Infants were diagnosed with liver cirrhosis due to biliary atresia following Kasai's operation.
- The patients two months old or older and exhibited signs of cirrhosis after the procedure, including hepatomegaly, congestive splenomegaly, elevated liver enzymes, esophageal varices (confirmed by endoscopy), and cirrhosis (confirmed by liver biopsy).
Key exclusion criteria (7 listed)
- Epilepsy
- Neurological disorders
- Coagulation disorders
- Diabetes
- Syndromic type biliary atresia
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 |
|---|---|---|
| Stem CellUC-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. | Drug | Stem Cell Application Group |
| ControlIn 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. | Other | Control |
Primary outcome
Adverse events
Time frame: 2 years
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.
13 secondary outcomes
Pediatric End-Stage Liver Disease (PELD) score
Time frame: 2 years
ALT levels (Alanine transaminase)
Time frame: 2 years
AST levels (Aspartate trasnaminase)
Time frame: 2 years
Direct bilirubin levels
Time frame: 2 years
Total bilirubin levels
Time frame: 2 years
GGT levels (Gama glutamil transferase)
Time frame: 2 years
Liver biopsy
Time frame: 2 years
Level of cirrhosis
Time frame: 2 years
Re-operation rate
Time frame: 2 years
Liver transplantation
Time frame: 2 years
Albumin levels
Time frame: 2 years
INR (international normalized ratio)
Time frame: 2 years
Cholangitis
Time frame: 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- 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.
Mustafa Azizoglu, MD, PhD principal investigator
Esenyurt State Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Stem Cell in people with Biliary Atresia and Fibrosis, Liver, run by Necmi Kadıoğlu Hospital at 1 site in Turkey (Türkiye).
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 2 inclusion and 7 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 19 Sep 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 Turkey (Türkiye). 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. Some participants in this study may receive a placebo. 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 Mustafa Azizoglu, MD, PhD (+905447448244, mdmazizoglu@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse events (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 19 Sep 2024.