Trial Outcomes & Findings for Treatment With AMD3100 (Plerixafor) in Non-Hodgkin's Lymphoma and Multiple Myeloma Patients (NCT NCT00322842)

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 step scale from 'not related' to 'definitely related').

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

35 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	4	31
Overall Study COMPLETED	2	29
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 Withdrawal by Subject	0	1
Overall Study Lost to Follow-up	0	1
Overall Study Refused transplant	1	0
Overall Study Death	1	0

Baseline Characteristics

Treatment With AMD3100 (Plerixafor) in Non-Hodgkin's Lymphoma and Multiple Myeloma Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Non-Hodgkin's Lymphoma (NHL) n=4 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=31 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=35 Participants Total of all reporting groups
Age, Continuous	59.8 years STANDARD_DEVIATION 8.1 • n=5 Participants	58.8 years STANDARD_DEVIATION 7.3 • n=7 Participants	58.9 years STANDARD_DEVIATION 7.3 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants	12 Participants n=7 Participants	15 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants	19 Participants n=7 Participants	20 Participants n=5 Participants
Race/Ethnicity, Customized Caucasian	4 participants n=5 Participants	30 participants n=7 Participants	34 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=4 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=31 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=35 Participants
Number of Participants in Overall Safety Summary of Treatment Emergent Adverse Events (TEAE) Participants reporting ≥ 1 AE	4 participants	20 participants	24 participants
Number of Participants in Overall Safety Summary of Treatment Emergent Adverse Events (TEAE) AE Severity (Moderate)	2 participants	4 participants	6 participants
Number of Participants in Overall Safety Summary of Treatment Emergent Adverse Events (TEAE) AE Relationship to Drug (Probably Not Related)	0 participants	0 participants	0 participants
Number of Participants in Overall Safety Summary of Treatment Emergent Adverse Events (TEAE) AE Relationship to Drug (Probably Related)	0 participants	0 participants	0 participants
Number of Participants in Overall Safety Summary of Treatment Emergent Adverse Events (TEAE) AE Severity (Mild)	2 participants	16 participants	18 participants
Number of Participants in Overall Safety Summary of Treatment Emergent Adverse Events (TEAE) AE Severity (Severe)	0 participants	0 participants	0 participants
Number of Participants in Overall Safety Summary of Treatment Emergent Adverse Events (TEAE) AE Relationship to Drug (Not Related)	4 participants	18 participants	22 participants
Number of Participants in Overall Safety Summary of Treatment Emergent Adverse Events (TEAE) AE Relationship to Drug (Possibly Related)	0 participants	2 participants	2 participants
Number of Participants in Overall Safety Summary of Treatment Emergent Adverse Events (TEAE) AE Relationship to Drug (Definitely Related)	0 participants	0 participants	0 participants

SECONDARY outcome

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

Population: Intent to treat population

The fold increase was measured using local lab values and is the ratio of post first dose (pre-apheresis) PB CD34+ cells/µL)/pre-plerixafor dosing PB CD34+ cells/µL)

Outcome measures

Outcome measures
Measure	Non-Hodgkin's Lymphoma (NHL) n=4 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=31 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=35 Participants
Fold (i.e., Relative) Increase in Peripheral Blood (PB) CD34+ Cells/µL After First Dose of Plerixafor	2.6 ratio Full Range 1.2 • Interval 2.0 to 4.7	2.6 ratio Full Range 1.4 • Interval 1.1 to 6.9	2.6 ratio Full Range 1.3 • Interval 1.1 to 6.9

SECONDARY outcome

Timeframe: 2 months

Population: Participants who had transplants. Fifteen participants had tandem transplants. The date of initial PMN engraftment was missing for 11 transplants, which were later shown to have durable grafts.

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=3 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=45 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 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	2 number of transplants	25 number of transplants	27 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	1 number of transplants	1 number of transplants	2 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 12	0 number of transplants	8 number of transplants	8 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 Unknown	0 number of transplants	11 number of transplants	11 number of transplants

SECONDARY outcome

Timeframe: up to day 8

Population: Analysis was not performed. Historical data was not available.

A comparison of the effectiveness in mobilizing peripheral blood CD34+ cells between this study's treatment regimen (G-CSF plus plerixafor) to other treatment options: G-CSF alone, and chemotherapy with G-CSF.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Days 5-8

Population: Intent to treat population. Samples from two participants were not analyzed by the central lab.

Median total number of CD34+ cells collected during apheresis as measured by a central lab.

Outcome measures

Outcome measures
Measure	Non-Hodgkin's Lymphoma (NHL) n=4 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=29 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=35 Participants
Median Cumulative Number of CD34+ Cells Collected During Apheresis	8.3 CD34+ cells (*10^6 / kg) Interval 4.9 to 17.6	7.1 CD34+ cells (*10^6 / kg) Interval 0.1 to 35.7	7.4 CD34+ cells (*10^6 / kg) Interval 0.1 to 35.7

OTHER_PRE_SPECIFIED outcome

Timeframe: 2 months

Population: Participants who had transplants. Fifteen participants had tandem transplants.

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=3 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=45 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 13 to Day 21	2 number of transplants	13 number of transplants	15 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	1 number of transplants	0 number of transplants	1 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 12	0 number of transplants	32 number of transplants	32 number of transplants

OTHER_PRE_SPECIFIED outcome

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

Population: Participants who had transplants and engraftment data 12 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=2 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=29 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=31 Participants
Number of Participants With Durable Engraftment 12 Months After Transplantation	2 participants	29 participants	31 participants

Adverse Events

Non-Hodgkin's Lymphoma (NHL)

Serious events: 2 serious events

Other events: 4 other events

Deaths: 0 deaths

Multiple Myeloma (MM)

Serious events: 0 serious events

Other events: 20 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Non-Hodgkin's Lymphoma (NHL) n=4 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=31 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 Pyrexia	25.0% 1/4 • 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.	0.00% 0/31 • 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.
Metabolism and nutrition disorders Diabetes mellitus non-insulin-dependent	25.0% 1/4 • 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.	0.00% 0/31 • 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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