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Study of T Cells Targeting B-Cell Maturation Antigen for Previously Treated Multiple Myeloma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02215967
Recruitment Status : Completed
First Posted : August 13, 2014
Results First Posted : October 8, 2019
Last Update Posted : October 8, 2019
Sponsor:
Information provided by (Responsible Party):
James Kochenderfer, M.D., National Cancer Institute (NCI)

Study Type Interventional
Study Design Intervention Model: Sequential Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Myeloma, Plasma-Cell
Myeloma-Multiple
Interventions Drug: Cyclophosphamide
Drug: Fludarabine
Biological: Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells
Enrollment 30
Recruitment Details  
Pre-assignment Details  
Arm/Group Title 0.3 x 10^6 CAR-BCMA T-cells Infused 1 x 10^6 CAR-BCMA T-cells Infused 3 x 10^6 CAR-BCMA T-cells Infused 9 x 10^6 CAR-BCMA T-cells Infused
Hide Arm/Group Description

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Period Title: Overall Study
Started 3 3 5 19
Completed 3 3 4 16
Not Completed 0 0 1 3
Reason Not Completed
Patient became ineligible for treatment             0             0             0             3
Patient did not get CAR T-Cells             0             0             1             0
Arm/Group Title 0.3 x 10^6 CAR-BCMA T-cells Infused 1 x 10^6 CAR-BCMA T-cells Infused 3 x 10^6 CAR-BCMA T-cells Infused 9 x 10^6 CAR-BCMA T-cells Infused Total
Hide Arm/Group Description

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Total of all reporting groups
Overall Number of Baseline Participants 3 3 5 19 30
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 5 participants 19 participants 30 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
3
 100.0%
3
 100.0%
5
 100.0%
17
  89.5%
28
  93.3%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
2
  10.5%
2
   6.7%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 3 participants 3 participants 5 participants 19 participants 30 participants
58.93  (4.87) 54.0  (.71) 55.76  (5.65) 54.13  (6.86) 56.49  (5.8)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 5 participants 19 participants 30 participants
Female
3
 100.0%
1
  33.3%
4
  80.0%
7
  36.8%
15
  50.0%
Male
0
   0.0%
2
  66.7%
1
  20.0%
12
  63.2%
15
  50.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 5 participants 19 participants 30 participants
Hispanic or Latino
0
   0.0%
0
   0.0%
0
   0.0%
4
  21.1%
4
  13.3%
Not Hispanic or Latino
3
 100.0%
3
 100.0%
5
 100.0%
14
  73.7%
25
  83.3%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
1
   5.3%
1
   3.3%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 5 participants 19 participants 30 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
1
  33.3%
0
   0.0%
2
  40.0%
2
  10.5%
5
  16.7%
White
2
  66.7%
3
 100.0%
3
  60.0%
15
  78.9%
23
  76.7%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
2
  10.5%
2
   6.7%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 3 participants 3 participants 5 participants 19 participants 30 participants
3 3 5 19 30
1.Primary Outcome
Title Number of Participants With Dose Limiting Toxicities
Hide Description Dose limiting toxicities are defined as follows: Grade 3 toxicities possibly or probably related to either the anti-BCMA CAR T cells or the fludarabine and cyclophosphamide chemotherapy and lasting more than 7 days. Grade 4 toxicities possibly or probably related to the study interventions.
Time Frame After the start of treatment and up to 60 days
Hide Outcome Measure Data
Hide Analysis Population Description
One participant in Group 9x10(6) did not get CAR T-cells and was deemed ineligible, and 3 participants in Group 9x10(6) were found to be ineligible for treatment.
Arm/Group Title 0.3 x 10^6 CAR-BCMA T-cells Infused 1 x 10^6 CAR-BCMA T-cells Infused 3 x 10^6 CAR-BCMA T-cells Infused 9 x 10^6 CAR-BCMA T-cells Infused
Hide Arm/Group Description:

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Overall Number of Participants Analyzed 3 3 4 16
Measure Type: Count of Participants
Unit of Measure: Participants
Hypophosphatemia
0
   0.0%
0
   0.0%
0
   0.0%
4
  25.0%
Confusion
0
   0.0%
0
   0.0%
0
   0.0%
3
  18.8%
Dyspnea (intubated)
0
   0.0%
0
   0.0%
0
   0.0%
4
  25.0%
Ejection fraction decreased
0
   0.0%
0
   0.0%
0
   0.0%
4
  25.0%
Encephalopathy
0
   0.0%
0
   0.0%
0
   0.0%
3
  18.8%
Hypoxia
0
   0.0%
0
   0.0%
0
   0.0%
3
  18.8%
Acute kidney injury (CVVH)
0
   0.0%
0
   0.0%
0
   0.0%
4
  25.0%
Sepsis (Staph Aureus)
0
   0.0%
0
   0.0%
0
   0.0%
4
  25.0%
Muscle weakness lower limbs
0
   0.0%
0
   0.0%
0
   0.0%
3
  18.8%
Muscle weakness upper limbs
0
   0.0%
0
   0.0%
0
   0.0%
3
  18.8%
Platelet count decreased
0
   0.0%
0
   0.0%
0
   0.0%
3
  18.8%
Neutrophil count decreased
0
   0.0%
0
   0.0%
0
   0.0%
4
  25.0%
Acute kidney injury
0
   0.0%
0
   0.0%
0
   0.0%
3
  18.8%
Hypotension
0
   0.0%
0
   0.0%
0
   0.0%
4
  25.0%
CPK increased
0
   0.0%
0
   0.0%
0
   0.0%
4
  25.0%
Musculoskeletal/connective tissue disorders-Rhabd.
0
   0.0%
0
   0.0%
0
   0.0%
3
  18.8%
2.Primary Outcome
Title Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0)
Hide Description Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.
Time Frame Date treatment consent signed to date off study, approx. 3 mos and 7 days for DL 0.3 x 10^6 CAR + T cells, 4 mos and 4 days for 1.0 x 10^6 CAR + T cells, 9 mos and 13 days for 3.0 x 10^6 CAR + T cells, and 48 mos and 12 days for 9.0 x 10^6 CAR + T cells.
Hide Outcome Measure Data
Hide Analysis Population Description
One participant in Group 9x10(6) did not get CAR T-cells and was deemed ineligible, and 3 participants in Group 9x10(6) were found to be ineligible for treatment.
Arm/Group Title 0.3 x 10^6 CAR-BCMA T-cells Infused 1 x 10^6 CAR-BCMA T-cells Infused 3 x 10^6 CAR-BCMA T-cells Infused 9 x 10^6 CAR-BCMA T-cells Infused
Hide Arm/Group Description:

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Overall Number of Participants Analyzed 3 3 4 16
Measure Type: Count of Participants
Unit of Measure: Participants
3
 100.0%
3
 100.0%
4
 100.0%
16
 100.0%
3.Secondary Outcome
Title Number of Participants With Best Response
Hide Description Best response was assessed by the International Myeloma Working Group response criteria. Partial Remission (PR) is 50% or greater reduction of serum M-protein and 90% or greater reduction in 24-h urinary M-protein. Progressive Disease (PD) is increases of greater or equal to 25% from the lowest post-treatment (nadir) value in serum M-component or urine component or percentage of bone marrow plasma cells. Definite development of new bone lesions or new plasmacytoma. Very Good Partial Remission (VGPR) is serum and urine M-protein detectable by immunofixation but not on electrophoresis. Stringent Complete Remission (sCR) is normal free light chain ratio and absence of clonal cells in bone marrow by immunohistochemistry. Complete Remission is negative immunofixation on the serum and urine. Stable Disease (SD) is not meeting criteria for CR, VGPR, PR or PD.
Time Frame From start of treatment up to 84 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
One participant in Group 9x10(6) did not get CAR T-cells and was deemed ineligible, and 3 participants in Group 9x10(6) were found to be ineligible for treatment.
Arm/Group Title 0.3 x 10^6 CAR-BCMA T-cells Infused 1 x 10^6 CAR-BCMA T-cells Infused 3 x 10^6 CAR-BCMA T-cells Infused 9 x 10^6 CAR-BCMA T-cells Infused
Hide Arm/Group Description:

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Overall Number of Participants Analyzed 3 3 4 16
Measure Type: Count of Participants
Unit of Measure: Participants
Partial Remission
1
  33.3%
0
   0.0%
0
   0.0%
4
  25.0%
Stable Disease
2
  66.7%
3
 100.0%
3
  75.0%
2
  12.5%
Very Good Partial Remission
0
   0.0%
0
   0.0%
1
  25.0%
6
  37.5%
Complete Remission
0
   0.0%
0
   0.0%
0
   0.0%
1
   6.3%
Stringent Complete Remission
0
   0.0%
0
   0.0%
0
   0.0%
2
  12.5%
Progressive Disease
0
   0.0%
0
   0.0%
0
   0.0%
1
   6.3%
Time Frame Date treatment consent signed to date off study, approximately 3 months and 7 days for dose level 0.3 x 10^6 CAR + T cells, 4 months and 4 days for 1.0 x 10^6 CAR + T cells, 9 months and 13 days for 3.0 x 10^6 CAR + T cells, and 48 months and 12 days for 9.0 x 10^6 CAR + T cells.
Adverse Event Reporting Description One participant in Group 9x10(6) did not get CAR T-cells and was deemed ineligible, and 3 participants in Group 9x10(6) were found to be ineligible for treatment.
 
Arm/Group Title 0.3 x 10^6 CAR-BCMA T-cells Infused 1 x 10^6 CAR-BCMA T-cells Infused 3 x 10^6 CAR-BCMA T-cells Infused 9 x 10^6 CAR-BCMA T-cells Infused
Hide Arm/Group Description

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

Dose Escalation with 5 dose levels based on the patients actual bodyweight

Cyclophosphamide: 300 mg/m^2 intravenous (IV) over 30 minutes on days -5, -4, and -3

Fludarabine: 30 mg/m^2 intravenous (IV) over 30 minutes immediately following the cyclophosphamide on day -5, -4, and -3

Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells: 0.3x10^6- 15.0x10^6 CAR+ T cells per kg of recipient bodyweight one time dose on day 0

All-Cause Mortality
0.3 x 10^6 CAR-BCMA T-cells Infused 1 x 10^6 CAR-BCMA T-cells Infused 3 x 10^6 CAR-BCMA T-cells Infused 9 x 10^6 CAR-BCMA T-cells Infused
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/3 (0.00%)      0/3 (0.00%)      0/4 (0.00%)      0/16 (0.00%)    
Hide Serious Adverse Events
0.3 x 10^6 CAR-BCMA T-cells Infused 1 x 10^6 CAR-BCMA T-cells Infused 3 x 10^6 CAR-BCMA T-cells Infused 9 x 10^6 CAR-BCMA T-cells Infused
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/3 (33.33%)      0/3 (0.00%)      1/4 (25.00%)      11/16 (68.75%)    
Blood and lymphatic system disorders         
Disseminated intravascular coagulation  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Cardiac disorders         
Sinus tachycardia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/4 (25.00%)  1 3/16 (18.75%)  5
Supraventricular tachycardia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Gastrointestinal disorders         
Diarrhea  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 2/16 (12.50%)  5
General disorders         
Fever  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/4 (25.00%)  1 1/16 (6.25%)  2
Hypothermia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Pain  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Infections and infestations         
Infections and infestations - Other, CMV viremia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Infections and infestations - Other, Salmonella  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  3
Lung infection  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 2/16 (12.50%)  2
Sepsis  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 2/16 (12.50%)  2
Upper respiratory infection  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 2/16 (12.50%)  2
Investigations         
Neutrophil count decreased  1  1/3 (33.33%)  1 0/3 (0.00%)  0 1/4 (25.00%)  1 2/16 (12.50%)  2
White blood cell decreased  1  1/3 (33.33%)  1 0/3 (0.00%)  0 1/4 (25.00%)  2 0/16 (0.00%)  0
CPK increased  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  5
Cardiac troponin T increased  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Ejection fraction decreased  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  4
Platelet count decreased  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 4/16 (25.00%)  4
Metabolism and nutrition disorders         
Acidosis  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  2
Hypernatremia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Musculoskeletal and connective tissue disorders         
Muscle weakness lower limb  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Muscle weakness upper limb  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Musculoskeletal and connective tissue disorder - Other, Rhabdomyalysis  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  2
Nervous system disorders         
Encephalopathy  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Psychiatric disorders         
Confusion  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  3
Delirium  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 2/16 (12.50%)  2
Renal and urinary disorders         
Acute kidney injury  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 2/16 (12.50%)  2
Respiratory, thoracic and mediastinal disorders         
Dyspnea  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/4 (25.00%)  1 5/16 (31.25%)  7
Hypoxia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 3/16 (18.75%)  5
Vascular disorders         
Hypotension  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/4 (25.00%)  1 5/16 (31.25%)  6
Thromboembolic event  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
1
Term from vocabulary, CTCAE (4.0)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
0.3 x 10^6 CAR-BCMA T-cells Infused 1 x 10^6 CAR-BCMA T-cells Infused 3 x 10^6 CAR-BCMA T-cells Infused 9 x 10^6 CAR-BCMA T-cells Infused
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   3/3 (100.00%)      3/3 (100.00%)      4/4 (100.00%)      16/16 (100.00%)    
Blood and lymphatic system disorders         
Anemia  1  3/3 (100.00%)  4 2/3 (66.67%)  2 4/4 (100.00%)  7 13/16 (81.25%)  42
Febrile neutropenia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/4 (25.00%)  1 1/16 (6.25%)  1
Cardiac disorders         
Atrial fibrillation  1  0/3 (0.00%)  0 1/3 (33.33%)  1 0/4 (0.00%)  0 2/16 (12.50%)  2
Electrocardiogram QT corrected interval prolonged  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 3/16 (18.75%)  6
Sinus tachycardia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 11/16 (68.75%)  14
Ventricular tachycardia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Gastrointestinal disorders         
Nausea  1  2/3 (66.67%)  2 2/3 (66.67%)  2 1/4 (25.00%)  1 12/16 (75.00%)  15
Fever  1  0/3 (0.00%)  0 2/3 (66.67%)  2 0/4 (0.00%)  0 15/16 (93.75%)  36
Vomiting  1  0/3 (0.00%)  0 1/3 (33.33%)  1 1/4 (25.00%)  1 1/16 (6.25%)  1
Diarrhea  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/4 (25.00%)  1 7/16 (43.75%)  11
Abdominal distension  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Abdominal pain  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 2/16 (12.50%)  2
Constipation  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
General disorders         
Fatigue  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 2/16 (12.50%)  2
Hypothermia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Surgical and medical procedures - Other, specify  1 [1]  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Infections and infestations         
Upper respiratory infection  1  1/3 (33.33%)  1 0/3 (0.00%)  0 1/4 (25.00%)  1 8/16 (50.00%)  11
Catheter related infection  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Infections and infestations - Other, Clostridium difficile stool  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Lung infection  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Skin infection  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Urinary tract infection  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 2/16 (12.50%)  2
Infections and infestations - Other, Clostridium difficile  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 3/16 (18.75%)  3
Investigations         
Lymphocyte count decreased  1  3/3 (100.00%)  9 3/3 (100.00%)  7 4/4 (100.00%)  10 14/16 (87.50%)  49
Neutrophil count decreased  1  3/3 (100.00%)  13 3/3 (100.00%)  12 4/4 (100.00%)  14 16/16 (100.00%)  68
Platelet count decreased  1  2/3 (66.67%)  5 1/3 (33.33%)  1 2/4 (50.00%)  5 11/16 (68.75%)  58
White blood cell decreased  1  3/3 (100.00%)  14 3/3 (100.00%)  14 4/4 (100.00%)  18 16/16 (100.00%)  69
Activated partial thromboplastin time prolonged  1  0/3 (0.00%)  0 1/3 (33.33%)  1 0/4 (0.00%)  0 7/16 (43.75%)  9
Hyponatremia  1  0/3 (0.00%)  0 1/3 (33.33%)  1 0/4 (0.00%)  0 0/16 (0.00%)  0
Alanine aminotransferase increased  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/4 (25.00%)  1 3/16 (18.75%)  3
Acidosis  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Alkalosis  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Aspartate aminotransferase increased  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 3/16 (18.75%)  6
Blood bilirubin increased  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 6/16 (37.50%)  8
Creatinine increased  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 4/16 (25.00%)  4
Ejection fraction decreased  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 4/16 (25.00%)  5
Fibrinogen decreased  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 3/16 (18.75%)  8
INR increased  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Metabolism and nutrition disorders         
Hypocalcemia  1  1/3 (33.33%)  1 1/3 (33.33%)  1 1/4 (25.00%)  1 7/16 (43.75%)  9
Hypophosphatemia  1  1/3 (33.33%)  2 1/3 (33.33%)  1 2/4 (50.00%)  5 14/16 (87.50%)  59
Anorexia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/4 (25.00%)  1 0/16 (0.00%)  0
CPK increased  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/4 (25.00%)  2 2/16 (12.50%)  4
Hypoalbuminemia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/4 (25.00%)  1 15/16 (93.75%)  31
Hyperglycemia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 4/16 (25.00%)  4
Hypermagnesemia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 2/16 (12.50%)  3
Hypernatremia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  2
Hyperuricemia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Hypokalemia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 10/16 (62.50%)  18
Hypomagnesemia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 3/16 (18.75%)  3
Hyponatremia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 3/16 (18.75%)  3
Musculoskeletal and connective tissue disorders         
Bone pain  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Non-cardiac chest pain  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Pain in extremity  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)         
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify  1 [2]  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify  1 [3]  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify  1 [4]  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Nervous system disorders         
Headache  1  1/3 (33.33%)  1 0/3 (0.00%)  0 0/4 (0.00%)  0 3/16 (18.75%)  4
Somnolence  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Syncope  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Psychiatric disorders         
Anxiety  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Confusion  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Delirium  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 4/16 (25.00%)  7
Renal and urinary disorders         
Acute kidney injury  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Respiratory, thoracic and mediastinal disorders         
Dyspnea  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 2/16 (12.50%)  2
Epistaxis  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 1/16 (6.25%)  1
Hypoxia  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 5/16 (31.25%)  5
Skin and subcutaneous tissue disorders         
Rash maculo-papular  1  0/3 (0.00%)  0 0/3 (0.00%)  0 1/4 (25.00%)  1 0/16 (0.00%)  0
Vascular disorders         
Thromboembolic event  1  0/3 (0.00%)  0 1/3 (33.33%)  1 0/4 (0.00%)  0 1/16 (6.25%)  1
Hypertension  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 5/16 (31.25%)  5
Hypotension  1  0/3 (0.00%)  0 0/3 (0.00%)  0 0/4 (0.00%)  0 7/16 (43.75%)  9
1
Term from vocabulary, CTCAE (4.0)
Indicates events were collected by systematic assessment
[1]
Basal cell carcinoma removal (face and upper back)
[2]
Basal cell carcinoma (L base of neck)
[3]
Basal cell carcinoma (L cheek)
[4]
Basal cell carcinoma (upper back)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. James N. Kochenderfer
Organization: National Cancer Institute
Phone: 240-760-6062
EMail: kochendj@mail.nih.gov
Layout table for additonal information
Responsible Party: James Kochenderfer, M.D., National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT02215967    
Other Study ID Numbers: 140168
14-C-0168
First Submitted: August 12, 2014
First Posted: August 13, 2014
Results First Submitted: August 26, 2019
Results First Posted: October 8, 2019
Last Update Posted: October 8, 2019