Ignite Creation Date:
2024-05-05 @ 6:38 PM
Last Modification Date:
2024-10-26 @ 9:35 AM
Study NCT ID:
NCT00515307
Status:
COMPLETED
Last Update Posted:
2008-08-29
First Post:
2007-08-09
Brief Title:
Bone Marrow Stem Cells as a Source of Allogenic Hepatocyte Transplantation in Homozygous Familial Hypercholesterolemia
Sponsor:
University of Tehran
Organization:
University of Tehran
Study Overview
Official Title:
In-Vitro Transdifferentiation of Mesenchymal Stem Cells to Hepatocytes and Allogenic Transplantation of Hepatocytes to the Patients With Homozygous Familial Hypercholesterolemia
Status:
COMPLETED
Status Verified Date:
2008-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Patients with homozygous familial hypercholesterolemia has very high serum cholesterol levels despite receiving lipid lowering drugs eg statins etc Most of such patients die before the age of 20 due to myocardial infarction etc Orthotopic liver transplantation OLT is an effective treatment for that Hepatocyte transplantation is an alternative to OLT that may help to overcome the shortage of donor organs There have been reports of successful treatment of different kinds of metabolic liver disorders by hepatocyte transplantation The major problem with hepatocyte transplantation is that the source of hepatocytes is very limited Bone marrow stem cells are the potential source of hepatocytes In the in-vitro culture system successful and efficient transdifferentiation of mesenchymal stem cells into hepatocytes has been documented We have already shown that infusion of mesenchymal stem cells is safe and feasible in cirrhosis Mohamadnejad M et al Arch Iran Med 2007 In Press In this study 2 patients with homozygous familial hypercholesterolemia will be included The bone marrow of healthy volunteers with a normal lipid profile will be taken then bone marrow mesenchymal stem cells MSCs will be cultured and then MSCs will be trans-differentiate into hepatocytes and the cells will be infused through the portal vein into the patients The duration of follow up will be 6 months post-transplantation
Detailed Description:
Patients with homozygous familial hypercholesterolemia has very high serum cholesterol levels despite receiving lipid lowering drugs eg statins etc Most of such patients die before the age of 20 due to myocardial infarction etc Orthotopic liver transplantation OLT is an effective treatment and can decrease their serum cholesterol to near normal levels Bilheimer DW N Engl J Med 1984 3111658-64 Shortage of donor organ is a major problem for OLT Hepatocyte transplantation is an alternative to OLT that may help to overcome the shortage of donor organ There have been reports of successful treatment of different kinds of metabolic liver disorders such as Crigler Najjar Syndrome Fox IJ et al N Engl J Med 19983381422-6 Factor VII deficiency Dhawan A et al Transplantation 2004781812-4 Glycogen storage disease type Ia Muraca M et al Lancet 2002359317-8 etc by hepatocyte transplantation The major problem with hepatocyte transplantation is that the source of hepatocytes is very limited Bone marrow stem cells are the potential source of hepatocytes Although in the in-vivo system there is a controversy that if stem cells transdifferentiate into hepatocytes or fusion of stem cells and hepatocytes occur however in the in-vitro culture system successful and efficient transdifferentiation of mesenchymal stem cells into hepatocytes has been documented Lee KD et al Hepatology 2004401275-1284 Banas A et al Hepatology 200746219-28 We have already shown that infusion of mesenchymal stem cells is safe and feasible in cirrhosis Mohamadnejad M et al Arch Iran Med 2007 In Press In this study 2 female patients with homozygous familial hypercholesterolemia will be included The bone marrow of ABO compatible healthy male volunteers with a normal lipid profile will be taken then bone marrow mesenchymal stem cells MSCs will be cultured and then MSCs will be trans-differentiate into hepatocytes in the in-vitro culture system Then the cells will be infused through the portal vein into the patients The duration of follow up will be 6 months post-transplantation
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None