Trial Outcomes & Findings for AMD3100 (Plerixafor) in Multiple Myeloma (MM) or Non-Hodgkin's Lymphoma (NHL) Patients Predicted to be Unable to Mobilize With G-CSF Alone (NCT NCT00395967)

Last Updated: 2015-05-01

Results Overview

The number of patients with a circulating CD34+ count \>= 5 and \< 20 cells/ml after 5 days of mobilization with G-CSF alone who achieved cumulative apheresis yields of ≥2\*10\^6 CD34+ cells/kg within 3 days of apheresis after receiving G-CSF plus plerixafor. Outcome was based on laboratory results from a central lab.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

5 participants

Primary outcome timeframe

approximately days 6-9

Results posted on

2015-05-01

Participant Flow

Study enrollment began in April 2005 and the study was terminated in August 2006. Target enrollment was 15 patients, however the trial was terminated early after five patients were enrolled.

Participant milestones

Participant milestones
Measure	All Patients Mobilization with Granulocyte Colony Stimulating Factor (G-CSF) (10 µg/kg once a day) for 5 days. Patients received once daily plerixafor treatment (240 mg/kg) in the evening (10 to 11 hours prior to apheresis) for up to 3 days if peripheral blood CD34+ cell counts on Day 5 met the entry criteria. Morning doses of G-CSF (10 µg/kg) continued throughout apheresis.
Treatment Period STARTED	5
Treatment Period COMPLETED	4
Treatment Period NOT COMPLETED	1
Follow-up Period STARTED	5
Follow-up Period COMPLETED	3
Follow-up Period NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	All Patients Mobilization with Granulocyte Colony Stimulating Factor (G-CSF) (10 µg/kg once a day) for 5 days. Patients received once daily plerixafor treatment (240 mg/kg) in the evening (10 to 11 hours prior to apheresis) for up to 3 days if peripheral blood CD34+ cell counts on Day 5 met the entry criteria. Morning doses of G-CSF (10 µg/kg) continued throughout apheresis.
Treatment Period Failed Mobilization	1
Follow-up Period Death	1
Follow-up Period Lost to Follow-up	1

Baseline Characteristics

AMD3100 (Plerixafor) in Multiple Myeloma (MM) or Non-Hodgkin's Lymphoma (NHL) Patients Predicted to be Unable to Mobilize With G-CSF Alone

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Patients n=5 Participants Mobilization with Granulocyte Colony Stimulating Factor (G-CSF) (10 µg/kg once a day) for 5 days. Patients received once daily plerixafor treatment (240 mg/kg) in the evening (10 to 11 hours prior to apheresis) for up to 3 days if peripheral blood CD34+ cell counts on Day 5 met the entry criteria. Morning doses of G-CSF (10 µg/kg) continued throughout apheresis.
Age, Continuous	52.2 years STANDARD_DEVIATION 11.5 • n=93 Participants
Sex: Female, Male Female	2 Participants n=93 Participants
Sex: Female, Male Male	3 Participants n=93 Participants
Race/Ethnicity, Customized Caucasian	5 participants n=93 Participants
Disease Diagnosis Multiple Myeloma (MM)	1 participants n=93 Participants
Disease Diagnosis Non-Hodgkin's lymphoma (NHL)	3 participants n=93 Participants
Disease Diagnosis Hodgkin's disease (HD)	1 participants n=93 Participants

PRIMARY outcome

Timeframe: approximately days 6-9

Population: All patients who received plerixafor.

Outcome measures

Outcome measures
Measure	All Patients n=5 Participants Mobilization with Granulocyte Colony Stimulating Factor (G-CSF) (10 µg/kg once a day) for 5 days. Patients received once daily plerixafor treatment (240 mg/kg) in the evening (10 to 11 hours prior to apheresis) for up to 3 days if peripheral blood CD34+ cell counts on Day 5 met the entry criteria. Morning doses of G-CSF (10 µg/kg) continued throughout apheresis.
Number of Patients Who Achieved ≥210^6 CD34+ Cells/kg Following Treatment With Plerixafor 240 µg/kg and G-CSF for up to 3 Consecutive Days Participants with ≥210^6 CD34+ cells/kg	3 participants
Number of Patients Who Achieved ≥210^6 CD34+ Cells/kg Following Treatment With Plerixafor 240 µg/kg and G-CSF for up to 3 Consecutive Days Participants with <210^6 CD34+ cells/kg	2 participants

SECONDARY outcome

Timeframe: up to 13 months

Population: Safety Population defined as all participants who received G-CSF and/or plerixafor.

Numbers of participants with adverse events (AEs) collected from Day 1 (start of G-CSF Mobilization) to 12 months after transplantation. AEs were reported regardless of relationship to study treatment. The investigator graded each AE using the World Health Organization (WHO) Adverse Event Grading Scale and provided assessments of seriousness and relatedness to study treatment.

Outcome measures

Outcome measures
Measure	All Patients n=5 Participants Mobilization with Granulocyte Colony Stimulating Factor (G-CSF) (10 µg/kg once a day) for 5 days. Patients received once daily plerixafor treatment (240 mg/kg) in the evening (10 to 11 hours prior to apheresis) for up to 3 days if peripheral blood CD34+ cell counts on Day 5 met the entry criteria. Morning doses of G-CSF (10 µg/kg) continued throughout apheresis.
Overall Participants Counts of Adverse Events Participants Reporting At Least One AE	5 participants
Overall Participants Counts of Adverse Events Participants Reporting a Severe AE	4 participants
Overall Participants Counts of Adverse Events Participants Reporting a Life-threatening AE	2 participants
Overall Participants Counts of Adverse Events AE Relation to Drug: Not Related or Probably Not	3 participants
Overall Participants Counts of Adverse Events AE Relation to Drug: Possibly Related	2 participants
Overall Participants Counts of Adverse Events AE Relation to Drug: Probably or Definitely Relate	0 participants
Overall Participants Counts of Adverse Events Serious Adverse Events	4 participants
Overall Participants Counts of Adverse Events AEs Leading to Discontinuation	0 participants

SECONDARY outcome

Timeframe: Days 5-6

Population: This study was terminated early and analysis was not done.

The fold increase was measured by fluorescence activated cell sorting (FACS) analysis and expressed as a ratio. Fold increase = pre-apheresis PB CD34+ cells/µL)/(pre-plerixafor dosing PB CD34+ cells/µL). This study was terminated early and analysis was not done.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 months

Population: This study was terminated early and analysis was not done.

The median number of days to PMN engraftment criteria was PMN counts ≥ 0.5\*10\^9/L for 3 consecutive days or ≥ 1.0\*10\^9/L for 1 day. Time to engraftment corresponded to the first day that criteria were met. This study was terminated early and analysis was not done.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 months

Population: This study was terminated early and analysis was not done.

The median number of days to platelet (PLT) engraftment criteria was ≥ 20\*10\^9/L platelets without transfusion for the preceding 7 days. Time to engraftment corresponded to the first day that criteria were met. This study was terminated early and analysis was not done.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 13 months

Population: This study was terminated early and analysis was not done.

Participants with durable grafts. Graft durability was assessed by complete blood count (CBC) and differential analysis at 12 months post-transplantation. This study was terminated early and analysis was not done.

Outcome measures

Outcome data not reported

Adverse Events

All Patients

Serious events: 4 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	All Patients n=5 participants at risk All patients (3 NHL, 1 MM, and 1 HD).
Cardiac disorders Acute myocardial infarction	20.0% 1/5 • All treatment-emergent AEs are summarized. An event whose onset was on or after the first administration of study treatment (either G-CSF or plerixafor) was considered treatment-emergent. 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.
Gastrointestinal disorders Vomiting	20.0% 1/5 • All treatment-emergent AEs are summarized. An event whose onset was on or after the first administration of study treatment (either G-CSF or plerixafor) was considered treatment-emergent. 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.
General disorders Disease progression	20.0% 1/5 • All treatment-emergent AEs are summarized. An event whose onset was on or after the first administration of study treatment (either G-CSF or plerixafor) was considered treatment-emergent. 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.
Renal and urinary disorders Renal failure acute	20.0% 1/5 • All treatment-emergent AEs are summarized. An event whose onset was on or after the first administration of study treatment (either G-CSF or plerixafor) was considered treatment-emergent. 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.
Respiratory, thoracic and mediastinal disorders Interstitial lung disease	20.0% 1/5 • All treatment-emergent AEs are summarized. An event whose onset was on or after the first administration of study treatment (either G-CSF or plerixafor) was considered treatment-emergent. 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

Other adverse events
Measure	All Patients n=5 participants at risk All patients (3 NHL, 1 MM, and 1 HD).
General disorders Chills	20.0% 1/5 • All treatment-emergent AEs are summarized. An event whose onset was on or after the first administration of study treatment (either G-CSF or plerixafor) was considered treatment-emergent. 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.
Gastrointestinal disorders Abdominal pain	20.0% 1/5 • All treatment-emergent AEs are summarized. An event whose onset was on or after the first administration of study treatment (either G-CSF or plerixafor) was considered treatment-emergent. 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.
Gastrointestinal disorders Constipation	20.0% 1/5 • All treatment-emergent AEs are summarized. An event whose onset was on or after the first administration of study treatment (either G-CSF or plerixafor) was considered treatment-emergent. 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.
General disorders Fatigue	20.0% 1/5 • All treatment-emergent AEs are summarized. An event whose onset was on or after the first administration of study treatment (either G-CSF or plerixafor) was considered treatment-emergent. 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.
Gastrointestinal disorders Nausea	60.0% 3/5 • All treatment-emergent AEs are summarized. An event whose onset was on or after the first administration of study treatment (either G-CSF or plerixafor) was considered treatment-emergent. 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.
General disorders Pyrexia	20.0% 1/5 • All treatment-emergent AEs are summarized. An event whose onset was on or after the first administration of study treatment (either G-CSF or plerixafor) was considered treatment-emergent. 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.
Infections and infestations Infection	20.0% 1/5 • All treatment-emergent AEs are summarized. An event whose onset was on or after the first administration of study treatment (either G-CSF or plerixafor) was considered treatment-emergent. 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 Hypokalaemia	20.0% 1/5 • All treatment-emergent AEs are summarized. An event whose onset was on or after the first administration of study treatment (either G-CSF or plerixafor) was considered treatment-emergent. 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.
Musculoskeletal and connective tissue disorders Back pain	20.0% 1/5 • All treatment-emergent AEs are summarized. An event whose onset was on or after the first administration of study treatment (either G-CSF or plerixafor) was considered treatment-emergent. 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.
Musculoskeletal and connective tissue disorders Musculoskeletal pain	20.0% 1/5 • All treatment-emergent AEs are summarized. An event whose onset was on or after the first administration of study treatment (either G-CSF or plerixafor) was considered treatment-emergent. 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.
Musculoskeletal and connective tissue disorders Pain in extremity	20.0% 1/5 • All treatment-emergent AEs are summarized. An event whose onset was on or after the first administration of study treatment (either G-CSF or plerixafor) was considered treatment-emergent. 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.
Nervous system disorders Headache	40.0% 2/5 • All treatment-emergent AEs are summarized. An event whose onset was on or after the first administration of study treatment (either G-CSF or plerixafor) was considered treatment-emergent. 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.
Nervous system disorders Paraesthesia	20.0% 1/5 • All treatment-emergent AEs are summarized. An event whose onset was on or after the first administration of study treatment (either G-CSF or plerixafor) was considered treatment-emergent. 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.
Respiratory, thoracic and mediastinal disorders Cough	60.0% 3/5 • All treatment-emergent AEs are summarized. An event whose onset was on or after the first administration of study treatment (either G-CSF or plerixafor) was considered treatment-emergent. 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.
Respiratory, thoracic and mediastinal disorders Paranasal sinus hypersecretion	20.0% 1/5 • All treatment-emergent AEs are summarized. An event whose onset was on or after the first administration of study treatment (either G-CSF or plerixafor) was considered treatment-emergent. 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.
Skin and subcutaneous tissue disorders Skin reaction	20.0% 1/5 • All treatment-emergent AEs are summarized. An event whose onset was on or after the first administration of study treatment (either G-CSF or plerixafor) was considered treatment-emergent. 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.
Skin and subcutaneous tissue disorders Skin tightness	20.0% 1/5 • All treatment-emergent AEs are summarized. An event whose onset was on or after the first administration of study treatment (either G-CSF or plerixafor) was considered treatment-emergent. 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.

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