NCT00001529 · National Heart, Lung, and Blood Institute (NHLBI)
Improved Methods of Cell Selection for Bone Marrow Transplant Alternatives
What this study is about
Bone marrow transplants (BMT) are one form of treatment for disorders of the blood, including leukemia. However, because the procedure is often associated with potentially life-threatening reactions, it is usually reserved for patients with serious illnesses under the age of 60 years old. One serious reaction complicating bone marrow transplants is referred to as graft-versus-host disease (GVHD).
View original scientific description
Bone marrow transplants (BMT) are one form of treatment for disorders of the blood, including leukemia. However, because the procedure is often associated with potentially life-threatening reactions, it is usually reserved for patients with serious illnesses under the age of 60 years old. One serious reaction complicating bone marrow transplants is referred to as graft-versus-host disease (GVHD). GVHD is a potentially fatal incompatibility reaction. The reaction is caused by antigens found on the cells of the patient that are not present on the cells of the donor. The antigens are recognized by transplanted white blood cells (lymphocytes). These lymphocytes begin attacking the recipient s cells and tissues and may lead to death. In order to avoid GVHD, researchers have developed a technique using peripheral blood instead of bone marrow that allows transplantation of stem cells and removal of lymphocytes. Stem cells are the cells responsible for returning blood cell production to normal. Lymphocytes are the white blood cells that can cause GVHD. The technique requires two steps. In the first step blood cells are collected from donors who have received doses of a growth factor. The growth factor (granulocyte colony stimulating factor) is designed to increase the production of donor stem cells. In the second step white blood cell lymphocytes are removed from the collected blood, leaving only the stem cells. The main goal of this study is to develop and improve the method of processing cells that are collected after stimulation with growth factor (G-CSF), by removing the white blood cell lymphocytes which can cause graft-versus-host disease (GVHD) while keeping the stem cells necessary for healthy blood cell building. In addition, researchers are interested in studying whether giving G-CSF has an effect on lymphocyte function, which may influence the immune reactions occurring in bone marrow transplantation.
Interventions
DRUG
G-CSF
After medical clearance, volunteers will undergo outpatient mobilization with daily subcutaneous injections of filgrastim (G-CSF). The first dose will be administered at the NIH Clinical Center, with one-hour monitoring for immediate reactions. Volunteers may either return to NIH for daily injections or self-administer at home after training.
Primary outcome measures
Provide a source of primitive hematopoietic cells from mobilized blood for laboratory studies including optimization of culture and expansion, preservation techniques, gene transfer, analysis of cell surface antigens, & analysis of migra...
Time frame: End of Study
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Healthy individual aged 18 to 60 years.
- No active infection or history of recurrent infection.
- Normal renal function: creatinine \<1.5 mg/dL or estimated glomerular filtration rate (eGFR) \>=60 mL/min/1.73 m\^2, with no significant proteinuria. Normal liver function: bilirubin \<2.0 mg/dL (when unconjugated), transaminases \<2.0x ULN in the absence of known liver disease. Normal blood count: WBC 2,500-11,000/microliter, ANC \>1,500/microliter, platelets \>150,000/microliter, hemoglobin \>12.0 g/dL.
- Normal cardiovascular function, no history of chest pain, myocardial infarction, peripheral vascular disease, transient ischemic attack, or stroke.
- Healthy female subjects of childbearing age should have a negative serum pregnancy test with one week of beginning G-CSF administration.
- Female subjects should not be lactating.
- Subject must be eligible for normal blood donation. He or she must be tested negative for syphilis (RPR), hepatitis B and C (HBsAg, Anti-HBc, Anti-HCV), HIV, HTLV-1, West Nile virus, T. Cruzi and Babesia test.
- Subject must be able to comprehend the investigational nature of the study and provide informed consent to participate in the protocol.
- Antecubital veins must be adequate for peripheral access during apheresis. Potential participants must be screened by an apheresis nurse to check venous access before protocol entry.
Exclusion criteria
- Active viral, bacterial, fungal or parasite infection.
- Female with positive pregnancy test or lactating.
- Active or moderate-to-severe autoimmune disease that is currently treated or expected to require immunosuppressive therapy. Candidates with stable, well-controlled mild autoimmune disease may be considered on a case-by-case basis.
- Active or recent malignancy within the past 5 years. Individuals with remote (\>5 years) histories of low-risk malignancies in remission (e.g., localized prostate cancer) or treated basal cell carcinoma may be included.
- History of any hematologic disorders.
- History of clinically significant cardiovascular disease (e.g., symptomatic coronary artery disease, uncontrolled hypertension). Minor risk factors must be evaluated on a case-by-case basis (e.g., controlled hypertension).
- Any positive serum screening test as listed in eligibility.
- Allergy to G-CSF or bacterial E coli products.
- Administration of NSAID within. 5-7 days of starting the protocol, depending on drug half-life.
- History of G-CSF administration and leukapheresis within past 3 months.
Where
- Bethesda, Maryland
Collaborators
New York Genome Center
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Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 30, 2026 · Source of record for eligibility and locations