Ignite Creation Date:
2024-05-05 @ 11:21 AM
Last Modification Date:
2024-10-26 @ 9:02 AM
Study NCT ID:
NCT00001405
Status:
RECRUITING
Last Update Posted:
2024-07-12
First Post:
1999-11-03
Brief Title:
Recruitment and Apheresis Collection of Peripheral Blood Hematopoietic Stem Cells Mononuclear Cells and Granulocytes
Sponsor:
National Institute of Allergy and Infectious Diseases NIAID
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Recruitment and Apheresis Collection of Peripheral Blood Hematopoietic Stem Cells Mononuclear Cells and Granulocytes
Status:
RECRUITING
Status Verified Date:
2024-10-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The research goal of this study is to obtain CD34 hematopoietic stem cells HSC from peripheral blood andor bone marrow and Mononuclear Cells lymphocytes and monocytes and granulocytes grans from peripheral blood that will be used in the laboratory andor 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 andor 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 andor for clinical treatment in other approved protocols
Detailed Description:
The research goal of this study is to obtain CD34 hematopoietic stem cells HSC from peripheral blood andor bone marrow and Mononuclear Cells lymphocytes and monocytes and granulocytes grans from peripheral blood that will be used in the laboratory andor 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 andor 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 andor for clinical treatment in other approved protocols
Participants include 1 Adult patients with any primary immune deficiency PID or other blood disorder where collection is both for clinical use in another approved treatment protocol to benefit the patient and for laboratory research 2 Adult patients with any primary immune deficiency PID or blood disorder where collection is for laboratory research use only 3 Healthy adult volunteers where the collection is for laboratory research use only
The majority of subjects will have HSC collected from peripheral blood by apheresis Daily subcutaneous injections of G-CSF granulocyte colony stimulating factorfilgrastim for 5 to 6 days is a standard of care method used to mobilize HSC to the peripheral blood prior to apheresis and will be used for most subjects Plerixafor Mozobil is approved as standard of care for use in combination with G-CSF to mobilize HSC
Some adult patients will have a clinical scale aspiration collection of bone marrow to obtain HSC for clinical use in another approved treatment protocol Some adult participants may have a small sample needle aspiration collection of bone marrow obtained for laboratory research purposes only
Mononuclear cells andor Granulocytes gran will be collected from peripheral blood by apheresis following no stimulation using G-CSF alone or a using a combined single dose of G-CSF 480mcg and Dexamethasone 8mg prior to collection as is the standard of care pre-treatment used in the NIH DTM for collection of granulocyte transfusions from healthy donors
As noted HSC mononuclear cells and gran collection from patients with PID or other blood disorders may be used for laboratory research or may be designated for future clinical treatment of the patient under separate treatment protocol HSC mononuclear cells and gran collection from healthy volunteers will be designated entirely for laboratory research
HSC will be used for the following clinical purposes where clinical treatments would occur under separate IRB approved protocols 1 Autologous HSC from patients may be genetically modified and infused into the patient for treatment of an infection or the underlying disease Gene therapy 2 Autologous HSC from patients may serve as back up rescue product for patients undergoing matched unrelated donor transplantation or haploidential related or unrelated donor transplantation
Mononuclear Cells lymphocytes and monocytes and granulocytes will be used for the following clinical purposes 1 Autologous lymphocytes may be genetically modified and infused into the patient for treatment of an infection or the underlying disease Gene therapy 2 Autologous lymphocytes monocytes and granulocytes neutrophils may be transfected with mRNAs to transiently express a therapeutic protein for treatment of an infection or the underlying disease Gene therapy
HSC lymphocytes monocytes and granulocytes will be used for laboratory research studies that include Delineating the pathophysiology of inherited immune deficiencies Delineating the physiology of and improving engraftment of hematopoietic stem cells Determining how hematopoietic stem cells may be maintained in ex vivo culture without losing pluripotent potential Delineating the molecular mechanisms responsible for lineage specific differentiation Developing efficient methods for gene transfer into hematopoietic stem cells for corrective gene therapy Developing methods for restoration of function in defective peripheral blood monocytes andor granulocytes Further characterization of peripheral blood monocytes andor granulocytes from patients with PID
Participants include 1 Adult patients with any primary immune deficiency PID or other blood disorder where collection is both for clinical use in another approved treatment protocol to benefit the patient and for laboratory research 2 Adult patients with any primary immune deficiency PID or blood disorder where collection is for laboratory research use only 3 Healthy adult volunteers where the collection is for laboratory research use only
The majority of subjects will have HSC collected from peripheral blood by apheresis Daily subcutaneous injections of G-CSF granulocyte colony stimulating factorfilgrastim for 5 to 6 days is a standard of care method used to mobilize HSC to the peripheral blood prior to apheresis and will be used for most subjects Plerixafor Mozobil is approved as standard of care for use in combination with G-CSF to mobilize HSC
Some adult patients will have a clinical scale aspiration collection of bone marrow to obtain HSC for clinical use in another approved treatment protocol Some adult participants may have a small sample needle aspiration collection of bone marrow obtained for laboratory research purposes only
Mononuclear cells andor Granulocytes gran will be collected from peripheral blood by apheresis following no stimulation using G-CSF alone or a using a combined single dose of G-CSF 480mcg and Dexamethasone 8mg prior to collection as is the standard of care pre-treatment used in the NIH DTM for collection of granulocyte transfusions from healthy donors
As noted HSC mononuclear cells and gran collection from patients with PID or other blood disorders may be used for laboratory research or may be designated for future clinical treatment of the patient under separate treatment protocol HSC mononuclear cells and gran collection from healthy volunteers will be designated entirely for laboratory research
HSC will be used for the following clinical purposes where clinical treatments would occur under separate IRB approved protocols 1 Autologous HSC from patients may be genetically modified and infused into the patient for treatment of an infection or the underlying disease Gene therapy 2 Autologous HSC from patients may serve as back up rescue product for patients undergoing matched unrelated donor transplantation or haploidential related or unrelated donor transplantation
Mononuclear Cells lymphocytes and monocytes and granulocytes will be used for the following clinical purposes 1 Autologous lymphocytes may be genetically modified and infused into the patient for treatment of an infection or the underlying disease Gene therapy 2 Autologous lymphocytes monocytes and granulocytes neutrophils may be transfected with mRNAs to transiently express a therapeutic protein for treatment of an infection or the underlying disease Gene therapy
HSC lymphocytes monocytes and granulocytes will be used for laboratory research studies that include Delineating the pathophysiology of inherited immune deficiencies Delineating the physiology of and improving engraftment of hematopoietic stem cells Determining how hematopoietic stem cells may be maintained in ex vivo culture without losing pluripotent potential Delineating the molecular mechanisms responsible for lineage specific differentiation Developing efficient methods for gene transfer into hematopoietic stem cells for corrective gene therapy Developing methods for restoration of function in defective peripheral blood monocytes andor granulocytes Further characterization of peripheral blood monocytes andor granulocytes from patients with PID
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 94-I-0073 | None | None | None |