RecruitingNot Applicable

Recruitment and Apheresis Collection of Peripheral Blood Hematopoietic Stem Cells, Mononuclear Cells and Granulocytes

United States850 participantsStarted 1994-02-27

Plain-language summary

The research goal of this study is to obtain CD34+ hematopoietic stem cells (HSC) from peripheral blood and/or bone marrow, and Mononuclear Cells (lymphocytes and monocytes), and granulocytes (grans) from peripheral blood that will be used in the laboratory and/or in the clinic to develop new cell therapies for patients with inherited or acquired disorders of immunity or blood cells. Development of novel cellular therapies requires access to HSC, Mononuclear Cells and/or granulocytes as the essential starting materials for the pre-clinical laboratory development of gene therapies and other engineered cell products. HSC or blood cells from healthy adult volunteers serve both as necessary experimental controls and also as surrogates for patient cells for clinical scale-up development. HSC or blood cells from patients serve both as the necessary experimental substrate for novel gene therapy and cellular engineering development for specific disorders and as pre-clinical scale up of cellular therapies. Collection of cells from adult patients collected in the NIH Department of Transfusion Medicine (DTM) under conditions conforming to accepted blood banking clinical practice may also be used directly in or cryopreserved for future use in other NIH protocols that have all required regulatory approvals allowing such use. In summary, the research goal of this protocol is the collection of HSC or blood cells that may be used for both laboratory research and/or for clinical treatment in other approved protocols.

Who can participate

Age range

18 Years – 70 Years

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.

Exclusion criteria

. Patients who are hemodynamically unstable (systolic or diastolic blood pressure fall of 20 mm Hg from the stable patient s baseline measurement) or requiring mechanical respiratory assistance are excluded.
. Female patients who are pregnant or lactating as determined by history and/or positive pregnancy test are excluded.
. Must be negative by routine blood donor eligibility testing criteria including tests for syphilis (RPR) and TTV Donor Transplant Panel testing (list is modified periodically, but may include hepatitis B and C, HIV and HTLV, T. cruzi). This does not apply to leukapheresis patients, as these tests are not required by DTM.
. XSCID patients do not make antibodies and false positives may occur because they receive periodic infusions of pooled donations of IVIG. We have observed positive anti-HBc testing in these patients. If this occurs, more specific DNA or antigen testing will be done and must be negative.
. Patients with CGD and other patients with autoimmunity as part of their PID phenotype may have false positive antibody tests and if this occurs more specific DNA or antigen testing will be done and must be negative.

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.

What they're measuring

Mobilize to peripheral blood and apheresis collect CD34+PBSC of PID patients for clinical treatment and in part (or entirely) for laboratory research

Timeframe: 1/1/2025

Mobilize to the peripheral blood and apheresis collect CD34+PBSC from healthy volunteers for research

Timeframe: 1/1/2025

Mobilize to peripheral blood mononuclear cells and granulocytes

Timeframe: 1/1/2025

bone marrow harvest

Timeframe: 1/1/2025

bone marrow by needle aspiration

Timeframe: 1/1/2025

laboratory studies of PBSC and bone marrow HSC

Timeframe: 1/1/2025

laboratory studies of mononuclear cells and granulocytes obtained from patients with PID or healthy volunteers

Timeframe: 1/1/2025

Trial details

NCT IDNCT00001405

SponsorNational Institute of Allergy and Infectious Diseases (NIAID)

Sponsor typeNIH

Study typeOBSERVATIONAL

Contact for this trial

Joanna L Peterson

(301) 346-9780 joanna.peterson@nih.gov

View on ClinicalTrials.gov More Granuloma trials

Plain-language summary

Who can participate

Age range

18 Years – 70 Years

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.

Exclusion criteria

. Patients who are hemodynamically unstable (systolic or diastolic blood pressure fall of 20 mm Hg from the stable patient s baseline measurement) or requiring mechanical respiratory assistance are excluded.
. Female patients who are pregnant or lactating as determined by history and/or positive pregnancy test are excluded.
. Must be negative by routine blood donor eligibility testing criteria including tests for syphilis (RPR) and TTV Donor Transplant Panel testing (list is modified periodically, but may include hepatitis B and C, HIV and HTLV, T. cruzi). This does not apply to leukapheresis patients, as these tests are not required by DTM.
. XSCID patients do not make antibodies and false positives may occur because they receive periodic infusions of pooled donations of IVIG. We have observed positive anti-HBc testing in these patients. If this occurs, more specific DNA or antigen testing will be done and must be negative.
. Patients with CGD and other patients with autoimmunity as part of their PID phenotype may have false positive antibody tests and if this occurs more specific DNA or antigen testing will be done and must be negative.

What they're measuring

Mobilize to peripheral blood and apheresis collect CD34+PBSC of PID patients for clinical treatment and in part (or entirely) for laboratory research

Timeframe: 1/1/2025

Mobilize to the peripheral blood and apheresis collect CD34+PBSC from healthy volunteers for research

Timeframe: 1/1/2025

Mobilize to peripheral blood mononuclear cells and granulocytes

Timeframe: 1/1/2025

bone marrow harvest

Timeframe: 1/1/2025

bone marrow by needle aspiration

Timeframe: 1/1/2025

laboratory studies of PBSC and bone marrow HSC

Timeframe: 1/1/2025

laboratory studies of mononuclear cells and granulocytes obtained from patients with PID or healthy volunteers

Timeframe: 1/1/2025