What is this study trying to find out about blood and immune disorders?

This study is collecting specific blood cells called hematopoietic stem cells, mononuclear cells, and granulocytes to support research and clinical development of new cell therapies for immune and blood disorders. The research aims to advance laboratory and clinical treatment approaches by using these cells from patients and healthy volunteers. The National Institute of Allergy and Infectious Diseases (NIAID) leads this study to improve understanding and treatment options.

Who may be eligible for this blood and immune disorder clinical trial?

Adults aged 18 to 70 with a primary immune deficiency or blood disorder, as well as healthy adults aged 18 to 65, may be eligible. Participants should not be pregnant, have active infections, and must have adequate vein access for cell collection. Both patients and healthy volunteers must meet health criteria related to kidney, liver, and blood function. Please review eligibility criteria for more details.

Is there any cost to participate in this blood and immune disorder clinical trial?

Study-related care, including cell collection procedures and all trial visits, is provided at no cost. However, personal travel and related expenses may vary and are not guaranteed to be covered. Participants should plan accordingly for any costs associated with attending study visits.

Actively Recruiting

Age: 18Years - 70Years

All Genders

Healthy Volunteers

ID00001405

Collection of Blood and Bone Marrow Stem Cells and Immune Cells for Research and Clinical Treatments in Adults with Immune or Blood Disorders and Healthy Volunteers

Led by National Institute of Allergy and Infectious Diseases (NIAID) · Updated on 2026-08-11

850

Participants Needed

Research Sites

N/A

Total Duration

AI-Summary

What this Trial Is About

This research aims to collect CD34 hematopoietic stem cells HSC from peripheral blood andor bone marrow, along with mononuclear cells and granulocytes from peripheral blood. These cells will be used in laboratory studies and clinical treatments to develop new cell therapies for patients with inherited or acquired immune or blood disorders. The study includes adult patients with primary immune deficiencies or blood disorders and healthy adult volunteers to support the development and scale-up of gene therapies and cell engineering products. The main collection method for HSC is apheresis from peripheral blood after mobilization with daily subcutaneous injections of granulocyte colony stimulating factor G-CSF for 5 to 6 days, sometimes combined with Plerixafor. Some patients may also undergo bone marrow aspiration for clinical or research purposes. Mononuclear cells and granulocytes are collected from peripheral blood by apheresis, with or without pre-treatment using G-CSF alone or combined with dexamethasone, following standard care practices. Collected cells from patients may be used for clinical treatments under separate protocols or for laboratory research, while cells from healthy volunteers are used for research only. Participants provide blood andor bone marrow samples that may be stored or modified for research. Assessments include monitoring cell mobilization and collection through apheresis and bone marrow harvests. The collected cells contribute to laboratory studies of immune deficiencies, stem cell biology, and gene therapy development. The study evaluates the ability to mobilize and collect these cells safely and effectively. Participation involves collection visits and allows for ongoing research and potential clinical use of the samples in approved protocols.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 18Years - 70Years

All Genders

Healthy Volunteers

Eligibility Criteria

You may qualify if you...

Patients aged 18 to 70 with a genetically defined primary immune deficiency (PID) or clinical history consistent with PID or other blood disorder
Patients may have active bacterial or fungal infections at study entry
Preserved kidney, liver, and blood function within specified laboratory limits
Patients of childbearing potential must use effective contraception and have a negative pregnancy test within one week before G-CSF administration
Willingness to allow blood cell samples to be stored and modified to induced pluripotent stem (iPS) cells
Healthy adults aged 18 to 65 without active or recurrent infections
Healthy volunteers must weigh at least 50 kg
Normal kidney, liver, and blood function within specified laboratory limits
Healthy female volunteers of childbearing potential must use effective contraception and have a negative pregnancy test within one week before G-CSF administration
Adult subjects with known genetic mutations may participate as healthy volunteers if other criteria are met

You will not qualify if you...

Patients who are hemodynamically unstable or require mechanical respiratory assistance
Female patients who are pregnant or lactating
Patients positive for certain infections or antibodies unless specific DNA or antigen tests are negative
Healthy volunteers with active bacterial, fungal, or viral infection or fever above 38°C
Healthy females who are pregnant or lactating
Healthy volunteers who do not have adequate peripheral venous access in arm veins for apheresis
Healthy volunteers deemed by the investigator to be at undue risk
Patients unable to lie prone or tolerate general anesthesia for bone marrow harvesting

Research Team

Joanna L Peterson

Harry L Malech, M.D.

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