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Bone Marrow Stem Cells as a Source of Allogenic Hepatocyte Transplantation in Homozygous Familial Hypercholesterolemia

NCT00515307 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2008-08-29

No results posted yet for this study

Summary

Patients with homozygous familial hypercholesterolemia has very high serum cholesterol levels despite receiving lipid lowering drugs (e.g. 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.

Conditions

  • Hypercholesterolemia, Familial

Interventions

PROCEDURE

Cellular transplantation

600 million to 1 billion cells will be infused through the portal vein over 30 minutes. Infusion will be done one time.

Sponsors & Collaborators

  • University of Tehran

    lead OTHER

Principal Investigators

  • Reza Malekzadeh, M.D. · Digestive Disease Research Center, Medical Sciences/ Tehran University, Tehran, Iran

  • Hamid Goorabi, Phd · Royan Institute, Tehran, Iran

  • Mehdi Mohamadnejad, M.D. · Digestive Disease Research Center, Medical Sciences/ Tehran University, Tehran, Iran

  • Hossein Baharvand, Phd · Department of Stem Cells, Royan Institute, Tehran, Iran

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
FEMALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2007-06-30
Primary Completion
2008-05-31
Completion
2008-06-30

Countries

  • Iran

Study Locations

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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 NCT00515307 on ClinicalTrials.gov