Collecting Blood Cells for Research on Immune and Blood Disorders
This study is collecting different types of blood cells, including hematopoietic stem cells (HSC), mononuclear cells, and granulocytes, from both patients and healthy volunteers. These cells are essential starting materials for laboratory research to develop new cell therapies for people with inherited or acquired immune system or blood disorders. The goal is to create new treatments like gene therapies. You may be able to join if you are between 18 and 70 years old and have a genetically defined primary immunodeficiency (PID) or another blood disorder, or a history of infections suggesting one. Healthy volunteers within the same age range can also participate. The study aims to collect these cells by January 1, 2025.
- Study design
- This is an observational study planning to enroll 850 participants. It is collecting blood cells from both patients with certain immune or blood disorders and healthy volunteers.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- The primary goals of collecting cells are measured by January 1, 2025.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Recruitment and Apheresis Collection of Peripheral Blood Hematopoietic Stem Cells, Mononuclear Cells and Granulocytes
At a glance
Conditions
Where it's being run
1 sites across 1 statesStudy leadership
- Harry L Malech, M.D. · PRINCIPAL_INVESTIGATOR · National Institute of Allergy and Infectious Diseases (NIAID)
Who to contact
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What this trial measures
- Mobilize to peripheral blood and apheresis collect CD34+PBSC of PID patients for clinical treatment and in part (or entirely) for laboratory research1/1/2025
1\. To efficiently and safely mobilize to the peripheral blood and apheresis collect CD34+ PBSC patients with any inherited primary immune deficiency (PID) or other blood disorders where PBSC from these patients may be designated entirely or in part for future clinical treatment of the patient
- Mobilize to the peripheral blood and apheresis collect CD34+PBSC from healthy volunteers for research1/1/2025
2\. To efficiently and safely mobilize to the peripheral blood and apheresis collect CD34+ PBSC from healthy volunteers, which will be designated entirely for laboratory research.
- Mobilize to peripheral blood mononuclear cells and granulocytes1/1/2025
3.To efficiently and safely collect peripheral blood mononuclear cells and granulocytes from adult subjects with chronic granulomatous disease or other PID affecting granulocyte function, or from adult healthy donors volunteers for preclinical cell therapy studies, following either no stimulation, G-CSF or dexamethasone alone, or combined single dose G-CSF and dexamethasone as per DTM protocol depending on the number of cells desired.
- bone marrow harvest1/1/2025
4.To perform a bone marrow harvest by needle aspiration using standard of medical care methods from adult patients with PID or other blood disorder in sufficient quantity cryopreserved in CC DTM to serve as the source of autologous HSC for gene transfer ex vivo transduction if that patient is enrolled in a separate IRB approved protocol of gene transfer treatment designed to treat the genetically defined type of PID affecting that patient; and the patient has been unable to mobilize and have collected sufficient PBSC to qualify for treatment in that protocol.
- bone marrow by needle aspiration1/1/2025
5.To obtain from adult patients with PID or other blood disorder and from adult healthy volunteers a small sample of bone marrow by needle aspiration from bone marrow aspirates, where the purpose for this aspirate is to obtain bone marrow HSC for laboratory research comparing these cells to the biological properties of PBSC.
- laboratory studies of PBSC and bone marrow HSC1/1/2025
6.To conduct laboratory studies of PBSC and bone marrow HSC obtained from patients with PID or healthy volunteers in order to: study the functional properties of immune cells arising from hematopoietic stem cells in ex vivo culture systems and in NOD/SCID or NOG mice capable of supporting a human hematopoietic stem cellchimeric marrow xenograft.
- laboratory studies of mononuclear cells and granulocytes obtained from patients with PID or healthy volunteers1/1/2025
7.To conduct laboratory studies of mononuclear cells and granulocytes obtained from patients with PID or healthy volunteers in order to: Develop effective methods to restore immunologic function to defective granulocytes/mononuclear cells utilizing mRNA electroporation. Develop methodology of using autologous corrected granulocytes/mononuclear cells for the treatment of infections in immunedeficient patients.