Blood Collection From Healthy Volunteers and Patients for the Production of Clinical Grade Induced Pluripotent Stem Cell (iPSC) Products

Induced Pluripotent Stem Cells Clinical Trial

Official title: Collection of Blood From Healthy Volunteers and Clinical Research Subjects for the Production of Clinical Grade Induced Pluripotent Stem Cell (iPSC) Products

Status Withdrawn

Clinical Trial Summary

Background:

• Pluripotent stem cells are cells that can be converted to any type of cell (muscle, nerve, liver, etc.). Researchers are collecting blood samples to develop way to make a special cell product called induced pluripotent stem cells (iPSCs). iPSCs may be used to repair or replace organs and tissues, produce blood and immune cells for transfusion, or correct inherited immune and blood diseases. iPSCs can be made from many kinds samples, such as skin, blood, or hair. This is different from embryonic stem cells, which can only be derived from embryos.

Objectives:

• To develop new methods to make iPSCs; to identify better ways to collect, produce, and grow them; and to make an iPSC bank.

Eligibility:

• Healthy adults and adults already in a treatment protocol who have a medical condition that could be treated with iPSCs.

Design:

• All participants will be screened with questionnaire, physical exam, and blood and HIV tests.

• All participants:

• Will donate 4 tablespoons of blood. It will be taken by needle from a vein in their arm.

• Participants already in another protocol:

• Will have their blood collected, separated in a lab, and iPSCs grown in large numbers. Some may have their sample given back to them as a treatment.

• Samples will be kept in the study up to 5 years or until a participant withdraws from the study or becomes ineligible to participate, or the study is closed. If participants have not withdrawn their consent, they may be contacted in the future to donate again.

Clinical Trial Description

Induced pluripotent stem cells (iPSCs) can be produced from small quantities of blood. Clinical protocols to treat several diseases using iPSCs or cells derived from iPSCs are being developed by intramural investigators, i.e., investigators in National Institute of Allergy and Infectious Diseases (NIAID) and National Eye Institute (NEI). This clinical protocol will allow personnel in the Cell Processing Service (CPS) of the Department of Transfusion Medicine (DTM) to collect blood from healthy donors or patients with diseases targeted in IRB-approved treatment protocols to manufacture iPSCs or iPSC derived products to support these intramural investigators. CPS DTM will submit a Drug Master File (DMF) to the US Food and Drug Administration (FDA) for the manufacture and testing of these products. When iPSCs are used clinically in some cases the donors will be the recipients of the iPSCs, or the ex vivo expanded and differentiated iPSCs (autologous therapy). In other cases, healthy subjects will serve as donors of the blood used as starting material for manufacturing iPSCs (allogeneic therapy) where no matching of HLA or other antigens is required between the healthy blood donor and the iPSC recipient. While methods to produce and expand iPSCs have been described, they must be modified periodically to meet changing Good Manufacturing Practices (GMP) requirements for manufacturing clinical cell and gene therapies, therefore peripheral blood for iPSCs from normal donors is needed for research and development. ;

Related Conditions & MeSH terms

• Induced Pluripotent Stem Cells

• NCT number: NCT02056613
• Study type: Observational
• Source: National Institutes of Health Clinical Center (CC)
• Status: Withdrawn
• Start date: February 5, 2014
• Completion date: November 1, 2018