Lymphoma, Non-Hodgkin, Multiple Myeloma
Conditions
Brief summary
This study will compare the results of stem cell mobilization using drugs called filgrastim (Neupogen) and plerixafor with the results of stem cell mobilization using drugs called XM02 filgrastim (Granix) and plerixafor.
Detailed description
This study will compare the results of stem cell mobilization using drugs called filgrastim (Neupogen) and plerixafor with the results of stem cell mobilization using drugs called XM02 filgrastim (Granix) and plerixafor. The FDA has determined that Granix is biosimilar to Neupogen, which means that they are similar in terms of quality, safety, and efficacy; however, Granix has not been tested in the context of stem cell mobilization to see how its effectiveness compares to that of Neupogen
Interventions
DRUGXM02 Filgrastim
DRUGFilgrastim
PROCEDUREApheresis
DRUGPlerixafor
PROCEDUREStem Cell Transplant
Sponsors
Washington University School of Medicine
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* At least 18 years of age * Diagnosis of multiple myeloma or non-Hodgkin lymphoma * Eligible for autologous transplantation * Adequate bone marrow function as defined as: * White Blood Cell Count ≥ 3.0x109/L * Absolute Neutrophil Count ≥ 1.5x109/L * Platelet Count ≥ 100x109/L * Able to understand and willing to sign an IRB-approved informed consent document * Surgically or biologically sterile or willing to practice acceptable birth control, as follows: * Women of childbearing potential must have a negative serum or urine pregnancy test within 7 days of Day 1 of study treatment. Women of childbearing potential must agree to abstain from sexual activity or use a medically approved contraceptive measure/regimen during and for 3 months after the treatment period. Acceptable methods of birth control include: barriers (condoms), oral contraceptive, intrauterine device (IUD), transdermal/implanted or injected contraceptives, and abstinence * Males must agree to abstain from sexual activity or agree to utilize a medically approved contraception method during and for 3 months after the treatment period. Acceptable methods of birth control include: barriers (condoms), oral contraceptive, intrauterine device (IUD), transdermal/implanted or injected contraceptives, and abstinence
Exclusion criteria
* Previous autologous stem cell collection * Known hypersensitivity to filgrastim, plerixafor, or E. coli derived products * Pregnant or breastfeeding
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Comparison of the Mean Day 5 CD34+Cells/kg Yield Between the Two Arms | Day 5 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Comparison of the Time to Neutrophil Engraftment Between the Two Arms | Up to Day 30 post-infusion | Time to neutrophil engraftment is measured by determining the first of 3 consecutive measurements of neutrophil count ≥ 500/µl following conditioning regimen-induced nadir. Patients who do not have neutrophil engraftment by Day 30 post-infusion of mobilized PBSC product will be considered a neutrophil engraftment failure. |
| Comparison of the Time to Platelet Engraftment Between the Two Arms | Up to Day 100 | Time to platelet engraftment is measured by determining the first of 3 consecutive measurements of platelet count ≥ 50,000/µl without platelet transfusion support for 7 days. Patients who do not have platelet engraftment by Day 100 post-infusion of mobilized PBSC product will be considered a platelet engraftment failure. |
| Comparison of the Readmission Rate Between the Two Arms | Up to Day 100 | Readmission rate is defined as the frequency at which patients are readmitted (after initial post-transplant discharge) following post-infusion of mobilized PBSC product for reasons other than progressive disease/relapse |
| Comparison of the Most Commonly Reported Adverse Events (Safety) Experienced by Participants Between the Two Arms | Up to 20 days after last apheresis (Day 25-Day 28) | -Adverse events will be assessed using CTCAE version 4.0 |
| Comparison of the Percentage of Patients Who Collect > 5.0x10^6 CD34+Cells/kg Following PBSC Mobilization Between the Two Arms | Up to Day 8 (total collection) | — |
| Comparison of the Percentage of Patients Who Collect > 2.0x10^6 CD34+Cells/kg in One Apheresis Procedure Following PBSC Mobilization Between the Two Arms | Day 5 | — |
| Comparison of the Percentage of Patients Who Collect > 5.0x10^6 CD34+Cells/kg in One Apheresis Procedure Following PBSC Mobilization Between the Two Arms | Day 5 | — |
| Comparison of the Percentage of Patients Who Collect > 2.0x10^6 CD34+Cells/kg Following PBSC Mobilization Between the Two Arms | Up to Day 8 (total collection) | — |
Countries
United States
Participant flow
Recruitment details
The study opened to participant enrollment on 08/20/2014 and closed to participant enrollment on 06/14/2016.
Participants by arm
| Arm | Count |
|---|---|
| XM02 Filgrastim (Granix) and Plerixafor XM02 Filgrastim (Granix) 10 mg/kg Days 1 through 4 (Days 5 through 8 may be required if target collection goal has not be met)
Plerixafor 0.24 mg/kg Day 4 (Days 5 through 7 may be required if target collection goal has not be met)
Apheresis on Day 5 (may need to be done on Days 6-8 if target collection goal has not been met) | 46 |
| Filgrastim (Neupogen) and Plerixafor Filgrastim (Neupogen) 10 mg/kg Days 1 through 4 (Days 5 through 8 may be required if target collection goal has not be met)
Plerixafor 0.24 mg/kg Day 4 (Days 5 through 7 may be required if target collection goal has not be met)
Apheresis on Day 5 (may need to be done on Days 6-8 if target collection goal has not been met) | 51 |
| Total | 97 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Protocol Violation | 3 | 0 |
Baseline characteristics
| Characteristic | Filgrastim (Neupogen) and Plerixafor | Total | XM02 Filgrastim (Granix) and Plerixafor |
|---|---|---|---|
| Age, Continuous | 58.5 years STANDARD_DEVIATION 9 | 60.6 years STANDARD_DEVIATION 8.9 | 62.9 years STANDARD_DEVIATION 8.3 |
| Diagnosis Multiple myeloma | 43 Participants | 86 Participants | 43 Participants |
| Diagnosis Non-Hodgkin's Lymphoma | 8 Participants | 11 Participants | 3 Participants |
| Prior Radiation | 13 Participants | 23 Participants | 10 Participants |
| Prior Therapies | 1.4 number of prior therapies STANDARD_DEVIATION 0.8 | 1.5 number of prior therapies STANDARD_DEVIATION 0.8 | 1.5 number of prior therapies STANDARD_DEVIATION 0.8 |
| 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 | 10 Participants | 7 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 1 Participants | 1 Participants | 0 Participants |
| Race (NIH/OMB) White | 47 Participants | 85 Participants | 38 Participants |
| Region of Enrollment United States | 51 participants | 97 participants | 46 participants |
| Sex: Female, Male Female | 21 Participants | 37 Participants | 16 Participants |
| Sex: Female, Male Male | 30 Participants | 60 Participants | 30 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 45 / 46 | 50 / 51 |
| serious Total, serious adverse events | 2 / 46 | 3 / 51 |
Outcome results
Primary
Comparison of the Mean Day 5 CD34+Cells/kg Yield Between the Two Arms
Time frame: Day 5
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| XM02 Filgrastim (Granix) and Plerixafor | Comparison of the Mean Day 5 CD34+Cells/kg Yield Between the Two Arms | 11.6 cells/kg | Standard Deviation 6.7 |
| Filgrastim (Neupogen) and Plerixafor | Comparison of the Mean Day 5 CD34+Cells/kg Yield Between the Two Arms | 10.0 cells/kg | Standard Deviation 6.8 |
Secondary
Comparison of the Most Commonly Reported Adverse Events (Safety) Experienced by Participants Between the Two Arms
-Adverse events will be assessed using CTCAE version 4.0
Time frame: Up to 20 days after last apheresis (Day 25-Day 28)
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| XM02 Filgrastim (Granix) and Plerixafor | Comparison of the Most Commonly Reported Adverse Events (Safety) Experienced by Participants Between the Two Arms | Bone pain | 19 participants |
| XM02 Filgrastim (Granix) and Plerixafor | Comparison of the Most Commonly Reported Adverse Events (Safety) Experienced by Participants Between the Two Arms | Thrombocytopenia | 18 participants |
| XM02 Filgrastim (Granix) and Plerixafor | Comparison of the Most Commonly Reported Adverse Events (Safety) Experienced by Participants Between the Two Arms | Anemia | 13 participants |
| XM02 Filgrastim (Granix) and Plerixafor | Comparison of the Most Commonly Reported Adverse Events (Safety) Experienced by Participants Between the Two Arms | Alkaline phosphatase increased | 10 participants |
| XM02 Filgrastim (Granix) and Plerixafor | Comparison of the Most Commonly Reported Adverse Events (Safety) Experienced by Participants Between the Two Arms | Nausea and/or vomiting | 10 participants |
| XM02 Filgrastim (Granix) and Plerixafor | Comparison of the Most Commonly Reported Adverse Events (Safety) Experienced by Participants Between the Two Arms | Leukocytosis | 8 participants |
| Filgrastim (Neupogen) and Plerixafor | Comparison of the Most Commonly Reported Adverse Events (Safety) Experienced by Participants Between the Two Arms | Nausea and/or vomiting | 10 participants |
| Filgrastim (Neupogen) and Plerixafor | Comparison of the Most Commonly Reported Adverse Events (Safety) Experienced by Participants Between the Two Arms | Bone pain | 22 participants |
| Filgrastim (Neupogen) and Plerixafor | Comparison of the Most Commonly Reported Adverse Events (Safety) Experienced by Participants Between the Two Arms | Alkaline phosphatase increased | 12 participants |
| Filgrastim (Neupogen) and Plerixafor | Comparison of the Most Commonly Reported Adverse Events (Safety) Experienced by Participants Between the Two Arms | Thrombocytopenia | 20 participants |
| Filgrastim (Neupogen) and Plerixafor | Comparison of the Most Commonly Reported Adverse Events (Safety) Experienced by Participants Between the Two Arms | Leukocytosis | 10 participants |
| Filgrastim (Neupogen) and Plerixafor | Comparison of the Most Commonly Reported Adverse Events (Safety) Experienced by Participants Between the Two Arms | Anemia | 18 participants |
Secondary
Comparison of the Percentage of Patients Who Collect > 2.0x10^6 CD34+Cells/kg Following PBSC Mobilization Between the Two Arms
Time frame: Up to Day 8 (total collection)
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| XM02 Filgrastim (Granix) and Plerixafor | Comparison of the Percentage of Patients Who Collect > 2.0x10^6 CD34+Cells/kg Following PBSC Mobilization Between the Two Arms | 100 percentage of participants |
| Filgrastim (Neupogen) and Plerixafor | Comparison of the Percentage of Patients Who Collect > 2.0x10^6 CD34+Cells/kg Following PBSC Mobilization Between the Two Arms | 100 percentage of participants |
Secondary
Comparison of the Percentage of Patients Who Collect > 2.0x10^6 CD34+Cells/kg in One Apheresis Procedure Following PBSC Mobilization Between the Two Arms
Time frame: Day 5
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| XM02 Filgrastim (Granix) and Plerixafor | Comparison of the Percentage of Patients Who Collect > 2.0x10^6 CD34+Cells/kg in One Apheresis Procedure Following PBSC Mobilization Between the Two Arms | 98 percentage of participants |
| Filgrastim (Neupogen) and Plerixafor | Comparison of the Percentage of Patients Who Collect > 2.0x10^6 CD34+Cells/kg in One Apheresis Procedure Following PBSC Mobilization Between the Two Arms | 90 percentage of participants |
Secondary
Comparison of the Percentage of Patients Who Collect > 5.0x10^6 CD34+Cells/kg Following PBSC Mobilization Between the Two Arms
Time frame: Up to Day 8 (total collection)
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| XM02 Filgrastim (Granix) and Plerixafor | Comparison of the Percentage of Patients Who Collect > 5.0x10^6 CD34+Cells/kg Following PBSC Mobilization Between the Two Arms | 96 percentage of participants |
| Filgrastim (Neupogen) and Plerixafor | Comparison of the Percentage of Patients Who Collect > 5.0x10^6 CD34+Cells/kg Following PBSC Mobilization Between the Two Arms | 96 percentage of participants |
Secondary
Comparison of the Percentage of Patients Who Collect > 5.0x10^6 CD34+Cells/kg in One Apheresis Procedure Following PBSC Mobilization Between the Two Arms
Time frame: Day 5
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| XM02 Filgrastim (Granix) and Plerixafor | Comparison of the Percentage of Patients Who Collect > 5.0x10^6 CD34+Cells/kg in One Apheresis Procedure Following PBSC Mobilization Between the Two Arms | 83 percentage of participants |
| Filgrastim (Neupogen) and Plerixafor | Comparison of the Percentage of Patients Who Collect > 5.0x10^6 CD34+Cells/kg in One Apheresis Procedure Following PBSC Mobilization Between the Two Arms | 76 percentage of participants |
Secondary
Comparison of the Readmission Rate Between the Two Arms
Readmission rate is defined as the frequency at which patients are readmitted (after initial post-transplant discharge) following post-infusion of mobilized PBSC product for reasons other than progressive disease/relapse
Time frame: Up to Day 100
Population: (2) participants in the XM02 Filgrastim (Granix) \& Plerixafor arm did not receive ASCT and were not evaluable for this outcome measure. (1) participant in the Filgrastim (Neupogen) \& Plerixafor arm did not receive ASCT and were not evaluable for this outcome measure.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| XM02 Filgrastim (Granix) and Plerixafor | Comparison of the Readmission Rate Between the Two Arms | 11 Participants |
| Filgrastim (Neupogen) and Plerixafor | Comparison of the Readmission Rate Between the Two Arms | 9 Participants |
Secondary
Comparison of the Time to Neutrophil Engraftment Between the Two Arms
Time to neutrophil engraftment is measured by determining the first of 3 consecutive measurements of neutrophil count ≥ 500/µl following conditioning regimen-induced nadir. Patients who do not have neutrophil engraftment by Day 30 post-infusion of mobilized PBSC product will be considered a neutrophil engraftment failure.
Time frame: Up to Day 30 post-infusion
Population: (2) participants in the XM02 Filgrastim (Granix) \& Plerixafor arm did not receive ASCT and were not evaluable for this outcome measure. (1) participant in the Filgrastim (Neupogen) \& Plerixafor arm did not receive ASCT and were not evaluable for this outcome measure.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| XM02 Filgrastim (Granix) and Plerixafor | Comparison of the Time to Neutrophil Engraftment Between the Two Arms | 11 days |
| Filgrastim (Neupogen) and Plerixafor | Comparison of the Time to Neutrophil Engraftment Between the Two Arms | 11.5 days |
Secondary
Comparison of the Time to Platelet Engraftment Between the Two Arms
Time to platelet engraftment is measured by determining the first of 3 consecutive measurements of platelet count ≥ 50,000/µl without platelet transfusion support for 7 days. Patients who do not have platelet engraftment by Day 100 post-infusion of mobilized PBSC product will be considered a platelet engraftment failure.
Time frame: Up to Day 100
Population: (2) participants in the XM02 Filgrastim (Granix) \& Plerixafor arm did not receive ASCT and were not evaluable for this outcome measure. (1) participant in the Filgrastim (Neupogen) \& Plerixafor arm did not receive ASCT and were not evaluable for this outcome measure.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| XM02 Filgrastim (Granix) and Plerixafor | Comparison of the Time to Platelet Engraftment Between the Two Arms | 18 days |
| Filgrastim (Neupogen) and Plerixafor | Comparison of the Time to Platelet Engraftment Between the Two Arms | 17.5 days |