Plerixafor for Stem Cell Mobilization in Normal Donors

Study Overview
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Brief Summary
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The goal of this clinical research study is to learn if treating stem cell donors with filgrastim (G-CSF) and plerixafor (Mozobil®) can cause them to produce a higher number of blood stem cells than filgrastim by itself. Researchers also want to learn if giving both of these drugs helps donors produce enough stem cells so that only 1 apheresis procedure needs to be performed. Researchers will study if using both drugs lowers the risk of the stem cell transplant recipients developing severe forms graft-versus-host disease (GVHD). GVHD is a condition in which transplanted tissue (such as blood stem cells) attacks the tissue of the recipient's body. The safety and effectiveness of this drug combination will also be studied. Filgrastim and plerixafor are both designed to help move or mobilize the stem cells from the bone marrow to the blood.

Detailed Description
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Stem Cell Transplant: You will receive blood stem cells from a donor on this study. You will sign a separate informed consent for the transplant procedure. Follow-Up Visits: About 1, 3, and 6 months after the transplant, an extra sample of bone marrow (about 2 teaspoons) will be collected at the same time as the standard of care bone marrow aspiration/biopsy procedures. This bone marrow sample will be tested to find out how well the donated stem cells have been accepted by your body. However, you will not have a separate bone marrow aspiration/biopsy only to collect bone marrow for this testing. When you return to the clinic at 6, 9, and 12 months for routine transplant follow-up visits, the study staff will try to get information on your health status from the clinic notes in your medical record. If this is not possible, you may receive a phone call from the study staff to check your health status. These calls will last about 10 minutes. Length of Treatment: You will be on study for about 1 year after the transplant (including follow-up contact by phone, if needed). You may be taken off study early if you are not able to follow study directions or if you decide to leave the study. This is an investigational study. Filgrastim is FDA approved for use in stem cell collection. Plerixafor is FDA approved for use in patients with multiple myeloma and non-Hodgkin's lymphoma. Up to 30 donor and recipient pairs will take part in this study. All will be enrolled at MD Anderson.

Official Title
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Plerixafor for Stem Cell Mobilization in Normal Donors

Conditions
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Blood And Marrow Transplantation

Intervention / Treatment
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* Drug: Filgrastim
* Drug: Plerixafor
* Procedure: Apheresis Procedure


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Donor eligibility: Age >/= 10 years. Donor eligibility: Related donors who met standard eligibility criteria and are willing to participate in this study. Donor eligibility: Able to provide informed consent. Recipient Eligibility: Patients who are scheduled to undergo an allogeneic related transplant and whose donors consented to participate in this study. Recipient Eligibility: Able to provide informed consent. Exclusion Criteria: 1) Donors who are on anti-coagulation or anti-platelet agents are not eligible.

Ages Eligible for Study
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Minimum Age: 10 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Filgrastim + Plerixafor<br>Each donor receives Filgrastim 5 µg/kg subcutaneously in the morning daily for 4 days. The dose of Filgrastim based on the donor's actual body weight. Donors will continue Filgrastim until completion of apheresis. Each donor receives Plerixafor 240 µg/kg subcutaneously in the evening on the fourth day of Filgrastim mobilization. The dose-volume of Plerixafor based on the donor's actual body weight. Apheresis procedure to start the morning of day 5, approximately 10 to 11 hours after the administration of Plerixafor. The apheresis procedure will start in the morning of day 5, approximately 10 to 11 hours after the administration of Plerixafor. The apheresis procedure may continue beyond day 1 until the target dose of 4x106 cluster of differentiation 34 (CD34+) cells/kg (recipient's weight) is obtained. | Drug: Filgrastim<br>* 5 µg/kg in the morning daily for 4 days.<br>* Other names: CSF-3;Drug: Plerixafor<br>* 240 µg/kg subcutaneously in the evening on the fourth day of Filgrastim mobilization.<br>* Other names: Mobozil;Procedure: Apheresis Procedure<br>* The apheresis procedure will start in the morning of day 5, approximately 10 to 11 hours after the administration of Plerixafor. The apheresis procedure may continue beyond day 1 until the target dose of 4x106 CD34+ cells/kg (recipient's weight) is obtained.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Summary of Most Common Toxicity: Donor Safety in Mobilizing Peripheral Blood Progenitor Cells (PBPC) | Primary safety endpoint is the development of any unexpected toxicity (any grade 2 or higher non-hematologic toxicity) in donors. The severity of the toxicity - adverse events (AEs) graded according to Common Terminology Criteria v4.0 (CTCAE). | 5 days | 
| Feasibility in Mobilizing PBPC in Donors: Number of Donors Reaching Stem Cell Target Collection on First Day of Collection Following Treatment of Filgrastim Plus Plerixafor | Study determined to be feasible if all donors were able to receive Plerixafor without developing any grade 2 or higher non-hematologic toxicity. Feasibility of the combination of Filgrastim, Granulocyte-colony stimulating factor (G-CSF) plus Plerixafor is to effectively mobilize CD34+ cells so that an adequate transplant (>4 x 10^6 CD34+ cells/kg) can be reliably collected with one apheresis for allogeneic HSCT. | 4 days | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Blood And Marrow Transplantation, Normal allogeneic donors, Stem cell mobilization, peripheral blood progenitor cells, PBPC, Allogeneic hematopoietic stem cell transplantation, HSCT, Donating blood stem cells, C-CSF, Neupogen, Filgrastim, Plerixafor, Mobozil, Apheresis