Ignite Creation Date:
2024-05-05 @ 10:56 PM
Last Modification Date:
2024-10-26 @ 10:25 AM
Study NCT ID:
NCT01212055
Status:
RECRUITING
Last Update Posted:
2024-07-12
First Post:
2010-09-29
Brief Title:
Apheresis of Patients With Immunodeficiency
Sponsor:
National Cancer Institute NCI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Apheresis and CD34 Selection of Mobilized Peripheral Blood CD34 Cells From Patients With DOCK8 Deficiency LAD-1 and GATA2 Deficiency
Status:
RECRUITING
Status Verified Date:
2024-10-22
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:
Background
- Gene therapy is being investigated as a possible treatment for individuals with immunodeficiency diseases or other conditions that make it difficult to fight off infection Gene therapy avoids problems with donor identification and possible rejection of bone marrow transplant by using the patient s own modified blood cells to help treat the disease Researchers are interested in collecting stem cells from the blood of individuals with immunodeficiency diseases in order to use the cells to develop potential gene therapy treatments
Objectives
- To collect blood stem cells from patients with immunodeficiency diseases tto test our ability to correct the defects of these cells in the test tube
Eligibility
Individuals between 18 and 40 years of age with immunodeficiency diseases
Individuals with human immunodeficiency virus HIV will not be able to participate in this study
Design
Participants will provide an initial blood sample for disease screening such as hepatitis B and C syphilis or viruses like the Epstein-Barr virus herpes simplex virus or toxoplasmosis and to check kidney and liver function
Starting 5 days before blood donation participants will receive daily injections of a drug called G-CSF granulocyte colony stimulating factor or filgrastim which pushes stem cells out of the bone marrow and into the bloodstream Participants will receive the injections at the National Institutes of Health Clinical Center
On day 5 participants will have a single leukapheresis procedure to collect the stem cells from the blood
No additional treatment will be provided as part of this protocol The cells that are collected will be used fore experiments in the lab and will not be used to treat individuals with these diseases
- Gene therapy is being investigated as a possible treatment for individuals with immunodeficiency diseases or other conditions that make it difficult to fight off infection Gene therapy avoids problems with donor identification and possible rejection of bone marrow transplant by using the patient s own modified blood cells to help treat the disease Researchers are interested in collecting stem cells from the blood of individuals with immunodeficiency diseases in order to use the cells to develop potential gene therapy treatments
Objectives
- To collect blood stem cells from patients with immunodeficiency diseases tto test our ability to correct the defects of these cells in the test tube
Eligibility
Individuals between 18 and 40 years of age with immunodeficiency diseases
Individuals with human immunodeficiency virus HIV will not be able to participate in this study
Design
Participants will provide an initial blood sample for disease screening such as hepatitis B and C syphilis or viruses like the Epstein-Barr virus herpes simplex virus or toxoplasmosis and to check kidney and liver function
Starting 5 days before blood donation participants will receive daily injections of a drug called G-CSF granulocyte colony stimulating factor or filgrastim which pushes stem cells out of the bone marrow and into the bloodstream Participants will receive the injections at the National Institutes of Health Clinical Center
On day 5 participants will have a single leukapheresis procedure to collect the stem cells from the blood
No additional treatment will be provided as part of this protocol The cells that are collected will be used fore experiments in the lab and will not be used to treat individuals with these diseases
Detailed Description:
Background
Primary immunodeficiency diseases PID represent candidate genetic disorders for new therapeutic approaches Our laboratory is developing new therapies for individuals with PID using autologous CD34 hematopoietic stem cells HSC Newer therapies may circumvent problems with allogeneic HSC transplantation especially graft rejection and graft-versus-host-disease We are particularly interested in three PID Dedicator of CytoKinesis-8 DOCK8 deficiency Leukocyte Adhesion Deficiency type 1 LAD-1 and GATA2 Deficiency For all three diseases the gene has been cloned Testing new therapies for these diseases would be considerably enhanced by the acquisition of peripheral blood CD34 cells from individuals with these immunodeficiency diseases
Objectives
To provide a source of filgrastim or filgrastim biosimilar mobilized peripheral blood CD34 hematopoietic stem cells HSC for laboratory research studies for DOCK8 deficiency LAD-1 and GATA2 Deficiency
Eligibility
Individuals 18-40 years old with DOCK8 deficiency LAD-1 and GATA2 Deficiency who meet the eligibility requirements will be considered for this protocol
Design
Individuals 18-40 years old with DOCK8 deficiency LAD-1 and GATA2 Deficiency will receive five days of filgrastim or filgrastim biosimilar followed by a single apheresis CD34 cells will be selected and frozen in aliquots by the Cell Processing Section of the Department of Transfusion Medicine No treatments or investigational therapy will be administered on this protocol
Primary immunodeficiency diseases PID represent candidate genetic disorders for new therapeutic approaches Our laboratory is developing new therapies for individuals with PID using autologous CD34 hematopoietic stem cells HSC Newer therapies may circumvent problems with allogeneic HSC transplantation especially graft rejection and graft-versus-host-disease We are particularly interested in three PID Dedicator of CytoKinesis-8 DOCK8 deficiency Leukocyte Adhesion Deficiency type 1 LAD-1 and GATA2 Deficiency For all three diseases the gene has been cloned Testing new therapies for these diseases would be considerably enhanced by the acquisition of peripheral blood CD34 cells from individuals with these immunodeficiency diseases
Objectives
To provide a source of filgrastim or filgrastim biosimilar mobilized peripheral blood CD34 hematopoietic stem cells HSC for laboratory research studies for DOCK8 deficiency LAD-1 and GATA2 Deficiency
Eligibility
Individuals 18-40 years old with DOCK8 deficiency LAD-1 and GATA2 Deficiency who meet the eligibility requirements will be considered for this protocol
Design
Individuals 18-40 years old with DOCK8 deficiency LAD-1 and GATA2 Deficiency will receive five days of filgrastim or filgrastim biosimilar followed by a single apheresis CD34 cells will be selected and frozen in aliquots by the Cell Processing Section of the Department of Transfusion Medicine No treatments or investigational therapy will be administered on this protocol
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 |
|---|---|---|---|
| 10-C-0201 | None | None | None |