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.|
|ClinicalTrials.gov 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
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)|
|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|
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
- Assessment of Safety / Efficacy [ Time Frame: 2 years ]Assessment of vitality, life style and bleeding times of the Patients.
- Assessment of Coagulation Profile [ Time Frame: 2 years ]
All Participants Subjected to :
- APPT (activated partial thromboplastin time) weekly
- Factor 8 assay will be done weekly.
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 ClinicalTrials.gov identifier (NCT number): NCT02108132
|Contact: Hala Gabr, M.D.||+firstname.lastname@example.org|
|Military Medical Academy||Not yet recruiting|
|Cairo, Egypt, 11451|
|Principal Investigator: wael Abou El-kheir, M.D.|
|Principal Investigator:||hala Gabr, M.D.||Cairo University|
|Study Chair:||Wael Abou El-Kheir, M.D.||Military Medical Academy|