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Completed

NCT Number: NCT05517317

Autologous BMNC Infusion for Liver Cirrhosis in Children With BA

To evaluate the safety and early outcomes of autologous bone marrow mononuclear cell (BMMNC) infusion for liver cirrhosis due to biliary atresia (BA) after Kasai operation. An open-label clinical trial was performed from January 2015 to December 2021. 12 children with liver cirrhosis due to BA at the time of Kasai or after Kasai were included. Bone marrow was harvested through anterior iliac crest puncture under general anesthesia. Mononuclear cells (MNCs) were isolated by Ficoll gradient centrifugation and then infused into the hepatic artery.

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Key information

Age range

2 month–15 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Vinmec Research Institute of Stem Cell and Gene Technology

Hanoi, 100000, Vietnam

About this study

Biliary atresia (BA) is a progressive fibro-obliterative cholangiopathy and a fatal disease. Without surgery, children with BA rarely survive beyond three years of age. The reported prevalence of BA ranges from 1 in 9640 to 1 in 19,500 live births. In the past, most children with a "non-correctable" type of BA died without adequate treatment. Recently, stem cell administration has been applied in adults with liver cirrhosis and has shown promising outcomes. An open-label clinical trial was performed from January 2015 to December 2021. 12 children with liver cirrhosis due to BA at the time of Kasai or after Kasai were included. Bone marrow was harvested through anterior iliac crest puncture under general anesthesia. Mononuclear cells (MNCs) were isolated by Ficoll gradient centrifugation and then infused into the hepatic artery. Serum bilirubin, albumin, alanine aminotransferase, aspartate aminotransferase, gamma-glutamyl transferase, and prothrombin time were monitored at baseline, three months, six months and 12 months after the transplantation. Esophagoscopies and liver biopsies were performed in patients whose parents provided consent. This study aimed to evaluate both safety and hepatic function after BMMNC administration in these children.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Children were diagnosed with Liver Cirrhosis Due to biliary atresia after Kasai's operation
  • Two months old or older
  • Patients with a manifestation of cirrhosis after Kasai's operation: hepatomegaly, congestive splenomegaly, elevated liver enzymes, Esophageal Varices (based on Endoscopic Diagnosis), cirrhosis (based on liver biopsy)

Exclusion criteria

  • Epilepsy
  • Coagulation disorders
  • Allergy to anesthetic agents
  • Severe health conditions such as cancer, failure of heart, lung, liver or kidney
  • Active infections
  • Severe psychiatric disorders

Treatment and study plan

Autologous BMMC transplantation

Combination Product

One administration of autologous bone marrow mononuclear cells via the hepatic artery

Primary outcomes

  1. Adverse events and serious adverse events

    Time frame: up to the 12-month period following treatment

    To assess safety, the number of AEs or SAEs during stem cell administration (72 h) at 1 month, 3 months, 6 months, and 9 months after discharge will be evaluated

Secondary outcomes

  1. The changes in cholestasis

    Time frame: up to the 12-month period following treatment

    Using Total Bilirubin (units: mg/dL) to measure the changes in cholestasis

  2. The changes in Liver function using Aspart transaminase

    Time frame: up to the 12-month period following treatment

    Using AST (Aspart transaminase) (units: U/L) to measure the changes in liver function.

  3. The changes in Liver function using Alanine transaminase

    Time frame: up to the 12-month period following treatment

    Using ALT (Alanine transaminase) (units: U/L) to measure the changes in liver function.

  4. The changes in Liver function using Gamma GT

    Time frame: up to the 12-month period following treatment

    Using GGT (Gamma GT) (units: U/L) to measure the changes in liver function.

  5. The changes in level of cirrhosis

    Time frame: up to the 12-month period following treatment

    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

  6. The changes in liver biopsy

    Time frame: up to the 12-month period following treatment

    Liver biopsy is a powerful clinical tool to evaluate the changes in the level of cirrhosis.

Sponsors and collaborators

Lead sponsor

Vinmec Research Institute of Stem Cell and Gene Technology

Other

Registry information

Official study title

Autologous Bone Marrow Mononuclear Cell Infusion for Liver Cirrhosis in Children With Biliary Atresia

Acronym: ABMNCBA

Important dates

Study start
2015
Primary completion
2022
Study completion
2022
First posted
Aug 26, 2022
Registry last updated
Aug 26, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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