Autologous Bone Marrow Mononuclear Stem Cell for Children Suffering From Liver Cirrhosis Due to Biliary Atresia

NCT03468699 | Completed Phase 2 DMC

• 1 other identifier: VinmecRISCGT78
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 2

Brief Summary

The aim of this study was to evaluate the safety and effectiveness of autologous bone marrow mononuclear stem cell transplantation for Children Suffering from Liver Cirrhosis Due to Biliary Atresia

Conditions

Liver Cirrhosis, Biliary

Keywords

Autologous bone marrow Transplantation; biliary atresia; Kasai's operation; liver cirrhosis

Outcome Measures

Primary Outcomes (4)

• Assess the changes in cholestasis

  Using Bilirubin (total, direct and indirect) (units: mg/dL) to measure the changes in cholestasis

  Baseline, 3, 6 months and 12 months after transplantation

• Assess the changes in Liver function

  Using AST (Aspart transaminase) and ALT (Alanin transaminase) (units: U/L), GGT (Gamma GT) (units: UI/L), and Bilirubin (units: mg/dL) to measure the changes in Liver function

  Baseline, 3, 6 months and 12 months after transplantation

• Assess the changes in level of cirrhosis

  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).

  Baseline, 3, 6 months and 12 months after transplantation

• Assess the changes in liver biopsy

  Liver biopsy, in combination with history and physical examination data, is a powerful clinical tool for diagnosing and treating liver disease to evaluate the changes in liver biopsy

  Baseline, 3, 6 months and 12 months after transplantation

Study Arms (1)

Autologous BMMC transplantation

EXPERIMENTAL

Stem cell transplantations include 2 administrations of autologous bone marrow mononuclear cells via the hepatic artery at baseline and 6 months afterward

Combination Product: Autologous BMMC transplantation

Interventions

Autologous BMMC transplantation COMBINATION_PRODUCT

Stem cell transplantation

Autologous BMMC transplantation

Eligibility Criteria

• Age: 1 Year - 15 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)

You may qualify if:

• Children were diagnosed with Liver Cirrhosis Due to biliary atresia after Kasai's operation
• From 5 months to 2 years old
• Weight ≥ 6 kg
• Patients with manifestation of cirrhosis after Kasai's operation: hepatomegaly, congestive splenomegaly, elevated liver enzymes, Esophageal Varices (based on Endoscopic Diagnosis), cirrhosis (based on liver biopsy)

You may not qualify if:

• Under 1 year old patients
• 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

Sponsors & Collaborators

• Vinmec Research Institute of Stem Cell and Gene Technology: lead

Study Sites (2)

Vinmec Research Institute of Stem Cell and Gene Technology

Hanoi, 100000, Vietnam

Location

Vinmec Research Institute of Stem Cell and Gene Technology

Hanoi, 10000, Vietnam

Location

Related Publications (6)

• Nicolas C, Wang Y, Luebke-Wheeler J, Nyberg SL. Stem Cell Therapies for Treatment of Liver Disease. Biomedicines. 2016 Jan 6;4(1):2. doi: 10.3390/biomedicines4010002.

  PMID: 28536370 BACKGROUND

• Levicar N, Dimarakis I, Flores C, Tracey J, Gordon MY, Habib NA. Stem cells as a treatment for chronic liver disease and diabetes. Handb Exp Pharmacol. 2007;(180):243-62. doi: 10.1007/978-3-540-68976-8_11.

  PMID: 17554512 BACKGROUND

• Irfan A, Ahmed I. Could Stem Cell Therapy be the Cure in Liver Cirrhosis? J Clin Exp Hepatol. 2015 Jun;5(2):142-6. doi: 10.1016/j.jceh.2014.03.042. Epub 2014 Mar 27.

  PMID: 26155042 BACKGROUND

• Sharma S, Kumar L, Mohanty S, Kumar R, Datta Gupta S, Gupta DK. Bone marrow mononuclear stem cell infusion improves biochemical parameters and scintigraphy in infants with biliary atresia. Pediatr Surg Int. 2011 Jan;27(1):81-9. doi: 10.1007/s00383-010-2712-4.

  PMID: 20857300 BACKGROUND

• Khan AA, Parveen N, Mahaboob VS, Rajendraprasad A, Ravindraprakash HR, Venkateswarlu J, Rao P, Pande G, Narusu ML, Khaja MN, Pramila R, Habeeb A, Habibullah CM. Management of hyperbilirubinemia in biliary atresia by hepatic progenitor cell transplantation through hepatic artery: a case report. Transplant Proc. 2008 May;40(4):1153-5. doi: 10.1016/j.transproceed.2008.03.110.

  PMID: 18555137 BACKGROUND

• Nguyen TL, Nguyen HP, Ngo DM, Ha THT, Mai KA, Bui TH, Nguyen PV, Pham LH, Hoang DM, Cao ADT. Autologous bone marrow mononuclear cell infusion for liver cirrhosis after the Kasai operation in children with biliary atresia. Stem Cell Res Ther. 2022 Mar 14;13(1):108. doi: 10.1186/s13287-022-02762-x.

  PMID: 35287722 DERIVED

MeSH Terms

Conditions

Liver Cirrhosis, Biliary; Biliary Atresia; Liver Cirrhosis

Condition Hierarchy (Ancestors)

Cholestasis, Intrahepatic; Cholestasis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Liver Diseases; Fibrosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Digestive System Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Liem Nguyen

  Vinmec Research Institute of Stem Cell and Gene Technology

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 26, 2018
• First Posted: March 16, 2018
• Study Start: January 2, 2017
• Primary Completion: September 30, 2021
• Study Completion: October 31, 2021
• Last Updated: June 4, 2024

Record last verified: 2024-06

Locations

VN (2)