Trial record 99 of 806 for:    Interventional Studies | mesenchymal

Allogenic Bone Marrow Derived Mesenchymal Stem Cell Therapy in Cases of Hemophilia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02108132
Recruitment Status : Unknown
Verified April 2014 by Hala Gabr, Cairo University.
Recruitment status was:  Not yet recruiting
First Posted : April 9, 2014
Last Update Posted : April 9, 2014
Affiliated Hospital to Academy of Military Medical Sciences
Information provided by (Responsible Party):
Hala Gabr, Cairo University

Brief Summary:

Hemophilia is caused by a single-gene defect resulting in familial bleeding disorder. Small increase in gene products could transform a severe form of hemophilia into a mild one. Stem cells from extrahepatic sources are being considered for clinical applications in liver cell therapy as they possess high in vitro culture potential and could be used in transplant procedures. We studied the differentiation of bone marrow hematopoietic stem cells (BM-HSCs) from hemophilia patients' relatives into factor 8 (FVIII)-producing hepatocyte-like cells aiming to expand patients' donor options for partial replacement of mutant liver cells by healthy cells in hemophilia A patients which could manage the severity of the bleeding disorder.

BM-HSCs from hemophilic families will be cultured in short-liquid hepatic induction medium. Appearance of hepatic phenotype will be evaluated by alpha-fetoprotein expression using immunocytochemistry. Functional evaluation of transdifferentiation will be done through detection of albumin synthesis using microalbumin assay kit, factor VIII activity by one-stage clotting assay and expression of FVIII messenger RNA( mRNA) by reverse transcription ( RT-PCR).

Inducing the differentiation of BM-HSCs by in-vitro manipulation may become a valuable tool to provide a cell source for hepatocyte transplant procedures for treatment of hemophilia patients.

Condition or disease Intervention/treatment Phase
Hemophilia Biological: Cellular therapy Biological: cellular therapy Phase 1

  Show Detailed Description

Study Type : Interventional
Estimated Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Allogenic Mesenchymal Stem Cells in Hemophilia: a Pilot Study
Study Start Date : August 2014
Estimated Primary Completion Date : August 2015
Estimated Study Completion Date : February 2016

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: Cellular therapy
Cellular therapy : injection of mesenchymal stem cells subjected to hepatogenic induction
Biological: Cellular therapy
bone marrow derived mesenchymal stem cells for normal subjects will be separated and induced to adopt the hepatocyte phenotype then injected through the portal vein to hemophilia patients

Biological: cellular therapy
Allogenic bone marrow derived MSCs will be isolated and subjected to induction of hepatic phenotype. After proof of in vitro secretion of albumin and factor 8 from the cell population. It will be injected into the portal vein via the spleen
Other Name: hepatocyte-like MSC

Primary Outcome Measures :
  1. Assessment of Safety / Efficacy [ Time Frame: 2 years ]
    Assessment of vitality, life style and bleeding times of the Patients.

Secondary Outcome Measures :
  1. Assessment of Coagulation Profile [ Time Frame: 2 years ]

    All Participants Subjected to :

    1. APPT (activated partial thromboplastin time) weekly
    2. Factor 8 assay will be done weekly.

Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years to 40 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Laboratory diagnosis of factor 8 deficiency
  • Dependent on exogenous factor 8 therapy

Exclusion Criteria:

  • Liver disease
  • History of allergy to factor therapy
  • Abnormal spleen by sonography

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02108132

Contact: Hala Gabr, M.D. +202-23644460

Military Medical Academy Not yet recruiting
Cairo, Egypt, 11451
Principal Investigator: wael Abou El-kheir, M.D.         
Sponsors and Collaborators
Cairo University
Affiliated Hospital to Academy of Military Medical Sciences
Principal Investigator: hala Gabr, M.D. Cairo University
Study Chair: Wael Abou El-Kheir, M.D. Military Medical Academy

Responsible Party: Hala Gabr, Professor, Cairo University Identifier: NCT02108132     History of Changes
Other Study ID Numbers: SC-HEM
First Posted: April 9, 2014    Key Record Dates
Last Update Posted: April 9, 2014
Last Verified: April 2014

Keywords provided by Hala Gabr, Cairo University:
mesenchymal stem cells
in-vitro transdifferentiation

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn