Trial Outcomes & Findings for Mobilization of Stem Cells With AMD3100 (Plerixafor) and G-CSF in Non-Hodgkin's Lymphoma and Multiple Myeloma Patients (NCT NCT00322491)

Last Updated: 2014-03-13

Results Overview

Number of participants with treatment emergent adverse events (TEAEs) collected from Day 1 (start of G-CSF mobilization) to the day before starting chemotherapy (approximately day 38). AEs were graded by the investigator using the World Health Organization (WHO) Adverse Event Grading Scale and were assessed for severity (mild, moderate, severe) and relatedness to study treatment (5 point scale from 'not related' to 'definitely related').

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

49 participants

Primary outcome timeframe

Day 1 to approximately Day 38 (before start of chemotherapy)

Results posted on

2014-03-13

Participant Flow

Participant milestones

Participant milestones
Measure	Non-Hodgkin's Lymphoma (NHL) Participants with NHL were mobilized with granulocyte colony-stimulating factor (G-CSF) 10 µg/kg/day for 4 days. Plerixafor 240 µg/kg was given the evening of day 4 and G-CSF given the next morning followed by apheresis. Evening doses of plerixafor and morning doses of G-CSF followed by apheresis continued for up to a maximum of 5 aphereses or until ≥ 5\*10\^6 CD34+ cells/kg were collected.	Multiple Myeloma (MM) Participants with MM were mobilized with granulocyte colony-stimulating factor (G-CSF) 10 µg/kg/day for 4 days. Plerixafor 240 µg/kg was given the evening of day 4 and G-CSF given the next morning followed by apheresis. Evening doses of plerixafor and morning doses of G-CSF followed by apheresis continued for up to a maximum of 5 aphereses or until ≥ 5\*10\^6 CD34+ cells/kg were collected.
Overall Study STARTED	23	26
Overall Study COMPLETED	21	24
Overall Study NOT COMPLETED	2	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Non-Hodgkin's Lymphoma (NHL) Participants with NHL were mobilized with granulocyte colony-stimulating factor (G-CSF) 10 µg/kg/day for 4 days. Plerixafor 240 µg/kg was given the evening of day 4 and G-CSF given the next morning followed by apheresis. Evening doses of plerixafor and morning doses of G-CSF followed by apheresis continued for up to a maximum of 5 aphereses or until ≥ 5\*10\^6 CD34+ cells/kg were collected.	Multiple Myeloma (MM) Participants with MM were mobilized with granulocyte colony-stimulating factor (G-CSF) 10 µg/kg/day for 4 days. Plerixafor 240 µg/kg was given the evening of day 4 and G-CSF given the next morning followed by apheresis. Evening doses of plerixafor and morning doses of G-CSF followed by apheresis continued for up to a maximum of 5 aphereses or until ≥ 5\*10\^6 CD34+ cells/kg were collected.
Overall Study Adverse Event	1	0
Overall Study Death	1	2

Baseline Characteristics

Mobilization of Stem Cells With AMD3100 (Plerixafor) and G-CSF in Non-Hodgkin's Lymphoma and Multiple Myeloma Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Non-Hodgkin's Lymphoma (NHL) n=23 Participants Participants with NHL were mobilized with granulocyte colony-stimulating factor (G-CSF) 10 µg/kg/day for 4 days. Plerixafor 240 µg/kg was given the evening of day 4 and G-CSF given the next morning followed by apheresis. Evening doses of plerixafor and morning doses of G-CSF followed by apheresis continued for up to a maximum of 5 aphereses or until ≥ 5\*10\^6 CD34+ cells/kg were collected.	Multiple Myeloma (MM) n=26 Participants Participants with MM were mobilized with granulocyte colony-stimulating factor (G-CSF) 10 µg/kg/day for 4 days. Plerixafor 240 µg/kg was given the evening of day 4 and G-CSF given the next morning followed by apheresis. Evening doses of plerixafor and morning doses of G-CSF followed by apheresis continued for up to a maximum of 5 aphereses or until ≥ 5\*10\^6 CD34+ cells/kg were collected.	Total n=49 Participants Total of all reporting groups
Age, Continuous	56.5 years STANDARD_DEVIATION 8.7 • n=5 Participants	57.6 years STANDARD_DEVIATION 8.1 • n=7 Participants	57.1 years STANDARD_DEVIATION 8.3 • n=5 Participants
Sex: Female, Male Female	9 Participants n=5 Participants	10 Participants n=7 Participants	19 Participants n=5 Participants
Sex: Female, Male Male	14 Participants n=5 Participants	16 Participants n=7 Participants	30 Participants n=5 Participants
Race/Ethnicity, Customized Caucasian	23 participants n=5 Participants	23 participants n=7 Participants	46 participants n=5 Participants
Race/Ethnicity, Customized African-American	0 participants n=5 Participants	1 participants n=7 Participants	1 participants n=5 Participants
Race/Ethnicity, Customized Hispanic/Latino	0 participants n=5 Participants	1 participants n=7 Participants	1 participants n=5 Participants
Race/Ethnicity, Customized Other	0 participants n=5 Participants	1 participants n=7 Participants	1 participants n=5 Participants

PRIMARY outcome

Timeframe: Day 1 to approximately Day 38 (before start of chemotherapy)

Population: Safety population - all participants who received at least 1 dose of plerixafor.

Outcome measures

Outcome measures
Measure	Non-Hodgkin's Lymphoma (NHL) n=23 Participants Participants with NHL were mobilized with granulocyte colony-stimulating factor (G-CSF) 10 µg/kg/day for 4 days. Plerixafor 240 µg/kg was given the evening of day 4 and G-CSF given the next morning followed by apheresis. Evening doses of plerixafor and morning doses of G-CSF followed by apheresis continued for up to a maximum of 5 aphereses or until ≥ 5\*10\^6 CD34+ cells/kg were collected.	Multiple Myeloma (MM) n=26 Participants Participants with MM were mobilized with granulocyte colony-stimulating factor (G-CSF) 10 µg/kg/day for 4 days. Plerixafor 240 µg/kg was given the evening of day 4 and G-CSF given the next morning followed by apheresis. Evening doses of plerixafor and morning doses of G-CSF followed by apheresis continued for up to a maximum of 5 aphereses or until ≥ 5\*10\^6 CD34+ cells/kg were collected.	All Participants n=49 Participants
Number of Participants in Overall Safety Summary of Treatment Emergent Adverse Events (TEAE) Participants reporting ≥ 1 Adverse Event	23 participants	26 participants	49 participants
Number of Participants in Overall Safety Summary of Treatment Emergent Adverse Events (TEAE) AE Severity (Mild)	8 participants	9 participants	17 participants
Number of Participants in Overall Safety Summary of Treatment Emergent Adverse Events (TEAE) AE Severity (Moderate)	5 participants	2 participants	7 participants
Number of Participants in Overall Safety Summary of Treatment Emergent Adverse Events (TEAE) AE Severity (Severe)	10 participants	15 participants	25 participants
Number of Participants in Overall Safety Summary of Treatment Emergent Adverse Events (TEAE) AE Relationship to Drug (Not Related)	0 participants	4 participants	4 participants
Number of Participants in Overall Safety Summary of Treatment Emergent Adverse Events (TEAE) AE Relationship to Drug (Probably Not Related)	1 participants	4 participants	5 participants
Number of Participants in Overall Safety Summary of Treatment Emergent Adverse Events (TEAE) AE Relationship to Drug (Possibly Related)	12 participants	5 participants	17 participants
Number of Participants in Overall Safety Summary of Treatment Emergent Adverse Events (TEAE) AE Relationship to Drug (Probably Related)	8 participants	11 participants	19 participants
Number of Participants in Overall Safety Summary of Treatment Emergent Adverse Events (TEAE) AE Relationship to Drug (Definitely Related)	2 participants	2 participants	4 participants

SECONDARY outcome

Timeframe: Days 4-5 (first dose of plerixafor to apheresis)

Population: The intent-to-treat population (defined as participants who received at least 1 dose of plerixafor).

The number of participants mobilized with G-CSF + plerixafor injection who have a ≥ 2-fold increase in CD34+ cells. Fold increase was expressed as a ratio. Fold increase = (pre-apheresis PB CD34+ cells/µL) / (pre-plerixafor dosing PB CD34+ cells/µL)

Outcome measures

Outcome measures
Measure	Non-Hodgkin's Lymphoma (NHL) n=23 Participants Participants with NHL were mobilized with granulocyte colony-stimulating factor (G-CSF) 10 µg/kg/day for 4 days. Plerixafor 240 µg/kg was given the evening of day 4 and G-CSF given the next morning followed by apheresis. Evening doses of plerixafor and morning doses of G-CSF followed by apheresis continued for up to a maximum of 5 aphereses or until ≥ 5\*10\^6 CD34+ cells/kg were collected.	Multiple Myeloma (MM) n=26 Participants Participants with MM were mobilized with granulocyte colony-stimulating factor (G-CSF) 10 µg/kg/day for 4 days. Plerixafor 240 µg/kg was given the evening of day 4 and G-CSF given the next morning followed by apheresis. Evening doses of plerixafor and morning doses of G-CSF followed by apheresis continued for up to a maximum of 5 aphereses or until ≥ 5\*10\^6 CD34+ cells/kg were collected.	All Participants n=49 Participants
Number of Participants Achieving a Two-Fold (Relative) Increase in Peripheral Blood (PB) CD34+ Cells/µL Following the First Dose of Plerixafor ≥ 2-fold Increase	20 participants	17 participants	37 participants
Number of Participants Achieving a Two-Fold (Relative) Increase in Peripheral Blood (PB) CD34+ Cells/µL Following the First Dose of Plerixafor < 2-fold Increase	3 participants	9 participants	12 participants

SECONDARY outcome

Timeframe: 2 months

Population: Participants who received a transplant. Two participants in the MM group received a second transplant.

Participants were monitored for polymorphonuclear leukocyte (PMN) engraftment as per the local standard of care. The target for engraftment was 12 days after PBSC transplant and no transplant taking longer than 21 days for engraftment.

Outcome measures

Outcome measures
Measure	Non-Hodgkin's Lymphoma (NHL) n=22 transplants Participants with NHL were mobilized with granulocyte colony-stimulating factor (G-CSF) 10 µg/kg/day for 4 days. Plerixafor 240 µg/kg was given the evening of day 4 and G-CSF given the next morning followed by apheresis. Evening doses of plerixafor and morning doses of G-CSF followed by apheresis continued for up to a maximum of 5 aphereses or until ≥ 5\*10\^6 CD34+ cells/kg were collected.	Multiple Myeloma (MM) n=27 transplants Participants with MM were mobilized with granulocyte colony-stimulating factor (G-CSF) 10 µg/kg/day for 4 days. Plerixafor 240 µg/kg was given the evening of day 4 and G-CSF given the next morning followed by apheresis. Evening doses of plerixafor and morning doses of G-CSF followed by apheresis continued for up to a maximum of 5 aphereses or until ≥ 5\*10\^6 CD34+ cells/kg were collected.	All Participants n=49 transplants
Number of Transplants in Which Participants Achieved Polymorphonuclear Leukocyte (PMN) Engraftment by Day 12 But No Later Than Day 21 Post Peripheral Blood Stem Cell (PBSC) Transplant ≤ Day 12	19 number of transplants	20 number of transplants	39 number of transplants
Number of Transplants in Which Participants Achieved Polymorphonuclear Leukocyte (PMN) Engraftment by Day 12 But No Later Than Day 21 Post Peripheral Blood Stem Cell (PBSC) Transplant Day 13 to Day 21	3 number of transplants	7 number of transplants	10 number of transplants
Number of Transplants in Which Participants Achieved Polymorphonuclear Leukocyte (PMN) Engraftment by Day 12 But No Later Than Day 21 Post Peripheral Blood Stem Cell (PBSC) Transplant ≥ Day 22	0 number of transplants	0 number of transplants	0 number of transplants

OTHER_PRE_SPECIFIED outcome

Timeframe: Days 5-8

Population: Participants who received at least one dose of plerixafor

Median cumulative total number of CD34+ cells collected during apheresis.

Outcome measures

Outcome measures
Measure	Non-Hodgkin's Lymphoma (NHL) n=23 Participants Participants with NHL were mobilized with granulocyte colony-stimulating factor (G-CSF) 10 µg/kg/day for 4 days. Plerixafor 240 µg/kg was given the evening of day 4 and G-CSF given the next morning followed by apheresis. Evening doses of plerixafor and morning doses of G-CSF followed by apheresis continued for up to a maximum of 5 aphereses or until ≥ 5\*10\^6 CD34+ cells/kg were collected.	Multiple Myeloma (MM) n=26 Participants Participants with MM were mobilized with granulocyte colony-stimulating factor (G-CSF) 10 µg/kg/day for 4 days. Plerixafor 240 µg/kg was given the evening of day 4 and G-CSF given the next morning followed by apheresis. Evening doses of plerixafor and morning doses of G-CSF followed by apheresis continued for up to a maximum of 5 aphereses or until ≥ 5\*10\^6 CD34+ cells/kg were collected.	All Participants n=49 Participants
Median Cumulative Number of CD34+ Cells Collected During Apheresis	5.2 CD34+ cells (*10^6 / kg) Interval 1.5 to 18.9	11.1 CD34+ cells (*10^6 / kg) Interval 4.4 to 22.5	5.9 CD34+ cells (*10^6 / kg) Interval 1.5 to 22.5

OTHER_PRE_SPECIFIED outcome

Timeframe: 2 months

Population: A total of 47 participants were transplanted. Two participants in the MM group received a second transplant using cells collected on study. One participant in the MM group did not have PLT engraftment information recorded, however did report a durable graft at month 12 post transplant.

Participants were monitored for platelet (PLT) engraftment as per the local standard of care. The target for engraftment was 12 days after PBSC transplant and no transplant taking longer than 21 days for engraftment.

Outcome measures

Outcome measures
Measure	Non-Hodgkin's Lymphoma (NHL) n=22 transplants Participants with NHL were mobilized with granulocyte colony-stimulating factor (G-CSF) 10 µg/kg/day for 4 days. Plerixafor 240 µg/kg was given the evening of day 4 and G-CSF given the next morning followed by apheresis. Evening doses of plerixafor and morning doses of G-CSF followed by apheresis continued for up to a maximum of 5 aphereses or until ≥ 5\*10\^6 CD34+ cells/kg were collected.	Multiple Myeloma (MM) n=26 transplants Participants with MM were mobilized with granulocyte colony-stimulating factor (G-CSF) 10 µg/kg/day for 4 days. Plerixafor 240 µg/kg was given the evening of day 4 and G-CSF given the next morning followed by apheresis. Evening doses of plerixafor and morning doses of G-CSF followed by apheresis continued for up to a maximum of 5 aphereses or until ≥ 5\*10\^6 CD34+ cells/kg were collected.	All Participants n=48 transplants
Number of Transplants in Which Participants Achieved Platelet (PLT) Engraftment by Day 12 But No Later Than Day 21 Post Peripheral Blood Stem Cell (PBSC) Transplant ≤ Day 12	7 number of transplants	7 number of transplants	14 number of transplants
Number of Transplants in Which Participants Achieved Platelet (PLT) Engraftment by Day 12 But No Later Than Day 21 Post Peripheral Blood Stem Cell (PBSC) Transplant Day 13 to Day 21	10 number of transplants	18 number of transplants	28 number of transplants
Number of Transplants in Which Participants Achieved Platelet (PLT) Engraftment by Day 12 But No Later Than Day 21 Post Peripheral Blood Stem Cell (PBSC) Transplant ≥ Day 22	5 number of transplants	1 number of transplants	6 number of transplants

OTHER_PRE_SPECIFIED outcome

Timeframe: Approximately 13 months (12 months post-transplant )

Population: Participants who received plerixafor, underwent transplantation, and were evaluable 12 months post transplant. The one participant who did not have a durable graft at 12 months had received chemotherapy for relapse approximately 9 months after transplantation.

The number of participants maintaining a durable graft 12 months after autologous transplantation. A durable graft is defined as the maintenance of normal blood counts.

Outcome measures

Outcome measures
Measure	Non-Hodgkin's Lymphoma (NHL) n=21 Participants Participants with NHL were mobilized with granulocyte colony-stimulating factor (G-CSF) 10 µg/kg/day for 4 days. Plerixafor 240 µg/kg was given the evening of day 4 and G-CSF given the next morning followed by apheresis. Evening doses of plerixafor and morning doses of G-CSF followed by apheresis continued for up to a maximum of 5 aphereses or until ≥ 5\*10\^6 CD34+ cells/kg were collected.	Multiple Myeloma (MM) n=23 Participants Participants with MM were mobilized with granulocyte colony-stimulating factor (G-CSF) 10 µg/kg/day for 4 days. Plerixafor 240 µg/kg was given the evening of day 4 and G-CSF given the next morning followed by apheresis. Evening doses of plerixafor and morning doses of G-CSF followed by apheresis continued for up to a maximum of 5 aphereses or until ≥ 5\*10\^6 CD34+ cells/kg were collected.	All Participants n=44 Participants
Number of Participants With Durable Engraftment 12 Months After Transplantation	21 participants	22 participants	43 participants

Adverse Events

Non-Hodgkin's Lymphoma (NHL)

Serious events: 0 serious events

Other events: 23 other events

Deaths: 0 deaths

Multiple Myeloma (MM)

Serious events: 1 serious events

Other events: 26 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Non-Hodgkin's Lymphoma (NHL) n=23 participants at risk Participants with NHL were mobilized with granulocyte colony-stimulating factor (G-CSF) 10 µg/kg/day for 4 days. Plerixafor 240 µg/kg was given the evening of day 4 and G-CSF given the next morning followed by apheresis. Evening doses of plerixafor and morning doses of G-CSF followed by apheresis continued for up to a maximum of 5 aphereses or until ≥ 5\*10\^6 CD34+ cells/kg were collected.	Multiple Myeloma (MM) n=26 participants at risk Participants with MM were mobilized with granulocyte colony-stimulating factor (G-CSF) 10 µg/kg/day for 4 days. Plerixafor 240 µg/kg was given the evening of day 4 and G-CSF given the next morning followed by apheresis. Evening doses of plerixafor and morning doses of G-CSF followed by apheresis continued for up to a maximum of 5 aphereses or until ≥ 5\*10\^6 CD34+ cells/kg were collected.
General disorders Heparin-induced thrombocytopenia	0.00% 0/23 • First day of G-CSF administration to the day prior to chemotherapy/ablative treatment in preparation of first transplant. This period includes the G-CSF, plerixafor treatment and rest period. In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables. Each AE table includes events, regardless of reported relationship to study treatment or grade.	3.8% 1/26 • First day of G-CSF administration to the day prior to chemotherapy/ablative treatment in preparation of first transplant. This period includes the G-CSF, plerixafor treatment and rest period. In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables. Each AE table includes events, regardless of reported relationship to study treatment or grade.

Other adverse events

Additional Information

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