NCT01661842

Brief Summary

Autoimmune hepatitis (AIH) is characterized by chronic inflammation of the liver, interface hepatitis, hypergammaglobulinemia, and the presence of autoantibodies. Disease presentation is varied but typically is based on characteristic aminotransferase elevations, histological abnormalities, elevated levels of serum globulins, and the presence of one or more autoantibodies. Two types of juvenile AIH have been identified according to seropositivity for smooth muscle and /or antinuclear antibody (AIH type 1) or liver kidney microsomal antibody (AIH type 2). Standard therapy in clinic consists of a combination of corticosteroids and azathioprine, which displays the efficacy in 80% of patients. However, 7% of patients deteriorate despite compliance with the standard corticosteroid regiments (treatment failure),13% of patients improve but not to a degree that satisfies remission criteria (incomplete response), 13% of patients develop serious drug-induced complications, and 50%-86% of patients will relapse after drug withdrawal. These serious drawbacks counterbalance the benefits of conventional therapy, and they are compelling reasons to refine current treatment strategies and pursue alternative therapies. UC-MSC has been the application for the treatment of several severe autoimmune diseases, such as immune thrombocytopenia, systemic lupus erythematosus, and therapy-resistant rheumatoid arthritis. In this study, the safety and efficacy of UC-MSC transplantation for AIH patients will be evaluated.

Trial Health

43
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
100

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Oct 2011

Typical duration for phase_1

Geographic Reach
1 country

1 active site

Status
unknown

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

October 1, 2011

Completed
10 months until next milestone

First Submitted

Initial submission to the registry

August 5, 2012

Completed
5 days until next milestone

First Posted

Study publicly available on registry

August 10, 2012

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2014

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2014

Completed
Last Updated

May 31, 2013

Status Verified

May 1, 2013

Enrollment Period

3 years

First QC Date

August 5, 2012

Last Update Submit

May 30, 2013

Conditions

Autoimmune Hepatitis

Keywords

Autoimmune HepatitisMesenchymal stem cellsserum albuminserum Tbilserum immunoglobulin G(IgG)

Outcome Measures

Primary Outcomes (2)

  • Liver Histology change

    baseline and 96 weeks

  • Serum alanine aminotransferase (ALT)

    0,12, 24, 36, 48, 72, 96 weeks after treatment

Secondary Outcomes (7)

  • Serum AST

    At baseline and at week 12, 24, 36, 48, 72, 96

  • Serum Tbil

    At baseline and at week 12, 24, 36, 48, 72, 96

  • Serum immunoglobulin G (IgG)

    At baseline and at week 12, 24, 36, 48, 72, 96

  • Serum γ-globulin

    At baseline and at week 12, 24, 36, 48, 72, 96

  • MELD score

    At base line and at week 12, 24, 36, 48, 72, 96

  • +2 more secondary outcomes

Study Arms (2)

Conventional plus UC-MSC treatment

EXPERIMENTAL

Participants will receive conventional treatment plus a dose of UC-MSC from day 0 through the week 12 study visit. Participants will then be followed until the week 96 study visit.

Other: conventional plus UC-MSC treatment

Conventional plus placebo treatment

EXPERIMENTAL

Participants will receive conventional plus placebo treatment from day 0 through the week 12 study visit. Participants will then be followed until the week 96 study visit.

Other: Conventional plus placebo treatment

Interventions

conventional plus UC-MSC treatmentOTHER

Received conventional treatment and taken i.v., once per 4 week, at a dose of 1×106 UC-MSC/kg body weight for 12 weeks.

Conventional plus UC-MSC treatment
Conventional plus placebo treatmentOTHER

Received conventional treatment and taken i.v., once per 4 week, at 50 ml saline for 12 weeks

Conventional plus placebo treatment

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Written informed consent
  • Autoimmune hepatitis (according to the criteria defined by the international autoimmune hepatitis Group ,Hepatology, 2008;48:169-176)
  • Negative pregnancy test (female patients in fertile age)

You may not qualify if:

  • Hepatocellular carcinoma or other Malignancies
  • Pregnant or lactating women
  • Viral Hepatitis ( HAV,HBV,HCV, et al )
  • Vital organs failure (Cardiac, Renal or Respiratory, et al)
  • Sepsis
  • Active thrombosis in the portal or hepatic veins

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Beijing 302 Hospital

Beijing, Beijing Municipality, 100039, China

RECRUITING

Related Publications (7)

  • Czaja AJ. Advances in the current treatment of autoimmune hepatitis. Dig Dis Sci. 2012 Aug;57(8):1996-2010. doi: 10.1007/s10620-012-2151-2. Epub 2012 Apr 3.

    PMID: 22476586BACKGROUND

  • Montano-Loza AJ, Carpenter HA, Czaja AJ. Features associated with treatment failure in type 1 autoimmune hepatitis and predictive value of the model of end-stage liver disease. Hepatology. 2007 Oct;46(4):1138-45. doi: 10.1002/hep.21787.

    PMID: 17668882BACKGROUND

  • Czaja AJ. Promising pharmacological, molecular and cellular treatments of autoimmune hepatitis. Curr Pharm Des. 2011;17(29):3120-40. doi: 10.2174/138161211798157568.

    PMID: 21902661BACKGROUND

  • Gossard AA, Lindor KD. Autoimmune hepatitis: a review. J Gastroenterol. 2012 May;47(5):498-503. doi: 10.1007/s00535-012-0586-z. Epub 2012 Apr 17.

    PMID: 22526272BACKGROUND

  • Malekzadeh Z, Haghazali S, Sepanlou SG, Vahedi H, Merat S, Sotoudeh M, Nasseri-Moghaddam S, Malekzadeh R. Clinical features and long term outcome of 102 treated autoimmune hepatitis patients. Hepat Mon. 2012 Feb;12(2):92-9. doi: 10.5812/hepatmon.808. Epub 2012 Feb 29.

    PMID: 22509185BACKGROUND

  • Yi T, Song SU. Immunomodulatory properties of mesenchymal stem cells and their therapeutic applications. Arch Pharm Res. 2012 Feb;35(2):213-21. doi: 10.1007/s12272-012-0202-z. Epub 2012 Feb 28.

    PMID: 22370776BACKGROUND

  • Holbro A, Abinun M, Daikeler T. Management of autoimmune diseases after haematopoietic stem cell transplantation. Br J Haematol. 2012 May;157(3):281-90. doi: 10.1111/j.1365-2141.2012.09070.x. Epub 2012 Feb 24.

    PMID: 22360687BACKGROUND

MeSH Terms

Conditions

Hepatitis, Autoimmune

Interventions

Congresses as Topic

Condition Hierarchy (Ancestors)

Hepatitis, ChronicHepatitisLiver DiseasesDigestive System DiseasesAutoimmune DiseasesImmune System Diseases

Intervention Hierarchy (Ancestors)

OrganizationsHealth Care Economics and Organizations

Study Officials

  • Fu-Sheng Wang, professor

    Beijing 302 Hospital

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Fu-Sheng Wang, professor

CONTACT

86-10-63879735fswang302@163.com

Zheng Zhang, Doctor

CONTACT

86-10-63879735Zhangzheng1975@yahoo.com.cn

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Director of both the Research Center for Biological Therapy and the Beijing Institute of Translational Hepatology

Study Record Dates

First Submitted

August 5, 2012

First Posted

August 10, 2012

Study Start

October 1, 2011

Primary Completion

October 1, 2014

Study Completion

October 1, 2014

Last Updated

May 31, 2013

Record last verified: 2013-05

Locations

CN(1)