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

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.

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