Stem Cell Mobilization for T Cell Lymphocytopenia
Trial Summary
Do I need to stop taking my current medications for the trial?
The trial protocol does not specify if you need to stop taking your current medications. However, you cannot participate if you are currently receiving lithium or have used systemic immune-modulatory agents in the past 6 months. It's best to discuss your specific medications with the trial team.
What data supports the idea that Stem Cell Mobilization for T Cell Lymphocytopenia is an effective treatment?
The available research shows that drugs like filgrastim, which are used for stem cell mobilization, are effective in other conditions such as multiple myeloma. Studies have compared different versions of filgrastim and found them effective in mobilizing stem cells, which is a key part of the treatment process. Although the research does not directly address T Cell Lymphocytopenia, the success in similar conditions suggests potential effectiveness.12345
What safety data is available for stem cell mobilization treatments like Filgrastim and its biosimilars?
Safety data for Filgrastim and its biosimilars, such as Neupogen, Zarxio, and Nivestym, primarily comes from their use in treating chemotherapy-induced neutropenia. A retrospective study from 1991 to 2018 identified 11,183 adverse drug reaction reports, with common side effects including pyrexia, myalgia, back pain, arthralgia, and bone pain. Differences in adverse events and efficacy were noted between the originator and biosimilars. Large epidemiologic studies are recommended to confirm these findings. Filgrastim and its biosimilars have been approved in the US and Europe, with Zarxio being the first biosimilar approved in the US in 2015.678910
Is the drug Filgrastim, Plerixafor a promising treatment for T Cell Lymphocytopenia?
Yes, Filgrastim and Plerixafor are promising because they help mobilize stem cells effectively, which is important for treatments like stem cell transplants. Studies show that these drugs work well together to collect the necessary stem cells, making them a valuable option for patients needing this type of therapy.1231112
What is the purpose of this trial?
Idiopathic CD4 lymphocytopenia (ICL) is a rare syndrome defined by consistently low CD4 T cell counts (\<300/mm3) without evidence of HIV infection or other known immunodeficiency. Patients with ICL are at risk for opportunistic infections typically associated with HIV/AIDS such as disseminated cryptococcal infection and severe human papillomavirus-related dysplasia. More than 20 years since the description of ICL, its etiology, pathogenesis, and management remain unclear. In this study we propose to administer the combination of granulocyte colony stimulating factor (G-CSF) and plerixafor to ICL patients and healthy volunteers with the objective of harvesting mobilized CD34+ hematopoietic progenitor cells (HPCs) by apheresis for transfer into immunocompromised mice and for study with in vitro assays. The mice studies would serve to investigate thymic development, survival, and trafficking of the mobilized human cells within murine lymphoid and non-lymphoid organs.HPCs are used for various therapies and there is an increasing use of agents that stimulate the bone marrow to produce progenitor cells and move them into the bloodstream where they may be harvested by apheresis. Not all patients respond to GCSF with vigorous HPC mobilization. The binding of chemokine receptor CXCR4 to stromal cell derived factor (SDF-1 or CXCL12) is an important interaction between a hematopoietic progenitor cell and its marrow environment. Plerixafor is a CXCR4 inhibitor which blocks binding to SDF-1 resulting in the release of hematopoietic progenitor cells (CD34+) into peripheral circulation. In pharmacodynamic studies of plerixafor in conjunction with G-CSF compared to G-CSF and placebo, a two-fold increase in CD34+ cell count was observed.Due to the important role CXCR4 plays in immune cell trafficking and its potential role in the pathogenesis of ICL, we propose as a secondary objective to assess peripheral CD4 T cell and CD34+ hematopoietic progenitor cell numbers and functions in ICL patients compared to controls following G-CSF and plerixafor administration.Study participants will be screened within 12 weeks prior to the study period. Eligible participants will receive G-CSF for 5 days with hospitalization on Day 4 for plerixafor injection followed by apheresis on Day 5. Participants will return for examinations and blood draws on Days 8 and 12....
Research Team
Irini Sereti, M.D.
Principal Investigator
National Institute of Allergy and Infectious Diseases (NIAID)
Eligibility Criteria
This trial is for adults aged 18-65 with a rare condition called Idiopathic CD4 Lymphocytopenia (ICL), which involves very low levels of certain immune cells. Participants must have documented ICL, be HIV negative, and not currently have any serious illnesses or conditions that could interfere with the study. They should also agree to use effective birth control during the study.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive G-CSF for 5 days with hospitalization on Day 4 for plerixafor injection followed by apheresis on Day 5
Follow-up
Participants return for examinations and blood draws to monitor safety and effectiveness
Treatment Details
Interventions
- Filgrastim
- Plerixafor
Filgrastim is already approved in United States, European Union, Canada, Japan for the following indications:
- Neutropenia
- Acute Myeloid Leukemia
- Bone Marrow Transplantation
- Chronic Neutropenia
- Leukemia
- Myelodysplastic Syndromes
- Neutropenia
- Acute Myeloid Leukemia
- Bone Marrow Transplantation
- Chronic Neutropenia
- Leukemia
- Myelodysplastic Syndromes
- Neutropenia
- Acute Myeloid Leukemia
- Bone Marrow Transplantation
- Chronic Neutropenia
- Leukemia
- Myelodysplastic Syndromes
- Neutropenia
- Acute Myeloid Leukemia
- Bone Marrow Transplantation
- Chronic Neutropenia
- Leukemia
- Myelodysplastic Syndromes
Find a Clinic Near You
Who Is Running the Clinical Trial?
National Institute of Allergy and Infectious Diseases (NIAID)
Lead Sponsor