Trial Outcomes & Findings for Evaluation of Approved Weight-Based Dose Compared to Fixed Dose of Plerixafor in Patients With Non-Hodgkin's Lymphoma (NHL) Weighing Less Than 70 Kilograms (NCT NCT01164475)

Last Updated: 2014-02-25

Results Overview

The cumulative number of CD34+ cells/kg (body weight) collected over all apheresis sessions (up to a maximum of 4 sessions) was used to determine if a patient has achieved the target of \>=5\*10\^6 CD34+ cells/kg (optimum number of CD34+ cells required for transplantation) within 4 days of apheresis. The proportion of patients who achieved the target was reported as percentages for each treatment arm.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

61 participants

Primary outcome timeframe

Day 5 up to Day 8

Results posted on

2014-02-25

Participant Flow

The study was conducted at 7 centers in 4 countries between October 13, 2010 and February 26, 2013.

A total of 71 patients were screened of which 10 patients were screen failures. A total of 61 patients were randomized.

Participant milestones

Participant milestones
Measure	Fixed Dose Plerixafor 10 microgram per kilogram (mcg/kg) granulocyte-colony stimulating factor (G-CSF) subcutaneous (SC) injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by 20 milligram (mg) plerixafor SC injection (fixed dose) in evening of Day 4 (10 to 11 hours prior to first apheresis), and then 10 mcg/kg G-CSF SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of cluster of differentiation 34 (CD34+) stem cells (greater than or equal to \[\>=\] 5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.	Weight-Based Plerixafor G-CSF 10 mcg/kg SC injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by plerixafor 0.24 milligram per kilogram (mg/kg) SC injection (weight-based dose) in evening of Day 4 (10 to 11 hours before first apheresis), and then G-CSF 10 mcg/kg SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of CD34+ stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.
Overall Study STARTED	30	31
Overall Study COMPLETED	28	29
Overall Study NOT COMPLETED	2	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Fixed Dose Plerixafor 10 microgram per kilogram (mcg/kg) granulocyte-colony stimulating factor (G-CSF) subcutaneous (SC) injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by 20 milligram (mg) plerixafor SC injection (fixed dose) in evening of Day 4 (10 to 11 hours prior to first apheresis), and then 10 mcg/kg G-CSF SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of cluster of differentiation 34 (CD34+) stem cells (greater than or equal to \[\>=\] 5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.	Weight-Based Plerixafor G-CSF 10 mcg/kg SC injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by plerixafor 0.24 milligram per kilogram (mg/kg) SC injection (weight-based dose) in evening of Day 4 (10 to 11 hours before first apheresis), and then G-CSF 10 mcg/kg SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of CD34+ stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.
Overall Study Lack of Efficacy	1	0
Overall Study Other	0	1
Overall Study Progressive disease	0	1
Overall Study Protocol Violation	1	0

Baseline Characteristics

Evaluation of Approved Weight-Based Dose Compared to Fixed Dose of Plerixafor in Patients With Non-Hodgkin's Lymphoma (NHL) Weighing Less Than 70 Kilograms

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Fixed Dose Plerixafor n=30 Participants 10 microgram per kilogram (mcg/kg) granulocyte-colony stimulating factor (G-CSF) subcutaneous (SC) injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by 20 milligram (mg) plerixafor SC injection (fixed dose) in evening of Day 4 (10 to 11 hours prior to first apheresis), and then 10 mcg/kg G-CSF SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of cluster of differentiation 34 (CD34+) stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.	Weight-Based Plerixafor n=31 Participants G-CSF 10 mcg/kg SC injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by plerixafor 0.24 milligram per kilogram (mg/kg) SC injection (weight-based dose) in evening of Day 4 (10 to 11 hours before first apheresis), and then G-CSF 10 mcg/kg SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of CD34+ stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.	Total n=61 Participants Total of all reporting groups
Age, Continuous	46.13 years STANDARD_DEVIATION 13.40 • n=5 Participants	47.84 years STANDARD_DEVIATION 13.59 • n=7 Participants	47.00 years STANDARD_DEVIATION 13.41 • n=5 Participants
Sex: Female, Male Female	18 Participants n=5 Participants	17 Participants n=7 Participants	35 Participants n=5 Participants
Sex: Female, Male Male	12 Participants n=5 Participants	14 Participants n=7 Participants	26 Participants n=5 Participants
Race/Ethnicity, Customized Race: Asian	28 participants n=5 Participants	28 participants n=7 Participants	56 participants n=5 Participants
Race/Ethnicity, Customized Race: White	2 participants n=5 Participants	3 participants n=7 Participants	5 participants n=5 Participants
Race/Ethnicity, Customized Ethnicity: Hispanic or Latino	1 participants n=5 Participants	2 participants n=7 Participants	3 participants n=5 Participants
Race/Ethnicity, Customized Ethnicity: Not Hispanic or Latino	29 participants n=5 Participants	29 participants n=7 Participants	58 participants n=5 Participants
Number of Patients With Peripheral-Blood (PB) Cluster of Differentiation 34+ (CD34+) Cell Count Less than 10 cells per microliter (mcL)	20 participants n=5 Participants	21 participants n=7 Participants	41 participants n=5 Participants
Number of Patients With Peripheral-Blood (PB) Cluster of Differentiation 34+ (CD34+) Cell Count Greater than or equal to 10 cells/mcL	10 participants n=5 Participants	10 participants n=7 Participants	20 participants n=5 Participants

PRIMARY outcome

Timeframe: Day 5 up to Day 8

Population: FAS included all randomized patients who signed informed consent form and received at least one dose of plerixafor.

Outcome measures

Outcome measures
Measure	Fixed Dose Plerixafor n=30 Participants 10 microgram per kilogram (mcg/kg) granulocyte-colony stimulating factor (G-CSF) subcutaneous (SC) injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by 20 milligram (mg) plerixafor SC injection (fixed dose) in evening of Day 4 (10 to 11 hours prior to first apheresis), and then 10 mcg/kg G-CSF SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of cluster of differentiation 34 (CD34+) stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.	Weight-Based Plerixafor n=31 Participants G-CSF 10 mcg/kg SC injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by plerixafor 0.24 milligram per kilogram (mg/kg) SC injection (weight-based dose) in evening of Day 4 (10 to 11 hours before first apheresis), and then G-CSF 10 mcg/kg SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of CD34+ stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.
Proportion of Patients Who Achieved at Least 5*10^6 Cluster of Differentiation 34+ (CD34+) Cells Per Kilogram (Cells/kg)	60.0 percentage of participants	54.8 percentage of participants

PRIMARY outcome

Timeframe: 0 (pre-plerixafor dose), 0.5, 1, 4 hours post-plerixafor dose on Day 4; 9-10 hours post-plerixafor dose (pre G-CSF dose) on Day 5, 10-11 hours post-plerixafor dose (prior to first apheresis) on Day 5

Population: FAS included all randomized patients who signed informed consent form and received at least one dose of plerixafor.

Outcome measures

Outcome measures
Measure	Fixed Dose Plerixafor n=30 Participants 10 microgram per kilogram (mcg/kg) granulocyte-colony stimulating factor (G-CSF) subcutaneous (SC) injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by 20 milligram (mg) plerixafor SC injection (fixed dose) in evening of Day 4 (10 to 11 hours prior to first apheresis), and then 10 mcg/kg G-CSF SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of cluster of differentiation 34 (CD34+) stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.	Weight-Based Plerixafor n=31 Participants G-CSF 10 mcg/kg SC injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by plerixafor 0.24 milligram per kilogram (mg/kg) SC injection (weight-based dose) in evening of Day 4 (10 to 11 hours before first apheresis), and then G-CSF 10 mcg/kg SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of CD34+ stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.
Area Under the Concentration-time Curve From Time 0 to 10 Hours (AUC [0-10])	3991.2 nanogramhour per milliliter (nghr/mL) Standard Error 1.03	2792.7 nanogramhour per milliliter (nghr/mL) Standard Error 1.03

SECONDARY outcome

Timeframe: Day 5 up to Day 8

Population: FAS included all randomized patients who signed informed consent form and received at least one dose of plerixafor.

The cumulative number of CD34+ cells/kg (body weight) collected over all apheresis sessions (up to a maximum of 4 sessions) was used to determine if a patient has achieved the target of \>= 2\*10\^6 CD34+ cells/kg (minimum number of CD34+ cells required for transplantation) within 4 days of apheresis. The proportion of patients who achieved the target was reported as percentages for each treatment arm.

Outcome measures

Outcome measures
Measure	Fixed Dose Plerixafor n=30 Participants 10 microgram per kilogram (mcg/kg) granulocyte-colony stimulating factor (G-CSF) subcutaneous (SC) injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by 20 milligram (mg) plerixafor SC injection (fixed dose) in evening of Day 4 (10 to 11 hours prior to first apheresis), and then 10 mcg/kg G-CSF SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of cluster of differentiation 34 (CD34+) stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.	Weight-Based Plerixafor n=31 Participants G-CSF 10 mcg/kg SC injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by plerixafor 0.24 milligram per kilogram (mg/kg) SC injection (weight-based dose) in evening of Day 4 (10 to 11 hours before first apheresis), and then G-CSF 10 mcg/kg SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of CD34+ stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.
Proportion of Patients Who Achieved at Least 2*10^6 CD34+ Cells/kg in Less Than or Equal to 4 Days of Apheresis	93.3 percentage of participants	90.3 percentage of participants

SECONDARY outcome

Timeframe: Day 5 up to Day 8

Population: FAS included all randomized patients who signed informed consent form and received at least one dose of plerixafor. Here, number of patients analyzed = the number of patients who were evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Fixed Dose Plerixafor n=28 Participants 10 microgram per kilogram (mcg/kg) granulocyte-colony stimulating factor (G-CSF) subcutaneous (SC) injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by 20 milligram (mg) plerixafor SC injection (fixed dose) in evening of Day 4 (10 to 11 hours prior to first apheresis), and then 10 mcg/kg G-CSF SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of cluster of differentiation 34 (CD34+) stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.	Weight-Based Plerixafor n=28 Participants G-CSF 10 mcg/kg SC injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by plerixafor 0.24 milligram per kilogram (mg/kg) SC injection (weight-based dose) in evening of Day 4 (10 to 11 hours before first apheresis), and then G-CSF 10 mcg/kg SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of CD34+ stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.
Median Number of Days of Apheresis to Collect at Least 2*10^6 CD34+ Cells/kg	1 days Interval 1.0 to 4.0	2 days Interval 1.0 to 4.0

SECONDARY outcome

Timeframe: Day 5 up to Day 8

Outcome measures

Outcome measures
Measure	Fixed Dose Plerixafor n=18 Participants 10 microgram per kilogram (mcg/kg) granulocyte-colony stimulating factor (G-CSF) subcutaneous (SC) injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by 20 milligram (mg) plerixafor SC injection (fixed dose) in evening of Day 4 (10 to 11 hours prior to first apheresis), and then 10 mcg/kg G-CSF SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of cluster of differentiation 34 (CD34+) stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.	Weight-Based Plerixafor n=17 Participants G-CSF 10 mcg/kg SC injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by plerixafor 0.24 milligram per kilogram (mg/kg) SC injection (weight-based dose) in evening of Day 4 (10 to 11 hours before first apheresis), and then G-CSF 10 mcg/kg SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of CD34+ stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.
Median Number of Days of Apheresis to Collect at Least 5*10^6 CD34+ Cells/kg	3 days Interval 1.0 to 4.0	3 days Interval 1.0 to 4.0

SECONDARY outcome

Timeframe: Day 5 up to Day 8

Population: FAS included all randomized patients who signed informed consent form and received at least one dose of plerixafor.

The cumulative number of CD34+ cells/kg (body weight) collected over all apheresis sessions (up to a maximum of 4 sessions) was reported.

Outcome measures

Outcome measures
Measure	Fixed Dose Plerixafor n=30 Participants 10 microgram per kilogram (mcg/kg) granulocyte-colony stimulating factor (G-CSF) subcutaneous (SC) injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by 20 milligram (mg) plerixafor SC injection (fixed dose) in evening of Day 4 (10 to 11 hours prior to first apheresis), and then 10 mcg/kg G-CSF SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of cluster of differentiation 34 (CD34+) stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.	Weight-Based Plerixafor n=31 Participants G-CSF 10 mcg/kg SC injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by plerixafor 0.24 milligram per kilogram (mg/kg) SC injection (weight-based dose) in evening of Day 4 (10 to 11 hours before first apheresis), and then G-CSF 10 mcg/kg SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of CD34+ stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.
Total Number of CD34+ Cells/kg Collected Over up to 4 Aphereses	5.35 10^6 cells/kg Interval 1.2 to 9.6	5.24 10^6 cells/kg Interval 1.3 to 372.8

SECONDARY outcome

Timeframe: Baseline (pre G-CSF dose on Day 4) to Day 5 (prior to first apheresis)

Population: FAS included all randomized patients who signed informed consent form and received at least one dose of plerixafor.

Fold increase was calculated as CD34+ cell count on Day 5 divided by CD34+ cell count on Day 4.

Outcome measures

Outcome measures
Measure	Fixed Dose Plerixafor n=30 Participants 10 microgram per kilogram (mcg/kg) granulocyte-colony stimulating factor (G-CSF) subcutaneous (SC) injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by 20 milligram (mg) plerixafor SC injection (fixed dose) in evening of Day 4 (10 to 11 hours prior to first apheresis), and then 10 mcg/kg G-CSF SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of cluster of differentiation 34 (CD34+) stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.	Weight-Based Plerixafor n=31 Participants G-CSF 10 mcg/kg SC injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by plerixafor 0.24 milligram per kilogram (mg/kg) SC injection (weight-based dose) in evening of Day 4 (10 to 11 hours before first apheresis), and then G-CSF 10 mcg/kg SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of CD34+ stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.
Mean Fold Increase in Peripheral Blood CD34+ Cell Count Following Plerixafor	5.43 fold increase Standard Deviation 4.02	5.09 fold increase Standard Deviation 2.81

SECONDARY outcome

Population: FAS included all randomized patients who signed informed consent form and received at least one dose of plerixafor.

Outcome measures

Outcome measures
Measure	Fixed Dose Plerixafor n=30 Participants 10 microgram per kilogram (mcg/kg) granulocyte-colony stimulating factor (G-CSF) subcutaneous (SC) injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by 20 milligram (mg) plerixafor SC injection (fixed dose) in evening of Day 4 (10 to 11 hours prior to first apheresis), and then 10 mcg/kg G-CSF SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of cluster of differentiation 34 (CD34+) stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.	Weight-Based Plerixafor n=31 Participants G-CSF 10 mcg/kg SC injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by plerixafor 0.24 milligram per kilogram (mg/kg) SC injection (weight-based dose) in evening of Day 4 (10 to 11 hours before first apheresis), and then G-CSF 10 mcg/kg SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of CD34+ stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.
Maximum Observed Plasma Concentration (Cmax)	957 ng/mL Standard Deviation 216	711 ng/mL Standard Deviation 136

SECONDARY outcome

Population: FAS included all patients who have signed informed consent and received at least one dose of study drug.

Outcome measures

Outcome measures
Measure	Fixed Dose Plerixafor n=30 Participants 10 microgram per kilogram (mcg/kg) granulocyte-colony stimulating factor (G-CSF) subcutaneous (SC) injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by 20 milligram (mg) plerixafor SC injection (fixed dose) in evening of Day 4 (10 to 11 hours prior to first apheresis), and then 10 mcg/kg G-CSF SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of cluster of differentiation 34 (CD34+) stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.	Weight-Based Plerixafor n=31 Participants G-CSF 10 mcg/kg SC injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by plerixafor 0.24 milligram per kilogram (mg/kg) SC injection (weight-based dose) in evening of Day 4 (10 to 11 hours before first apheresis), and then G-CSF 10 mcg/kg SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of CD34+ stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.
Time to Reach Maximum Plasma Concentration (Tmax)	0.50 hours Interval 0.42 to 1.08	0.50 hours Interval 0.42 to 1.02

SECONDARY outcome

Population: FAS included all randomized patients who signed informed consent form and received at least one dose of plerixafor.

T1/2 is the time required for the plasma concentration to decrease to one half.

Outcome measures

Outcome measures
Measure	Fixed Dose Plerixafor n=30 Participants 10 microgram per kilogram (mcg/kg) granulocyte-colony stimulating factor (G-CSF) subcutaneous (SC) injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by 20 milligram (mg) plerixafor SC injection (fixed dose) in evening of Day 4 (10 to 11 hours prior to first apheresis), and then 10 mcg/kg G-CSF SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of cluster of differentiation 34 (CD34+) stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.	Weight-Based Plerixafor n=31 Participants G-CSF 10 mcg/kg SC injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by plerixafor 0.24 milligram per kilogram (mg/kg) SC injection (weight-based dose) in evening of Day 4 (10 to 11 hours before first apheresis), and then G-CSF 10 mcg/kg SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of CD34+ stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.
Terminal Elimination Half-life (T1/2)	4.53 hours Standard Deviation 1.38	4.27 hours Standard Deviation 0.93

Adverse Events

Fixed Dose Plerixafor

Serious events: 0 serious events

Other events: 26 other events

Deaths: 0 deaths

Weight-Based Plerixafor

Serious events: 3 serious events

Other events: 25 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Fixed Dose Plerixafor n=30 participants at risk 10 microgram per kilogram (mcg/kg) granulocyte-colony stimulating factor (G-CSF) subcutaneous (SC) injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by 20 milligram (mg) plerixafor SC injection (fixed dose) in evening of Day 4 (10 to 11 hours prior to first apheresis), and then 10 mcg/kg G-CSF SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of cluster of differentiation 34 (CD34+) stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.	Weight-Based Plerixafor n=31 participants at risk G-CSF 10 mcg/kg SC injection once daily in morning from Day 1 through Day 4 (G-CSF mobilization period), followed by plerixafor 0.24 milligram per kilogram (mg/kg) SC injection (weight-based dose) in evening of Day 4 (10 to 11 hours before first apheresis), and then G-CSF 10 mcg/kg SC injection in morning of Day 5 (1 hour prior to first apheresis). Apheresis process and treatment with plerixafor (10 to 11 hours prior to apheresis) and G-CSF (1 hour prior to apheresis) was continued until the target number of CD34+ stem cells (\>=5\*10\^6 cells/kg) was collected or until a maximum 4 apheresis sessions occurred.
Infections and infestations Cellulitis	0.00% 0/30 • From signing of inform consent form up to 30 days after the last dose of plerixafor Median number of plerixafor doses was 3 doses for both treatment arms. The analysis was performed on safety population, defined as all patients who signed informed consent form and received at least one dose of plerixafor.	3.2% 1/31 • From signing of inform consent form up to 30 days after the last dose of plerixafor Median number of plerixafor doses was 3 doses for both treatment arms. The analysis was performed on safety population, defined as all patients who signed informed consent form and received at least one dose of plerixafor.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Diffuse large B-cell lymphoma	0.00% 0/30 • From signing of inform consent form up to 30 days after the last dose of plerixafor Median number of plerixafor doses was 3 doses for both treatment arms. The analysis was performed on safety population, defined as all patients who signed informed consent form and received at least one dose of plerixafor.	3.2% 1/31 • From signing of inform consent form up to 30 days after the last dose of plerixafor Median number of plerixafor doses was 3 doses for both treatment arms. The analysis was performed on safety population, defined as all patients who signed informed consent form and received at least one dose of plerixafor.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Non-Hodgkin's lymphoma	0.00% 0/30 • From signing of inform consent form up to 30 days after the last dose of plerixafor Median number of plerixafor doses was 3 doses for both treatment arms. The analysis was performed on safety population, defined as all patients who signed informed consent form and received at least one dose of plerixafor.	3.2% 1/31 • From signing of inform consent form up to 30 days after the last dose of plerixafor Median number of plerixafor doses was 3 doses for both treatment arms. The analysis was performed on safety population, defined as all patients who signed informed consent form and received at least one dose of plerixafor.
Respiratory, thoracic and mediastinal disorders Pleural effusion	0.00% 0/30 • From signing of inform consent form up to 30 days after the last dose of plerixafor Median number of plerixafor doses was 3 doses for both treatment arms. The analysis was performed on safety population, defined as all patients who signed informed consent form and received at least one dose of plerixafor.	3.2% 1/31 • From signing of inform consent form up to 30 days after the last dose of plerixafor Median number of plerixafor doses was 3 doses for both treatment arms. The analysis was performed on safety population, defined as all patients who signed informed consent form and received at least one dose of plerixafor.

Other adverse events

Additional Information

Trial Transparency Team

Sanofi

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee If no publication has occurred within 12 months of the completion of the study, the Investigator shall have the right to publish/present independently the results of the study. The Investigator shall provide the Sponsor with a copy of any such presentation/publication for comment at least 30 days before any presentation/submission for publication. If requested by the Sponsor, any presentation/submission shall be delayed up to 90 days, to allow the Sponsor to preserve its proprietary rights.
Publication restrictions are in place

Restriction type: OTHER