Trial Outcomes & Findings for Collection of Transplant Stem Cells for Plasma Cell Myeloma (NCT NCT01547806)

Results Overview

Progenitor cells by apheresis was determined by flow cytometry. The stated goal was a minimum dose of 2x10EE\^6/kg following apheresis.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

49 participants

Primary outcome timeframe

Day 1 of apheresis

Results posted on

2018-03-07

Participant Flow

Our study was a relatively small pilot study, with the primary emphasis being on expanding standard stem cell therapy options for patients with multiple myeloma. The plerixafor plus G-CSF combination is used relatively ubiquitously, and several larger studies relating to plerixafor have been performed at large centers that treat multiple myeloma.

Participant milestones

Participant milestones
Measure	Hematopoietic Progenitor Cells (HPC) Subjects will undergo mobilization and collection of HPC, Apheresis for subsequent use in various clinical protocols. Filgrastim: Filgrastim will be administered as a single daily dose in a dose range of 10-16ug/kg/day subcutaneously for 5-7days Plerixafor: Plerixafor will be given on day 4, 8-10 hours before the day 5 apheresis, dose calculated according to patient weight Apheresis: The minimum cluster of differentiation 34 (CD34)+ cell dose that must be collected in order to proceed with a single autologous transplantation is 2 x 10\^6 CD34+ cells/kg.
Overall Study STARTED	49
Overall Study COMPLETED	49
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Collection of Transplant Stem Cells for Plasma Cell Myeloma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Hematopoietic Progenitor Cells (HPC) n=49 Participants Subjects will undergo mobilization and collection of HPC, Apheresis for subsequent use in various clinical protocols. Filgrastim: Filgrastim will be administered as a single daily dose in a dose range of 10-16ug/kg/day subcutaneously for 5-7days Plerixafor: Plerixafor will be given on day 4, 8-10 hours before the day 5 apheresis, dose calculated according to patient weight Apheresis: The minimum cluster of differentiation 34 (CD34)+ cell dose that must be collected in order to proceed with a single autologous transplantation is 2 x 106\^ CD34+ cells/kg.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	40 Participants n=5 Participants
Age, Categorical >=65 years	9 Participants n=5 Participants
Age, Continuous	56.99 years STANDARD_DEVIATION 8.45 • n=5 Participants
Sex: Female, Male Female	23 Participants n=5 Participants
Sex: Female, Male Male	26 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	48 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	7 Participants n=5 Participants
Race (NIH/OMB) White	40 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	49 Participants n=5 Participants

PRIMARY outcome

Timeframe: Day 1 of apheresis

Progenitor cells by apheresis was determined by flow cytometry. The stated goal was a minimum dose of 2x10EE\^6/kg following apheresis.

Outcome measures

Outcome measures
Measure	Hematopoietic Progenitor Cells (HPC) n=49 Participants Subjects will undergo mobilization and collection of HPC, Apheresis for subsequent use in various clinical protocols. Filgrastim: Filgrastim will be administered as a single daily dose in a dose range of 10-16ug/kg/day subcutaneously for 5-7days Plerixafor: Plerixafor will be given on day 4, 8-10 hours before the day 5 apheresis, dose calculated according to patient weight Apheresis: The minimum cluster of differentiation 34 (CD34)+ cell dose that must be collected in order to proceed with a single autologous transplantation is 2 x 10\^6 CD34+ cells/kg.
Percentage of Patients Achieving at Least 2 x 10^6 Cluster of Differentiation 34 (CD34) Cells Per Kg Recipient Body Weight on Day 1 of Apheresis	98 percentage of patients

PRIMARY outcome

Timeframe: Through Day 2 of collection

Progenitor cells by apheresis was determined by flow cytometry.

Outcome measures

Outcome measures
Measure	Hematopoietic Progenitor Cells (HPC) n=49 Participants Subjects will undergo mobilization and collection of HPC, Apheresis for subsequent use in various clinical protocols. Filgrastim: Filgrastim will be administered as a single daily dose in a dose range of 10-16ug/kg/day subcutaneously for 5-7days Plerixafor: Plerixafor will be given on day 4, 8-10 hours before the day 5 apheresis, dose calculated according to patient weight Apheresis: The minimum cluster of differentiation 34 (CD34)+ cell dose that must be collected in order to proceed with a single autologous transplantation is 2 x 10\^6 CD34+ cells/kg.
Percentage of Patients Requiring 2 Days to Achieve at Least 2 x 10^6 Cluster of Differentiation 34 (CD34) Cells Per Kg Recipient Body Weight	2 percentage of patients

PRIMARY outcome

Timeframe: Through Day 2 of collection

Progenitor cells by apheresis was determined by flow cytometry.

Outcome measures

Outcome measures
Measure	Hematopoietic Progenitor Cells (HPC) n=49 Participants Subjects will undergo mobilization and collection of HPC, Apheresis for subsequent use in various clinical protocols. Filgrastim: Filgrastim will be administered as a single daily dose in a dose range of 10-16ug/kg/day subcutaneously for 5-7days Plerixafor: Plerixafor will be given on day 4, 8-10 hours before the day 5 apheresis, dose calculated according to patient weight Apheresis: The minimum cluster of differentiation 34 (CD34)+ cell dose that must be collected in order to proceed with a single autologous transplantation is 2 x 10\^6 CD34+ cells/kg.
Average Number of Cluster of Differentiation 34 (CD34) Cells Collected (Per kg Recipient Body Weight (BW))	6.4 Number of CD34 cells per kg/BW (x 10EE6) Standard Deviation 2.8

PRIMARY outcome

Timeframe: Through Day 2 of collection

Progenitor cells by apheresis was determined by flow cytometry.

Outcome measures

Outcome measures
Measure	Hematopoietic Progenitor Cells (HPC) n=49 Participants Subjects will undergo mobilization and collection of HPC, Apheresis for subsequent use in various clinical protocols. Filgrastim: Filgrastim will be administered as a single daily dose in a dose range of 10-16ug/kg/day subcutaneously for 5-7days Plerixafor: Plerixafor will be given on day 4, 8-10 hours before the day 5 apheresis, dose calculated according to patient weight Apheresis: The minimum cluster of differentiation 34 (CD34)+ cell dose that must be collected in order to proceed with a single autologous transplantation is 2 x 10\^6 CD34+ cells/kg.
Median and Standard Deviation of Cluster of Differentiation 34 (CD34) Cells Collected (Per Kg Recipient Body Weight) (BW)	6.3 Number of CD34 cells per kg/BW (x 10EE6) Standard Deviation 2.8

PRIMARY outcome

Timeframe: Through Day 2 of collection

Progenitor cells by apheresis was determined by flow cytometry.

Outcome measures

Outcome measures
Measure	Hematopoietic Progenitor Cells (HPC) n=49 Participants Subjects will undergo mobilization and collection of HPC, Apheresis for subsequent use in various clinical protocols. Filgrastim: Filgrastim will be administered as a single daily dose in a dose range of 10-16ug/kg/day subcutaneously for 5-7days Plerixafor: Plerixafor will be given on day 4, 8-10 hours before the day 5 apheresis, dose calculated according to patient weight Apheresis: The minimum cluster of differentiation 34 (CD34)+ cell dose that must be collected in order to proceed with a single autologous transplantation is 2 x 10\^6 CD34+ cells/kg.
Range of Cluster of Differentiation 34 (CD34) Cells Collected	6.3 Number of CD34 cells per kg/BW (x 10EE6) Interval 1.7 to 17.0

PRIMARY outcome

Timeframe: Through Day 2 of collection

Progenitor cells by apheresis was determined by flow cytometry.

Outcome measures

Outcome measures
Measure	Hematopoietic Progenitor Cells (HPC) n=49 Participants Subjects will undergo mobilization and collection of HPC, Apheresis for subsequent use in various clinical protocols. Filgrastim: Filgrastim will be administered as a single daily dose in a dose range of 10-16ug/kg/day subcutaneously for 5-7days Plerixafor: Plerixafor will be given on day 4, 8-10 hours before the day 5 apheresis, dose calculated according to patient weight Apheresis: The minimum cluster of differentiation 34 (CD34)+ cell dose that must be collected in order to proceed with a single autologous transplantation is 2 x 10\^6 CD34+ cells/kg.
25th and 75th Percentile Values of Cluster of Differentiation 34 (CD34) Cells Collected 25th percentile	4.0 Number of CD34 cells per kg/BW (x 10EE6)
25th and 75th Percentile Values of Cluster of Differentiation 34 (CD34) Cells Collected 75th percentile	8.0 Number of CD34 cells per kg/BW (x 10EE6)

PRIMARY outcome

Timeframe: Indefinitely until a referring physician requests the product for standard clinical care or until product(s) is no longer needed and disposed of

The cryopreserved stem cells are stored under Good Manufacturing Practice (GMP) conditions in the National Institutes of Health (NIH) Department of Transfusion Medicine until a referring physician requests the products for standard clinical care.

Outcome measures

Outcome measures
Measure	Hematopoietic Progenitor Cells (HPC) n=49 Participants Subjects will undergo mobilization and collection of HPC, Apheresis for subsequent use in various clinical protocols. Filgrastim: Filgrastim will be administered as a single daily dose in a dose range of 10-16ug/kg/day subcutaneously for 5-7days Plerixafor: Plerixafor will be given on day 4, 8-10 hours before the day 5 apheresis, dose calculated according to patient weight Apheresis: The minimum cluster of differentiation 34 (CD34)+ cell dose that must be collected in order to proceed with a single autologous transplantation is 2 x 10\^6 CD34+ cells/kg.
Number of Hematopoietic Progenitor Cell (HPC) Apheresis Products Collected and Cryopreserved for Subsequent Use in Autologous Hematopoietic Cell Transplantation (AHCT) in Subjects With Plasma Cell Myeloma (PCM)	49 products

SECONDARY outcome

Timeframe: 27 months and 27 days

Here is the number of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.

Outcome measures

Outcome measures
Measure	Hematopoietic Progenitor Cells (HPC) n=49 Participants Subjects will undergo mobilization and collection of HPC, Apheresis for subsequent use in various clinical protocols. Filgrastim: Filgrastim will be administered as a single daily dose in a dose range of 10-16ug/kg/day subcutaneously for 5-7days Plerixafor: Plerixafor will be given on day 4, 8-10 hours before the day 5 apheresis, dose calculated according to patient weight Apheresis: The minimum cluster of differentiation 34 (CD34)+ cell dose that must be collected in order to proceed with a single autologous transplantation is 2 x 10\^6 CD34+ cells/kg.
Number of Participants With Serious and Non-Serious Adverse Events	16 Participants

SECONDARY outcome

Timeframe: One week of mobilization therapy

Percentage of patients that required Plerixafor injection in addition to G-CSF mobilization or none at all

Outcome measures

Outcome measures
Measure	Hematopoietic Progenitor Cells (HPC) n=49 Participants Subjects will undergo mobilization and collection of HPC, Apheresis for subsequent use in various clinical protocols. Filgrastim: Filgrastim will be administered as a single daily dose in a dose range of 10-16ug/kg/day subcutaneously for 5-7days Plerixafor: Plerixafor will be given on day 4, 8-10 hours before the day 5 apheresis, dose calculated according to patient weight Apheresis: The minimum cluster of differentiation 34 (CD34)+ cell dose that must be collected in order to proceed with a single autologous transplantation is 2 x 10\^6 CD34+ cells/kg.
Percentage of Patients That Required Plerixafor + Granulocyte-colony Stimulating Factor (G-CSF) And Only G-CSF (no Plerixafor) Plerixafor + G-CSF	47 percentage of patients
Percentage of Patients That Required Plerixafor + Granulocyte-colony Stimulating Factor (G-CSF) And Only G-CSF (no Plerixafor) Only G-CSF (no plerixafor)	53 percentage of patients

SECONDARY outcome

Timeframe: Through Day 2 of collection

Here is the percentage of patients that achieved or did not achieve 5 x 10\^6 CD34 cells/kg in a single apheresis.

Outcome measures

Outcome measures
Measure	Hematopoietic Progenitor Cells (HPC) n=49 Participants Subjects will undergo mobilization and collection of HPC, Apheresis for subsequent use in various clinical protocols. Filgrastim: Filgrastim will be administered as a single daily dose in a dose range of 10-16ug/kg/day subcutaneously for 5-7days Plerixafor: Plerixafor will be given on day 4, 8-10 hours before the day 5 apheresis, dose calculated according to patient weight Apheresis: The minimum cluster of differentiation 34 (CD34)+ cell dose that must be collected in order to proceed with a single autologous transplantation is 2 x 10\^6 CD34+ cells/kg.
Percentage of Patients That Achieved or Did Not Achieve 5 x 10^6 Cluster of Differentiation 34 (CD34) Cells/kg Achieved 5 x 10EE CD34 cells/kg	65 percentage of patients
Percentage of Patients That Achieved or Did Not Achieve 5 x 10^6 Cluster of Differentiation 34 (CD34) Cells/kg Did not achieve 5 x 10EE CD34 cells/kg	35 percentage of patients

SECONDARY outcome

Timeframe: Day one of collection

Percentage of patents achieving collecting the minimum but not optimal CD34 cell number.

Outcome measures

Outcome measures
Measure	Hematopoietic Progenitor Cells (HPC) n=49 Participants Subjects will undergo mobilization and collection of HPC, Apheresis for subsequent use in various clinical protocols. Filgrastim: Filgrastim will be administered as a single daily dose in a dose range of 10-16ug/kg/day subcutaneously for 5-7days Plerixafor: Plerixafor will be given on day 4, 8-10 hours before the day 5 apheresis, dose calculated according to patient weight Apheresis: The minimum cluster of differentiation 34 (CD34)+ cell dose that must be collected in order to proceed with a single autologous transplantation is 2 x 10\^6 CD34+ cells/kg.
Percentage of Patients That Achieved ≥ 2 x 10^6 But Less Than 5 x 10^6 Cluster of Differentiation 34 (CD34) Cells/kg (Day One Collection)	31 percentage of patients

SECONDARY outcome

Timeframe: Day 1 of apheresis

Population: No data from any participant was sufficiently collected to assess tumor cell contamination.

Flow cytometry to detect tumor contamination.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Day 1 of apheresis

Population: Because no data from any participant was sufficiently collected to assess tumor cell contamination, we were not able to determine the effect of plerixafor on this parameter.

Flow cytometry to detect tumor contamination.

Outcome measures

Outcome data not reported

Adverse Events

Hematopoietic Progenitor Cells

Serious events: 0 serious events

Other events: 16 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Hematopoietic Progenitor Cells n=49 participants at risk Subjects will undergo mobilization and collection of HPC, Apheresis for subsequent use in various clinical protocols. Filgrastim: Filgrastim will be administered as a single daily dose in a dose range of 10-16ug/kg/day subcutaneously for 5-7days Plerixafor: Plerixafor will be given on day 4, 8-10 hours before the day 5 apheresis, dose calculated according to patient weight Apheresis: The minimum cluster of differentiation 34 (CD34)+ cell dose that must be collected in order to proceed with a single autologous transplantation is 2 x 106 CD34+ cells/kg.
Musculoskeletal and connective tissue disorders Back pain	2.0% 1/49 • Number of events 1 • 27 months and 27 days
Nervous system disorders Headache	2.0% 1/49 • Number of events 1 • 27 months and 27 days
Vascular disorders Hypertension	2.0% 1/49 • Number of events 1 • 27 months and 27 days
Metabolism and nutrition disorders Hypoalbuminemia	2.0% 1/49 • Number of events 1 • 27 months and 27 days
Metabolism and nutrition disorders Hypokalemia	4.1% 2/49 • Number of events 2 • 27 months and 27 days
Metabolism and nutrition disorders Hypomagnesemia	2.0% 1/49 • Number of events 1 • 27 months and 27 days
Metabolism and nutrition disorders Hypophosphatemia	2.0% 1/49 • Number of events 1 • 27 months and 27 days
Investigations Lymphocyte count decreased	8.2% 4/49 • Number of events 4 • 27 months and 27 days
Musculoskeletal and connective tissue disorders Myalgia	2.0% 1/49 • Number of events 1 • 27 months and 27 days
Vascular disorders Phlebitis	2.0% 1/49 • Number of events 1 • 27 months and 27 days
Investigations Platelet count decreased	10.2% 5/49 • Number of events 5 • 27 months and 27 days

Additional Information

Dr. Jennifer Kanakry

National Cancer Institute

Phone: 301-451-3787

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place