Blood And Marrow Transplantation
Conditions
Keywords
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
Brief summary
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
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.
Interventions
DRUGFilgrastim
5 µg/kg in the morning daily for 4 days.
DRUGPlerixafor
240 µg/kg subcutaneously in the evening on the fourth day of Filgrastim mobilization.
PROCEDUREApheresis Procedure
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.
Sponsors
Proteonomix, Inc.
M.D. Anderson Cancer Center
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
10 Years to No maximum
Healthy volunteers
Yes
Inclusion criteria
1. Donor eligibility: Age \>/= 10 years. 2. Donor eligibility: Related donors who met standard eligibility criteria and are willing to participate in this study. 3. Donor eligibility: Able to provide informed consent. 4. Recipient Eligibility: Patients who are scheduled to undergo an allogeneic related transplant and whose donors consented to participate in this study. 5. Recipient Eligibility: Able to provide informed consent.
Exclusion criteria
1\) Donors who are on anti-coagulation or anti-platelet agents are not eligible.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Summary of Most Common Toxicity: Donor Safety in Mobilizing Peripheral Blood Progenitor Cells (PBPC) | 5 days | 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). |
| 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 | 4 days | 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. |
Countries
United States
Participant flow
Recruitment details
Recruitment Period: October 16, 2013 to May 1, 2015. All recruitment done at The University of Texas MD Anderson Cancer Center.
Participants by arm
| Arm | Count |
|---|---|
| Donors Filgrastim + Plerixafor Filgrastim 5 µg/kg subcutaneously daily for 4 days until end of apheresis; Plerixafor 240 µg/kg subcutaneously on fourth day of Filgrastim mobilization; followed by Apheresis day 5 which may continue beyond day 1 until target dose of 4x10\^6 CD34+ cells/kg (recipient's weight) obtained. | 11 |
| Recipients Recipients received Plerixafor mobilized cells on day 0 of their designated treatment plan (following day 5 of donor's Plerixafor study specific treatment plan). | 11 |
| Total | 22 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Death | 0 | 2 |
| Overall Study | Not eligible due to White Blood Counts | 4 | 4 |
Baseline characteristics
| Characteristic | Donors Filgrastim + Plerixafor | Total | Recipients |
|---|---|---|---|
| Age, Continuous | 58 years | 58 years | 58 years |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 3 Participants | 6 Participants | 3 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 8 Participants | 16 Participants | 8 Participants |
| Region of Enrollment United States | 11 participants | 22 participants | 11 participants |
| Sex: Female, Male Female | 7 Participants | 12 Participants | 5 Participants |
| Sex: Female, Male Male | 4 Participants | 10 Participants | 6 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 5 / 11 | 7 / 11 |
| serious Total, serious adverse events | 0 / 11 | 0 / 11 |
Outcome results
Primary
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.
Time frame: 4 days
Population: Four participants did not receive Plerixafor due to a white blood cell count outside of the protocol specific window. It should be noted, the protocol threshold for white blood cell count was amended from 40,000 to 45,000.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Donors: Filgrastim + Plerixafor | 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 | 7 Participants |
Primary
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).
Time frame: 5 days
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Donors: Filgrastim + Plerixafor | Summary of Most Common Toxicity: Donor Safety in Mobilizing Peripheral Blood Progenitor Cells (PBPC) | Tachycardia | 1 adverse events |
| Donors: Filgrastim + Plerixafor | Summary of Most Common Toxicity: Donor Safety in Mobilizing Peripheral Blood Progenitor Cells (PBPC) | Nausea | 2 adverse events |
| Donors: Filgrastim + Plerixafor | Summary of Most Common Toxicity: Donor Safety in Mobilizing Peripheral Blood Progenitor Cells (PBPC) | Insomnia | 2 adverse events |
| Donors: Filgrastim + Plerixafor | Summary of Most Common Toxicity: Donor Safety in Mobilizing Peripheral Blood Progenitor Cells (PBPC) | Bone pain | 2 adverse events |
| Donors: Filgrastim + Plerixafor | Summary of Most Common Toxicity: Donor Safety in Mobilizing Peripheral Blood Progenitor Cells (PBPC) | Injection site reaction | 1 adverse events |
| Donors: Filgrastim + Plerixafor | Summary of Most Common Toxicity: Donor Safety in Mobilizing Peripheral Blood Progenitor Cells (PBPC) | Diarrhea | 1 adverse events |
| Donors: Filgrastim + Plerixafor | Summary of Most Common Toxicity: Donor Safety in Mobilizing Peripheral Blood Progenitor Cells (PBPC) | Chest Pain | 1 adverse events |