NCT05517317

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
12

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Jan 2015

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2015

Completed
7.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 30, 2022

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 30, 2022

Completed
3 months until next milestone

First Submitted

Initial submission to the registry

August 22, 2022

Completed
4 days until next milestone

First Posted

Study publicly available on registry

August 26, 2022

Completed
Last Updated

August 26, 2022

Status Verified

August 1, 2022

Enrollment Period

7.4 years

First QC Date

August 22, 2022

Last Update Submit

August 23, 2022

Conditions

Biliary Atresia

Outcome Measures

Primary Outcomes (1)

  • Adverse events and serious adverse events

    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

    up to the 12-month period following treatment

Secondary Outcomes (6)

  • The changes in cholestasis

    up to the 12-month period following treatment

  • The changes in Liver function using Aspart transaminase

    up to the 12-month period following treatment

  • The changes in Liver function using Alanine transaminase

    up to the 12-month period following treatment

  • The changes in Liver function using Gamma GT

    up to the 12-month period following treatment

  • The changes in level of cirrhosis

    up to the 12-month period following treatment

  • +1 more secondary outcomes

Study Arms (1)

Autologous BMMC infusion

EXPERIMENTAL

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

Combination Product: Autologous BMMC transplantation

Interventions

Autologous BMMC transplantationCOMBINATION_PRODUCT

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

Autologous BMMC infusion

Eligibility Criteria

Age2 Months - 15 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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)

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Vinmec Research Institute of Stem Cell and Gene Technology

Hanoi, 100000, Vietnam

Location

Related Publications (1)

  • Thanh LN, Nguyen HP, Kieu TPT, Duy MN, Ha HTT, Thi HB, Nguyen TQ, Pham HD, Tran TD. Modified Kasai operation combined with autologous bone marrow mononuclear cell infusion for biliary atresia. BMC Surg. 2024 Nov 20;24(1):368. doi: 10.1186/s12893-024-02669-9.

    PMID: 39568009DERIVED

MeSH Terms

Conditions

Biliary Atresia

Condition Hierarchy (Ancestors)

Bile Duct DiseasesBiliary Tract DiseasesDigestive System DiseasesDigestive System AbnormalitiesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Liem Thanh Nguyen, PhD

    Vinmec Research Institute of Stem Cell and Gene Technology

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Model Details: Autologous BMMC infusion
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 22, 2022

First Posted

August 26, 2022

Study Start

January 1, 2015

Primary Completion

May 30, 2022

Study Completion

May 30, 2022

Last Updated

August 26, 2022

Record last verified: 2022-08

Data Sharing

IPD Sharing
Will not share

Locations

VN(1)