Graft-Versus-Host DiseaseNCT00001529
Improved Methods of Cell Selection for Bone Marrow Transplant Alternatives
An observational study of Graft-Versus-Host Disease and Graft-versus-leukemia, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 60 yearsHealthy volunteers can take part
- Conditions the study is for
- Graft-Versus-Host Disease, Graft-versus-leukemia, Donor Apheresis
- What is being tested?
- G-CSF
- Type of treatment
- Injection or infusion
- How many people
- 500 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Mar 1996to not given
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- All sexes
- Healthy volunteers accepted
- Adult
Study population: Healthy Volunteers
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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).
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR), 800-411-1222 ext. TTY dial 711, ccopr@nih.gov
About this study
Official title: Use of Granulocyte Colony Stimulating Factor (G-CSF) Mobilized Leukapheresis Collections From Healthy Volunteers to Develop Improved Methods of Stem Cell and Lymphocyte Selection for Allogeneic Transplantation
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.
Read more
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.
Read the detailed description
The NHLBI Stem Cell Transplantation program is exploring ways to make allogeneic transplantation safer and more widely applicable. Prior NHLBI transplant protocols have evaluated the strategy of using T cell depleted marrow transplants followed by delayed lymphocyte add-back to control or prevent GVHD while conserving useful donor immune function against residual leukemia and infectious agents. Over the past ten years, a number of increasingly efficient methods have been used to deplete T cells but retain stem cells, and we have shown the safety and utility of the delayed T cell add-back approach. We have also found a positive relationship between the administration of higher CD34+ cell doses and outcome. Investigation of highly purified grafts with the add-back of specific T cell populations is ongoing, and the ability to test new purification approaches and devices on clinical-scale PBSC products is critical to the continued development of new transplantation approaches in our program. This requires testing the approaches on G-CSF mobilized PBSCs collected by apheresis from healthy donors since this is the cell source that will be used in all clinical allogeneic transplantation protocols in our program.
Therefore, the primary intent of this protocol is to provide a mechanism for mobilizing, collecting, storing, and analyzing G-CSF mobilized apheresis samples from healthy volunteers. Cells will be used to develop a method of processing the cells that are collected after stimulation with G-CSF, by removing the lymphocytes, which can mediate GVHD while retaining the stem cells which are necessary for hematopoietic reconstitution. At the same time, we will study whether G-CSF administration has an effect on lymphocyte, function which may influence the immune reactions occurring in allogeneic bone marrow transplantation. Furthermore, the CD34+ cells collected will be a valuable resource for experimental studies of lymphocyte-stem cell interactions in our laboratory.
- Being tested
- G-CSF
- Keywords
- G-CSF, Donor Apheresis, Graft Versus Host Disease, graft versus leukemia, Natural History, Normal Volunteer
- Medical subject headings
- Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
G-CSFDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Group 1 | Healthy volunteers Receives: Drug: G-CSF |
What is being measured
The primary outcome is the main question the study is designed to answer.
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...
Measured: End of Study
Secondary outcomes (2)
Use the cells to develop a reliable technique for T cell depletion of peripheral blood stem cell transplants, which conserves sufficient CD34 cells for safe engraftment while minimizing the risk of GVHD.
Study the effect of G-CSF on lymphocyte subsets and helper cytotoxic function.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Nov 1999 (estimated)
- Study start18 Mar 1996
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Graft-Versus-Host DiseaseStudies of the same condition, open and under way.
- More trials run by National Heart, Lung, and Blood Institute (NHLBI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 500 participants (estimated)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Collaborators
- New York Genome Center
- Responsible party
- Sponsor
- Investigators
- Andre Larochelle, M.D., Principal investigator, National Heart, Lung, and Blood Institute (NHLBI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00001529
- Sponsor's protocol ID
- 960049
- Other IDs
- 96-H-0049
- Status last verified
- 4 Nov 2026
- First submitted
- 3 Nov 1999
- Last updated
- 6 Nov 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Goldman J. Peripheral blood stem cells for allografting. Blood. 1995 Mar 15;85(6):1413-5. No abstract available.
PubMed 7534129Grigg AP, Roberts AW, Raunow H, Houghton S, Layton JE, Boyd AW, McGrath KM, Maher D. Optimizing dose and scheduling of filgrastim (granulocyte colony-stimulating factor) for mobilization and collection of peripheral blood progenitor cells in normal volunteers. Blood. 1995 Dec 15;86(12):4437-45.
PubMed 8541532Cottler-Fox M, Cipolone K, Yu M, Berenson R, O'Shaughnessy J, Dunbar C. Positive selection of CD34+ hematopoietic cells using an immunoaffinity column results in T cell-depletion equivalent to elutriation. Exp Hematol. 1995 Apr;23(4):320-2.
PubMed 7534711
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Use of Granulocyte Colony Stimulating Factor (G-CSF) Mobilized Leukapheresis Collections From Healthy Volunteers to Develop Improved Methods of Stem Cell and Lymphocyte Selection for Allogeneic Transplantation
- ClinicalTrials.gov ID
- NCT00001529
- Acronym / sponsor ID
- 960049
- Keywords
- G-CSF, Donor Apheresis, Graft Versus Host Disease, graft versus leukemia, Natural History, Normal Volunteer
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 500 participants (estimated)
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Collaborators
- New York Genome Center
- Study start
- 18 Mar 1996 (actual)
- First posted
- 4 Nov 1999 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The NHLBI Stem Cell Transplantation program is exploring ways to make allogeneic transplantation safer and more widely applicable. Prior NHLBI transplant protocols have evaluated the strategy of using T cell depleted marrow transplants followed by delayed lymphocyte add-back to control or prevent GVHD while conserving useful donor immune function against residual leukemia and infectious agents. Over the past ten years, a number of increasingly efficient methods have been used to deplete T cells but retain stem cells, and we have shown the safety and utility of the delayed T cell add-back approach. We have also found a positive relationship between the administration of higher CD34+ cell doses and outcome. Investigation of highly purified grafts with the add-back of specific T cell populations is ongoing, and the ability to test new purification approaches and devices on clinical-scale PBSC products is critical to the continued development of new transplantation approaches in our program. This requires testing the approaches on G-CSF mobilized PBSCs collected by apheresis from healthy donors since this is the cell source that will be used in all clinical allogeneic transplantation protocols in our program.
Therefore, the primary intent of this protocol is to provide a mechanism for mobilizing, collecting, storing, and analyzing G-CSF mobilized apheresis samples from healthy volunteers. Cells will be used to develop a method of processing the cells that are collected after stimulation with G-CSF, by removing the lymphocytes, which can mediate GVHD while retaining the stem cells which are necessary for hematopoietic reconstitution. At the same time, we will study whether G-CSF administration has an effect on lymphocyte, function which may influence the immune reactions occurring in allogeneic bone marrow transplantation. Furthermore, the CD34+ cells collected will be a valuable resource for experimental studies of lymphocyte-stem cell interactions in our laboratory.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Group 1 | – | Drug: G-CSFHealthy volunteers |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
- Study population
- Healthy Volunteers
Key inclusion criteria (11 listed)
- 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.
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| G-CSFAfter 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. | Drug | Group 1 |
Primary outcome
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
2 secondary outcomes
Use the cells to develop a reliable technique for T cell depletion of peripheral blood stem cell transplants, which conserves sufficient CD34 cells for safe engraftment while minimizing the risk of GVHD.
Study the effect of G-CSF on lymphocyte subsets and helper cytotoxic function.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Nov 2026.
- Background: PubMed 7534129Goldman J. Peripheral blood stem cells for allografting. Blood. 1995 Mar 15;85(6):1413-5. No abstract available.
- Background: PubMed 8541532Grigg AP, Roberts AW, Raunow H, Houghton S, Layton JE, Boyd AW, McGrath KM, Maher D. Optimizing dose and scheduling of filgrastim (granulocyte colony-stimulating factor) for mobilization and collection of peripheral blood progenitor cells in normal volunteers. Blood. 1995 Dec 15;86(12):4437-45.
- Background: PubMed 7534711Cottler-Fox M, Cipolone K, Yu M, Berenson R, O'Shaughnessy J, Dunbar C. Positive selection of CD34+ hematopoietic cells using an immunoaffinity column results in T cell-depletion equivalent to elutriation. Exp Hematol. 1995 Apr;23(4):320-2.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Andre Larochelle, M.D. principal investigator
National Heart, Lung, and Blood Institute (NHLBI)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Graft-Versus-Host Disease and Graft-versus-leukemia, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 6 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Richard A Gustafson, R.N. ((301) 402-5822, richard.gustafson@nih.gov), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00001529, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Nov 2026.