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NCT00001405
Recruitment and Apheresis Collection of Peripheral Blood Hematopoietic Stem Cells, Mononuclear Cells and Granulocytes
Conditions: Granuloma, Granulomatous Disease, Chronic, Leukocyte Disease, Genetic Disease, X-Linked, Genetic Disease, Inborn
Sex: All
Ages: 18 Years – 70 Years
Healthy volunteers: Yes
Enrollment: 850
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
Location: National Institutes of Health Clinical Center Bethesda Maryland
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.
Eligibility Criteria
* ELIGIBILITY CRITERIA:
Patients (Patients with a genetically defined PID or other blood disorder or clinical history consistent with PID or other blood disorder)
1. Patients will have a genetically defined PID or have a clinical history of recurrent infections or other problems suggestive of PID or other blood disorder, must be 18-70 years of age,
2. Some patients may have active bacterial or fungal infection at the time of study entry.
3. Preserved renal function (creatinine less than or equal to 2.5 mg/dL; less than or equal to 3+ proteinuria); preserved hepatic function (bilirubin less than or equal to 2.0 mg/dl); preserved hematologic function (WBC greater than or equal to1000/mm\^3;granulocytes greater than or equal to 500/mm\^3; platelets greater than or equal to 100,000; hematocrit greater than or equal to 25). Of note, patients with PID often have associated chronic thrombocytopenia. Patients with stable chronic thrombocytopenia will be eligible for collection, at the investigator s discretion, with the caveat that patients with platelet count \38 degress C), or WBC \>9000 are excluded.
2. Females who are pregnant or lactating as determined by history and/or pregnancy test are excluded.
3. Must be negative by routine blood donor eligibility testing criteria, including tests for syphilis (RPR) and TTV Recipient Transplant Panel (list is modified periodically, but may include hepatitis B and C, HIV and HTLV, T. cruzi) This does not apply to leukaphersis patients, as these tests are not required by DTM policy.
4. Someone without peripheral venous access in arm veins adequate for apheresis (healthy volunteers only).
5. If in the opinion of the investigator participation in this study places the healthy adult volunteer at undue risk.
Patients being considered for clinical scale bone marrow harvesting
1. Who are unable to lie prone during the bone marrow harvesting procedure.
2. Who are unable to tolerate general anesthesia during the bone marrow harvesting procedure.
Source: ClinicalTrials.gov (NCT00001405). StuddyBuddy aggregates publicly available trial information.