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

NCT02108132 · Status: UNKNOWN · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2014-04-09

No results posted yet for this study

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.

Conditions

Interventions

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

Sponsors & Collaborators

  • Affiliated Hospital to Academy of Military Medical Sciences

    collaborator OTHER

  • Cairo University

    lead OTHER

Principal Investigators

  • hala Gabr, M.D. · Cairo University

  • Wael Abou El-Kheir, M.D. · Military Medical Academy, Bulgaria

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Max Age
40 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-08-31
Primary Completion
2015-08-31
Completion
2016-02-29

Countries

  • Egypt

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02108132 on ClinicalTrials.gov