Mobilization and Handling of Stem Cells for Transplant From Healthy Volunteers With Sickle Cell Trait

March 3, 2008 updated by: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Leukapheresis of Volunteers With Sickle Cell Trait to Evaluate Mobilization of Stem Cells With Granulocyte Colony-Stimulating Factor and Stem Cell Collection and Storage for Allogeneic Transplantation

This study will examine the effects of granulocyte colony-stimulating factor (G-CSF) on bone marrow stem cells in healthy volunteers with sickle cell trait and determine if cells collected for transplantation from donors with sickle cell trait require special handling.

Stem cells, which the bone marrow produces, are responsible for making all the different kinds of blood cells. They are the cells used in bone marrow, or stem cell, transplantation. The drug G-CSF, which is a naturally occurring hormone, causes stem cells to mobilize-that is, to be released from the bone marrow and enter the blood stream. This drug is given to stem cell donors to increase the amount of cells that can be collected. Stem cell donors for patients with sickle cell disease are often healthy siblings of the patient who have a matching bone marrow type. Some siblings carry the sickle cell trait, however, and, even though they do not have sickle cell disease and their blood and bone marrow are normal, it is not known how their cells will react to G-CSF stimulation. Nor is it known if their stem cells require special methods of removal, processing or storing.

Healthy volunteers 18 years or older with sickle cell trait who have no history of sickle cell disease and no known medical problems may be eligible for this study. Participants will have a medical history and physical examination, including blood tests and urinalysis. They will receive injections of G-CSF under the skin once a day for 5 days. On the fifth day, stem cells will be collected through leukapheresis. In this procedure, whole blood is drawn from an arm vein, similar to donating whole blood. The blood then circulates through a cell separator machine, the stem cells are removed, and the rest of the blood is transfused back to the donor through a vein in the other arm.

The information gained from this study will be used to ensure the safety of stem cell donors with sickle cell trait and to better prepare stem cells for transplantation in sickle cell patients.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

Patients with sickle cell disease are increasingly becoming eligible for transplantation as the techniques of stem cell transplantation continue to improve, leading to less morbidity and mortality. However, the availability of a matched donor still remains one of the main obstacles. Given the inherited nature of this disease, many HLA-matched siblings of patients with sickle cell disease have sickle cell trait. Therefore, patients with sickle cell trait presumably have been used as donors for patients with sickle cell disease. However, there are no published data on whether special manipulations are required for the maintenance and storage of stem cells collected from patients with sickle cell trait. The effects of granulocyte-colony stimulating factor (G-CSF)-induced stem cell mobilization in these patients are also not known. Also, due to the potential of HbS gellation in red cells, the freezing of stem cells derived from individuals with sickle cell trait may require a more stringent red cell removal than is normally performed. We would like to evaluate these issues using volunteers with sickle cell trait prior to beginning our own transplant protocol for sickle cell disease.

Study Type

Interventional

Enrollment

12

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Maryland
      • Bethesda, Maryland, United States, 20892
        • National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Confirmed diagnosis of sickle cell trait.

Greater than age 17.

No active systemic infection or history of recurrent infections requiring intravenous antibiotic treatment .

Normal renal function: creatinine less than 2X normal, or proteinuria less than 1+.

Normal liver function: bilirubin less than 2X normal, transaminases within normal limits.

Normal blood count: WBC 3,000-10,000/mm3, granulocytes greater than 1,500/mm3, platelets greater than 150,000/mm3, hemoglobin greater than 12.5g/dL, MCV and MCHC within normal limits.

Female volunteers of childbearing age should have a negative serum pregnancy test within one week of beginning G-CSF administration.

Eligible for normal blood donation (i.e. tested negative for syphilis (RPR), hepatitis B and C (Hasbro, Anti-Hubcap, Anti-HCV), HIV, and HTLV-1.

Subject must give informed consent to participate in the protocol.

No history of sickle cell crises.

Children less than 18 years of age are excluded as is per routine for normal volunteer protocols.

No active systemic viral, bacterial, fungal, or parasitic infection.

No female volunteers with positive pregnancy test or who are lactating.

No history of autoimmune disease, such as rheumatoid arthritis and systemic lupus erythematosus.

No history of cancer excluding squamous or basal cell carcinoma of the skin.

No history of any malignant hematologic disorders.

No history of cardiovascular disease or related symptoms such as chest pain and shortness of breath..

No allergy to G-CSF or bacterial E. coli products.

No history of G-CSF administration or leukapheresis.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2000

Study Completion

July 1, 2002

Study Registration Dates

First Submitted

June 3, 2000

First Submitted That Met QC Criteria

June 3, 2000

First Posted (Estimate)

June 5, 2000

Study Record Updates

Last Update Posted (Estimate)

March 4, 2008

Last Update Submitted That Met QC Criteria

March 3, 2008

Last Verified

July 1, 2002

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 000060
  • 00-DK-0060

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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