CompletedPhase 3

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

United States12 participantsStarted 2000-01

Plain-language summary

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.

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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 aller…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

Trial details

NCT IDNCT00005782

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

Sponsor typeNIH

Study typeINTERVENTIONAL

View on ClinicalTrials.gov More Sickle Cell Trait trials

Plain-language summary

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.