Trial Outcomes & Findings for Activated White Blood Cells With ASCT for Newly Diagnosed Multiple Myeloma (NCT NCT00566098)
NCT ID: NCT00566098
Last Updated: 2023-05-26
Results Overview
Days to absolute neutrophil count \> 500 cells per microliter.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
26 participants
Primary outcome timeframe
Up to 1 year
Results posted on
2023-05-26
Participant Flow
There was one screen failure.
Participant milestones
| Measure |
MILs in Patients Undergoing an Autologous Peripheral SCT
therapeutic autologous lymphocytes
therapeutic tumor infiltrating lymphocytes
melphalan
autologous hematopoietic stem cell transplantation
|
|---|---|
|
Collection of MILs
STARTED
|
25
|
|
Collection of MILs
COMPLETED
|
23
|
|
Collection of MILs
NOT COMPLETED
|
2
|
|
Generation of MILs Product
STARTED
|
23
|
|
Generation of MILs Product
COMPLETED
|
22
|
|
Generation of MILs Product
NOT COMPLETED
|
1
|
|
Administration of ASCT+MILs
STARTED
|
22
|
|
Administration of ASCT+MILs
COMPLETED
|
4
|
|
Administration of ASCT+MILs
NOT COMPLETED
|
18
|
Reasons for withdrawal
| Measure |
MILs in Patients Undergoing an Autologous Peripheral SCT
therapeutic autologous lymphocytes
therapeutic tumor infiltrating lymphocytes
melphalan
autologous hematopoietic stem cell transplantation
|
|---|---|
|
Collection of MILs
Withdrawal by Subject
|
1
|
|
Collection of MILs
Relapsed
|
1
|
|
Generation of MILs Product
Relapsed
|
1
|
|
Administration of ASCT+MILs
Death
|
10
|
|
Administration of ASCT+MILs
Lack of Efficacy
|
8
|
Baseline Characteristics
Activated White Blood Cells With ASCT for Newly Diagnosed Multiple Myeloma
Baseline characteristics by cohort
| Measure |
MILs in Patients Undergoing an Autologous Peripheral SCT
n=22 Participants
therapeutic autologous lymphocytes
therapeutic tumor infiltrating lymphocytes
melphalan
autologous hematopoietic stem cell transplantation
|
|---|---|
|
Age, Continuous
|
56 years
n=39 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=39 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=39 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=39 Participants
|
|
Race (NIH/OMB)
White
|
18 Participants
n=39 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=39 Participants
|
|
Region of Enrollment
United States
|
22 Participants
n=39 Participants
|
PRIMARY outcome
Timeframe: Up to 1 yearDays to absolute neutrophil count \> 500 cells per microliter.
Outcome measures
| Measure |
MILs in Patients Undergoing an Autologous Peripheral SCT
n=22 Participants
therapeutic autologous lymphocytes
therapeutic tumor infiltrating lymphocytes
melphalan
autologous hematopoietic stem cell transplantation
|
|---|---|
|
Hematopoietic Engraftment
|
17.9 days
Interval 12.0 to 32.0
|
PRIMARY outcome
Timeframe: Up to 2 yearsPercentage of participants with partial or complete response by Bladé criteria. Partial response is defined as a \>= 50% decrease in serum paraprotein or 90% decrease in urinary light chains (for participants without measurable serum paraprotein). Complete response is defined as negative serum and urine immunofixation and a bone marrow aspirate with \< 5% plasma cells.
Outcome measures
| Measure |
MILs in Patients Undergoing an Autologous Peripheral SCT
n=22 Participants
therapeutic autologous lymphocytes
therapeutic tumor infiltrating lymphocytes
melphalan
autologous hematopoietic stem cell transplantation
|
|---|---|
|
Disease Response
Complete Response
|
7 Participants
|
|
Disease Response
Partial Response
|
6 Participants
|
|
Disease Response
Stable Disease
|
6 Participants
|
|
Disease Response
Progressive Disease
|
3 Participants
|
PRIMARY outcome
Timeframe: Up to 1 yearSuccess rate of expanding MILs in vitro and obtaining a protocol-specified product.
Outcome measures
| Measure |
MILs in Patients Undergoing an Autologous Peripheral SCT
n=22 Participants
therapeutic autologous lymphocytes
therapeutic tumor infiltrating lymphocytes
melphalan
autologous hematopoietic stem cell transplantation
|
|---|---|
|
Feasibility of MILs Generation as Assessed by Percentage of Participants With Successful MIL Generation
|
100 percentage of participants
|
SECONDARY outcome
Timeframe: Days 14, 28, 60, 180, and 360Population: Data was not collected on one participant at the Day 28 and 180 timepoints, and data was not collected for eight participants at the Day 360 timepoint. Data was not collected on CD3+/CD4+/CD8+ cells for all participants.
ALC counts trending over time.
Outcome measures
| Measure |
MILs in Patients Undergoing an Autologous Peripheral SCT
n=22 Participants
therapeutic autologous lymphocytes
therapeutic tumor infiltrating lymphocytes
melphalan
autologous hematopoietic stem cell transplantation
|
|---|---|
|
T-cell Reconstitution as Determined by Absolute Lymphocyte Count (ALC)
Day 360
|
1660 cells per microliter
Interval 690.0 to 2420.0
|
|
T-cell Reconstitution as Determined by Absolute Lymphocyte Count (ALC)
Day 14
|
756 cells per microliter
Interval 105.0 to 2397.0
|
|
T-cell Reconstitution as Determined by Absolute Lymphocyte Count (ALC)
Day 28
|
1768 cells per microliter
Interval 349.0 to 3760.0
|
|
T-cell Reconstitution as Determined by Absolute Lymphocyte Count (ALC)
Day 60
|
1700 cells per microliter
Interval 530.0 to 3500.0
|
|
T-cell Reconstitution as Determined by Absolute Lymphocyte Count (ALC)
Day 180
|
1430 cells per microliter
Interval 650.0 to 2340.0
|
SECONDARY outcome
Timeframe: Up to 129 monthsSurvival in months for participants who are alive (Overall Survival) and alive without disease progression (Progression-free survival). Disease progression is defined as a change from negative to positive on immunofixation or electrophoresis for participants previously in complete remission or a 25% increase in serum electrophoresis for participants not previously in complete remission. Partial response is defined as a \>= 50% decrease in serum paraprotein or 90% decrease in urinary light chains (for participants without measurable serum paraprotein). Complete response is defined as negative serum and urine immunofixation and a bone marrow aspirate with \< 5% plasma cells.
Outcome measures
| Measure |
MILs in Patients Undergoing an Autologous Peripheral SCT
n=22 Participants
therapeutic autologous lymphocytes
therapeutic tumor infiltrating lymphocytes
melphalan
autologous hematopoietic stem cell transplantation
|
|---|---|
|
Survival
Overall survival
|
112.1 months
Interval 39.9 to 128.7
|
|
Survival
Progression-free survival
|
12.3 months
Interval 8.8 to 69.03
|
SECONDARY outcome
Timeframe: At time of bone marrow harvest, Day 60 post-transplant, Day 180 post-transplant, and Day 360 post-transplantCRM-197 Prevnar-specific vaccine responses that measure the T-cell response of the vaccine quantified as %CD3+/CFSE-low/IFN-gamma+.
Outcome measures
| Measure |
MILs in Patients Undergoing an Autologous Peripheral SCT
n=22 Participants
therapeutic autologous lymphocytes
therapeutic tumor infiltrating lymphocytes
melphalan
autologous hematopoietic stem cell transplantation
|
|---|---|
|
Pneumococcal-specific Vaccine Responses
At time of bone marrow harvest
|
21.5 %CD3+/CFSE-low/IFN-gamma+
Interval 0.12 to 47.9
|
|
Pneumococcal-specific Vaccine Responses
Day 60 post-transplant
|
14.4 %CD3+/CFSE-low/IFN-gamma+
Interval 0.0 to 38.2
|
|
Pneumococcal-specific Vaccine Responses
Day 180 post-transplant
|
23.4 %CD3+/CFSE-low/IFN-gamma+
Interval 7.0 to 53.0
|
|
Pneumococcal-specific Vaccine Responses
Day 360 post-transplant
|
20.1 %CD3+/CFSE-low/IFN-gamma+
Interval 0.0 to 49.7
|
SECONDARY outcome
Timeframe: At time of bone marrow harvest, Day 60 post-transplant, Day 180 post-transplant, and Day 360 post-transplantMyeloma lysate response that measures the T-cell response quantified as %CD3+/CFSE-low/IFN-gamma+.
Outcome measures
| Measure |
MILs in Patients Undergoing an Autologous Peripheral SCT
n=22 Participants
therapeutic autologous lymphocytes
therapeutic tumor infiltrating lymphocytes
melphalan
autologous hematopoietic stem cell transplantation
|
|---|---|
|
Anti-tumor Immune Responses
Day 360 post-transplant
|
12.7 %CD3+/CFSE-low/IFN-gamma+
Interval 0.0 to 31.8
|
|
Anti-tumor Immune Responses
At time of bone marrow harvest
|
1.58 %CD3+/CFSE-low/IFN-gamma+
Interval 0.0 to 12.6
|
|
Anti-tumor Immune Responses
Day 60 post-transplant
|
14.1 %CD3+/CFSE-low/IFN-gamma+
Interval 0.0 to 44.3
|
|
Anti-tumor Immune Responses
Day 180 post-transplant
|
18.1 %CD3+/CFSE-low/IFN-gamma+
Interval 0.6 to 50.1
|
Adverse Events
ASCT+MILs
Serious events: 1 serious events
Other events: 21 other events
Deaths: 10 deaths
Serious adverse events
| Measure |
ASCT+MILs
n=22 participants at risk
Autologous stem cell transplant with a conditioning regimen of melphalan 100 mg/m\^2 on each of Days -2 and -1. Infusion of activated marrow infiltrating lymphocytes (MILs) on Day 3. PCV13 vaccine will be given before and/or after Day 0 depending on when participants are enrolled.
|
|---|---|
|
Nervous system disorders
Syncope
|
4.5%
1/22 • Number of events 1 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
Other adverse events
| Measure |
ASCT+MILs
n=22 participants at risk
Autologous stem cell transplant with a conditioning regimen of melphalan 100 mg/m\^2 on each of Days -2 and -1. Infusion of activated marrow infiltrating lymphocytes (MILs) on Day 3. PCV13 vaccine will be given before and/or after Day 0 depending on when participants are enrolled.
|
|---|---|
|
Musculoskeletal and connective tissue disorders
Pain - abdomen
|
22.7%
5/22 • Number of events 5 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
13.6%
3/22 • Number of events 3 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Nervous system disorders
Blurred vision
|
9.1%
2/22 • Number of events 2 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
13.6%
3/22 • Number of events 3 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Gastrointestinal disorders
Constipation
|
22.7%
5/22 • Number of events 5 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
13.6%
3/22 • Number of events 3 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Gastrointestinal disorders
Anorexia
|
40.9%
9/22 • Number of events 9 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Nervous system disorders
Depression
|
9.1%
2/22 • Number of events 2 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Gastrointestinal disorders
Diarrhea
|
54.5%
12/22 • Number of events 13 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
18.2%
4/22 • Number of events 4 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
22.7%
5/22 • Number of events 5 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
General disorders
Fatigue
|
77.3%
17/22 • Number of events 19 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Investigations
Fever
|
18.2%
4/22 • Number of events 4 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Nervous system disorders
Headache
|
31.8%
7/22 • Number of events 8 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Cardiac disorders
Hypotension
|
27.3%
6/22 • Number of events 6 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Nervous system disorders
Insomnia
|
9.1%
2/22 • Number of events 2 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Skin and subcutaneous tissue disorders
Itching
|
9.1%
2/22 • Number of events 3 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Nervous system disorders
Lightheadedness
|
9.1%
2/22 • Number of events 2 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Gastrointestinal disorders
Mucositis
|
40.9%
9/22 • Number of events 11 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Gastrointestinal disorders
Nausea
|
72.7%
16/22 • Number of events 18 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Nervous system disorders
Neuropathy
|
68.2%
15/22 • Number of events 21 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Infections and infestations
Neutropenic fever
|
22.7%
5/22 • Number of events 5 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Musculoskeletal and connective tissue disorders
Pain - calf
|
9.1%
2/22 • Number of events 2 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Skin and subcutaneous tissue disorders
Rash
|
54.5%
12/22 • Number of events 22 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Skin and subcutaneous tissue disorders
Sore throat
|
31.8%
7/22 • Number of events 7 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Cardiac disorders
Tachycardia
|
13.6%
3/22 • Number of events 3 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Infections and infestations
Upper respiratory infection
|
9.1%
2/22 • Number of events 3 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Renal and urinary disorders
Urinary frequency
|
18.2%
4/22 • Number of events 4 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
|
Gastrointestinal disorders
Vomiting
|
50.0%
11/22 • Number of events 11 • Up to Day 360
Per protocol, only adverse events (AEs) that were attributed to the study product (MILs) were collected and reported. AEs were collected daily through the date of engraftment, weekly through Day 28, and then at Days 60, 180, and 360.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place