Trial Outcomes & Findings for Treatment With AMD3100 (Plerixafor) in MM Patients to Mobilize PBCs For Collection and for Transplantation (NCT NCT00396383)
NCT ID: NCT00396383
Last Updated: 2014-03-13
Results Overview
Number of participants achieving a target of ≥ 4\*10\^6 CD34+ cells/kg during apheresis for up to 4 consecutive days. Apheresis was performed six hours following treatment with plerixafor 240 µg/kg (alone). Target was calculated as the sum of all daily values collected from central laboratory data over up to 4 apheresis days.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
9 participants
Primary outcome timeframe
Day 1 up to day 4
Results posted on
2014-03-13
Participant Flow
Study enrollment began in November 2004 and the study was terminated in May 2007. A total of 20 participants were planned for this study, however the study was terminated early because of insufficient mobilization of CD34+ cells after treatment with plerixafor alone for use in tandem transplants.
Participant milestones
| Measure |
Participants With Multiple Myeloma (MM)
Participants with MM who were eligible for autologous peripheral blood stem cell transplantation were given 240 µg/kg daily subcutaneous plerixafor for up to 4 days.
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|---|---|
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Overall Study
STARTED
|
9
|
|
Overall Study
COMPLETED
|
6
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
Participants With Multiple Myeloma (MM)
Participants with MM who were eligible for autologous peripheral blood stem cell transplantation were given 240 µg/kg daily subcutaneous plerixafor for up to 4 days.
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|---|---|
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Overall Study
Death
|
2
|
|
Overall Study
Lost to Follow-up
|
1
|
Baseline Characteristics
Treatment With AMD3100 (Plerixafor) in MM Patients to Mobilize PBCs For Collection and for Transplantation
Baseline characteristics by cohort
| Measure |
Participants With Multiple Myeloma (MM)
n=9 Participants
Participants with MM who were eligible for autologous peripheral blood stem cell transplantation were given 240 µg/kg daily subcutaneous plerixafor for up to 4 days.
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|---|---|
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Age, Continuous
|
62.0 years
STANDARD_DEVIATION 8.0 • n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 1 up to day 4Population: All participants who received plerixafor
Number of participants achieving a target of ≥ 4\*10\^6 CD34+ cells/kg during apheresis for up to 4 consecutive days. Apheresis was performed six hours following treatment with plerixafor 240 µg/kg (alone). Target was calculated as the sum of all daily values collected from central laboratory data over up to 4 apheresis days.
Outcome measures
| Measure |
Participants With Multiple Myeloma (MM)
n=9 Participants
Participants with MM who were eligible for autologous peripheral blood stem cell transplantation were given 240 µg/kg daily subcutaneous plerixafor for up to 4 days.
|
|---|---|
|
Number of Participants Who Achieved ≥4*10^6 CD34+ Cells/kg
Participants Who Achieved ≥4*10^6 cells
|
4 participants
|
|
Number of Participants Who Achieved ≥4*10^6 CD34+ Cells/kg
Participants Who Achieved <4*10^6 cells
|
5 participants
|
PRIMARY outcome
Timeframe: 1 monthPopulation: All participants who received plerixafor
Participant counts of summarized adverse events (AEs) which occurred from the first dose of plerixafor up to the day prior to chemotherapy/ablative treatment. Events were graded according to World Health Organization criteria: Mild (awareness of sign or symptom, but easily tolerated), Moderate (discomfort enough to cause interference with usual activity), Severe (incapacitating with inability to work or do usual activity).
Outcome measures
| Measure |
Participants With Multiple Myeloma (MM)
n=9 Participants
Participants with MM who were eligible for autologous peripheral blood stem cell transplantation were given 240 µg/kg daily subcutaneous plerixafor for up to 4 days.
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|---|---|
|
Participant Counts of Summarized Adverse Events (AE) During Treatment
AE Severity (Mild)
|
3 participants
|
|
Participant Counts of Summarized Adverse Events (AE) During Treatment
AE Severity (Moderate)
|
6 participants
|
|
Participant Counts of Summarized Adverse Events (AE) During Treatment
AE Severity (Severe)
|
0 participants
|
|
Participant Counts of Summarized Adverse Events (AE) During Treatment
Life-threatening AE
|
0 participants
|
|
Participant Counts of Summarized Adverse Events (AE) During Treatment
AE Relationship to Drug (Not related)
|
4 participants
|
|
Participant Counts of Summarized Adverse Events (AE) During Treatment
AE Relationship to Drug (Probably not related)
|
1 participants
|
|
Participant Counts of Summarized Adverse Events (AE) During Treatment
AE Relationship to Drug (Possibly related)
|
1 participants
|
|
Participant Counts of Summarized Adverse Events (AE) During Treatment
AE Relationship to Drug (Probably related)
|
3 participants
|
|
Participant Counts of Summarized Adverse Events (AE) During Treatment
AE Relationship to Drug (Definitely related)
|
0 participants
|
SECONDARY outcome
Timeframe: Approximately 2 monthsPopulation: Intent to treat population includes participants who received plerixafor and underwent transplantation. One participant had two transplants.
Polymorphonuclear cell (PMN) engraftment was defined as a PMN count ≥ 0.5\*10\^9/L for 3 consecutive days or ≥ 1\*10\^9/L for 1 day. Days to engraftment corresponded to the first day that the criteria were met after transplantation.
Outcome measures
| Measure |
Participants With Multiple Myeloma (MM)
n=10 transplantations
Participants with MM who were eligible for autologous peripheral blood stem cell transplantation were given 240 µg/kg daily subcutaneous plerixafor for up to 4 days.
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|---|---|
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Number of Transplantations That Achieved Polymorphonuclear Leukocyte (PMN) Engraftment Grouped by Days to Engraftment
<= Day 12 post transplant
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10 transplantations
|
|
Number of Transplantations That Achieved Polymorphonuclear Leukocyte (PMN) Engraftment Grouped by Days to Engraftment
Day 13 to 21 post transplant
|
0 transplantations
|
|
Number of Transplantations That Achieved Polymorphonuclear Leukocyte (PMN) Engraftment Grouped by Days to Engraftment
>= Day 22 post transplant
|
0 transplantations
|
SECONDARY outcome
Timeframe: Approximately 2 monthsPopulation: Intent to treat population includes participants who received plerixafor and underwent transplantation. One participant had two transplants. One participant did not have PLT samples collected (included as 'unknown' in data table).
Platelet (PLT) engraftment was defined as a PLT count of ≥ 20\*10\^9/L for 7 days without transfusion. Days to engraftment corresponded to the first day that the criteria were met after transplantation.
Outcome measures
| Measure |
Participants With Multiple Myeloma (MM)
n=10 transplantations
Participants with MM who were eligible for autologous peripheral blood stem cell transplantation were given 240 µg/kg daily subcutaneous plerixafor for up to 4 days.
|
|---|---|
|
Number of Transplantations That Achieved Platelet (PLT) Engraftment Grouped by Days to Engraftment
<= Day 12 post transplant
|
0 transplantations
|
|
Number of Transplantations That Achieved Platelet (PLT) Engraftment Grouped by Days to Engraftment
Day 13 to 21 post transplant
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6 transplantations
|
|
Number of Transplantations That Achieved Platelet (PLT) Engraftment Grouped by Days to Engraftment
>= Day 22 post transplant
|
3 transplantations
|
|
Number of Transplantations That Achieved Platelet (PLT) Engraftment Grouped by Days to Engraftment
Unknown
|
1 transplantations
|
SECONDARY outcome
Timeframe: Approximately month 13Population: Intent to treat population includes participants who received plerixafor, underwent transplantation, and were evaluable 12 months post transplant.
Graft durability was assessed by the Investigator based on complete blood count (CBC) and differential analyses at 12 months post transplantation.
Outcome measures
| Measure |
Participants With Multiple Myeloma (MM)
n=6 Participants
Participants with MM who were eligible for autologous peripheral blood stem cell transplantation were given 240 µg/kg daily subcutaneous plerixafor for up to 4 days.
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|---|---|
|
Number of Participants With a Durable Graft at 12 Months Post Transplantation
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6 participants
|
POST_HOC outcome
Timeframe: Day 1 up to day 4Population: All participants who received plerixafor
Number of participants achieving ≥ 2\*10\^6 CD34+ cells/kg during apheresis for up to 4 consecutive days. Apheresis was performed six hours following treatment with plerixafor 240 µg/kg (alone). Total was calculated as the sum of all daily values collected from central laboratory data over up to 4 apheresis days.
Outcome measures
| Measure |
Participants With Multiple Myeloma (MM)
n=9 Participants
Participants with MM who were eligible for autologous peripheral blood stem cell transplantation were given 240 µg/kg daily subcutaneous plerixafor for up to 4 days.
|
|---|---|
|
Number of Participants Who Achieved ≥2*10^6 CD34+ Cells/kg
|
9 participants
|
Adverse Events
Participants With Multiple Myeloma (MM)
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Participants With Multiple Myeloma (MM)
n=9 participants at risk
Participants with MM who were eligible for autologous peripheral blood stem cell transplantation were given 240 µg/kg daily subcutaneous plerixafor for up to 4 days.
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|---|---|
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Blood and lymphatic system disorders
Anaemia
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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 distension
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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
Diarrhoea
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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
Paraesthesia oral
|
33.3%
3/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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
Catheter site erythema
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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
Catheter site related reaction
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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
Infusion site bruising
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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
Injection site erythema
|
33.3%
3/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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
Oedema peripheral
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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
Pain
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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
Urinary tract infection
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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
Vulvovaginal mycotic infection
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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.
|
|
Injury, poisoning and procedural complications
Contusion
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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.
|
|
Investigations
Heart rate irregular
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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.
|
|
Investigations
Platelet count decreased
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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.
|
|
Investigations
Syphilis test positive
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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
Bone pain
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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
Dysgeusia
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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
|
22.2%
2/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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
Hypoaesthesia
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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
Poor quality sleep
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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.
|
|
Psychiatric disorders
Insomnia
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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.
|
|
Psychiatric disorders
Restlessness
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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
Night sweats
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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
Rash
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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
Swelling face
|
11.1%
1/9 • The first day of plerixafor administration up to the day prior to chemotherapy/ablative treatment.
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.
|
Additional Information
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