Multiple Myeloma
Conditions
Brief summary
The purpose of this study is to evaluate the efficacy and safety of cilta-cel out-of-specification (OOS).
Interventions
DRUGCilta-cel
Cilta-cel will be administered as an IV infusion.
DRUGLymphodepleting Therapy (Cyclophosphamide and Fludarabine)
Lymphodepleting therapy (cyclophosphamide and fludarabine) will be administered intravenously.
Sponsors
Janssen Scientific Affairs, LLC
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Eligible for treatment with cilta-cel per United States prescribing information (USPI) or locally approved label * Participant is suffering from serious or life threatening multiple myeloma per USPI (or locally approved label, respectively), and re-apheresis, re-manufacturing, or other anti-myeloma directed therapies is not considered feasible or adequate per investigator * Has adequate general health status and organ function per investigator assessment and meets the criteria to receive cilta-cel out-of-specifications (OOS) * Meets the criteria to receive lymphodepleting chemotherapy * A woman of childbearing potential must have a negative highly sensitive serum (beta-human chorionic gonadotropin \[beta-hCG\]) pregnancy test during screening and prior to the first dose of cyclophosphamide and fludarabine
Exclusion criteria
* History of active uncontrolled infection or condition where an administration of cilta-cel OOS constitutes serious health risk to the participant * Known allergies, hypersensitivity, or intolerance to the excipients of cilta-cel OOS including dimethyl sulfoxide (DMSO), dextran 40, or residual kanamycin per USPI * Hepatitis B infection * Hepatitis C infection defined as (anti hepatitis C virus \[HCV\] antibody positive or detectable HCV ribonucleic acid \[RNA\]) or known to have a history of hepatitis C * Seropositive for human immunodeficiency virus (HIV) * Uncontrolled autoimmune disease
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Overall Response Rate (ORR) | From Day 1 (post infusion) up to 18.6 months | ORR is defined as the percentage of participants who had partial response (PR) or better (sCR+CR+VGPR+PR) according to the International Myeloma Working Group (IMWG) response criteria, as assessed by the investigator. CR: Negative immunofixation on the serum and urine and disappearance of any soft tissue plasmacytomas and less than (\<) 5 percentage (%) plasma cells (PCs) in bone marrow; sCR: CR+Normal free light chain ratio and absence of clonal cells in bone marrow by immunohistochemistry or immuno fluorescence; PR: greater than equal to (\>=) 50% reduction of serum M-protein and reduction in 24-hour urinary M-protein by \>= 90 percentage (%) or to \<200 mg/24 hours; VGPR: Serum and urine M-protein detectable by immunofixation but not on electrophoresis or 90% or greater reduction in serum M-protein plus urine M-protein level \<100 mg per 24 hour. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Severity | From Day 1 up to 100 days post cilta-cel OOS infusion (Up to 100 days) | An AE was any untoward medical occurrence in a participant participating in a clinical study that did not necessarily had a causal relationship with the pharmaceutical/biological agent under study. TEAEs were defined as any AE that occurs on or after the start of cilta-cel OOS infusion through 100 days after the cilta-cel OOS infusion or the start of subsequent anti-myeloma therapy, whichever was earlier; or any AE that is considered related to study intervention regardless of the start date of the event; or any AE that was present at baseline (Day 1, pre-infusion of cilta-cel OOS) but worsens in toxicity grade or was subsequently considered related to study intervention by the investigator. Severity was assessed using National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0 as: Grade 1: Mild; Grade 2: Moderate; Grade 3: Severe; Grade 4: Life-threatening; Grade 5: Death related to adverse event. |
| Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs) | From Day 1 up to 100 days post cilta-cel OOS infusion (Up to 100 days) | Number of participants with serious TESAEs were reported. A SAE was defined as any untoward medical occurrence that results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, leads to a congenital anomaly/birth defect in the offspring of a participant, or was an important medical event. TESAEs were defined as any SAE that occurs on or after the start of cilta-cel OOS infusion through 100 days after the cilta-cel OOS infusion or the start of subsequent anti-myeloma therapy, whichever was earlier; or any SAE that was considered related to study intervention regardless of the start date of the event; or any SAE that was present at baseline but worsens in toxicity grade or was subsequently considered related to study intervention by the investigator. |
| Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Cytokine Release Syndrome (CRS), Immune Effector Cell-Associated Neurotoxicity (ICANS), Other Neurotoxicities, and Secondary Primary Malignancies | From Day 1 up to 100 days post cilta-cel OOS infusion (Up to 100 days) | Number of participants with treatment-emergent AESIs: CRS, ICANS, other neurotoxicities, and secondary primary malignancies were reported. AESIs include the anticipated risks of treatment with study drug and events that may be related to multiple sclerosis comorbidities. Treatment-emergent AESIs were defined as any AESI that occurs on or after the start of cilta-cel OOS infusion through 100 days after the cilta-cel OOS infusion or the start of subsequent anti-myeloma therapy, whichever was earlier; or any AESI that was considered related to study intervention regardless of the start date of the event; or any AESI that was present at baseline but worsens in toxicity grade or was subsequently considered related to study intervention by the investigator. |
| Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Prolonged and Recurrent Cytopenias | From Day 1 up to 100 days post cilta-cel OOS infusion (Up to 100 days) | Number of participants with treatment-emergent AESIs: prolonged and recurrent cytopenias were reported. AESIs include the anticipated risks of treatment with study drug and events that may be related to multiple sclerosis comorbidities. It included Thrombocytopenia, Neutropenia, Lymphopenia, and Anaemia. Treatment-emergent AESIs were defined as any AESI that occurs on or after the start of cilta-cel OOS infusion through 100 days after the cilta-cel OOS infusion or the start of subsequent anti-myeloma therapy, whichever was earlier; or any AESI that was considered related to study intervention regardless of the start date of the event; or any AESI that was present at baseline but worsens in toxicity grade or was subsequently considered related to study intervention by the investigator. Severity was assessed using NCI-CTCAE version 5.0 as: Grade 1: Mild; Grade 2: Moderate; Grade 3: Severe; Grade 4: Life-threatening; Grade 5: Death related to adverse event. |
| Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | From Day 1 (post infusion) up to 18.6 months | Number of participants by worst grade abnormalities in clinical laboratory tests: hematology (including coagulation) and chemistry, after cilta-cel OOS infusion were reported. Grades were assessed using NCI-CTCAE (version 5.0), as: Grade 1: Mild; Grade 2: Moderate; Grade 3: Severe; Grade 4: Life-threatening. Participants with normal values or a value in the opposite direction (for laboratory tests with bidirectional toxicities defined) were assigned Grade 0. Only those categories in which at least 1 participant had data were reported. |
| Partial Response (PR) Rate | From Day 1 (post infusion) up to 18.6 months | PR rate was defined as percentage of participants who achieved PR according to IMWG response criteria. PR was defined as per IMWG criteria as \>= 50% reduction of serum M-protein and reduction in 24 hours urinary M-protein by \>= 90% or to \< 200 mg/24hours. If the serum and urine M-protein were unmeasurable, a \>= 50% decrease in the difference between involved and uninvolved Free light chain (FLC) levels was required in the place of the M-protein criteria. If serum and urine M-protein were not measurable, and serum free light assay was also not measurable, \>=50% reduction in bone marrow plasma cells is required in place of M-protein, provided baseline bone marrow plasma cells percentage was \>=30%. In addition to the above listed criteria, if present at baseline, a \>= 50% reduction in the size of soft tissue plasmacytomas was also required. |
| Very Good Partial Response (VGPR) Rate | From Day 1 (post infusion) up to 18.6 months | VGPR rate is defined as percentage of participants who achieved best response of VGPR according to IMWG response criteria. As per IMWG response criteria, VPGR: serum and urine M-component detectable by immunofixation but not on electrophoresis, or \>=90% reduction in serum M-component plus urine M-component \<100 mg/24 hours. |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | From Day 1 up to 100 days post cilta-cel OOS infusion (Up to 100 days) | Number of participants with TEAEs were reported. An AE was any untoward medical occurrence in a participant participating in a clinical study that did not necessarily had a causal relationship with the pharmaceutical/biological agent under study. TEAEs are defined as any AE that occurred on or after the start of cilta-cel OOS infusion through 100 days after the cilta-cel OOS infusion or the start of subsequent anti-myeloma therapy, whichever was earlier; or any AE that was considered related to study intervention regardless of the start date of the event; or any AE that was present at baseline (Day 1, infusion of cilta-cel OOS) but worsens in toxicity grade or is subsequently considered related to study intervention by the investigator. |
| Clinical Benefit Rate (CBR) | From Day 1 (post infusion) up to 18.6 months | Clinical benefit rate was defined as the percentage of participants who achieved a minimal response (MR) or better (including MR, PR, VGPR, CR, and sCR). MR was defined as per IMWG criteria as \>=25% but less than or equal to (\<=) 49% reduction of serum M-protein and reduction in 24-hour urine M-protein by 50% to 89%. In addition to the above criteria, if present at baseline, \>=50% reduction in the size of soft tissue plasmacytomas was also required. |
| Stringent Complete Response (sCR) Rate | From Day 1 (post infusion) up to 18.6 months | sCR rate is defined as percentage of participants who achieved sCR according to IMWG response criteria. sCR is defined per IMWG criteria as CR plus normal FLC ratio, and absence of clonal PCs by immunohistochemistry or 2- to 4-color flow cytometry. CR is defined as per IMWG as negative immunofixation of serum and urine, and disappearance of any soft tissue plasmacytomas, and \<5% PCs in bone marrow. No evidence of initial monoclonal protein isotype(s) on immunofixation of the serum and urine. |
| Duration of Response (DOR) | From Day 1 (post infusion) up to 18.6 months | DOR calculated in responders (with PR or better response) from date of initial documentation of response (PR or better) to date of first documented evidence of progressive disease (PD) as per IMWG criteria, or death due to any cause, whichever occurs first. PD: increase of 25% from lowest response value: serum and urine M-component (absolute increase must be \>=0.5 grams/deciliter \[g/dL\] and \>=200 mg/24 hours, respectively); only in participants without measurable serum and urine M-protein levels, difference between involved and uninvolved FLC levels (absolute increase must be \>10 mg/dL); only in participants without measurable serum and/or urine M-protein levels and without measurable disease by FLC levels, bone marrow PC % (absolute increase must be \>=10%), appearance of new lesion; definite development of new bone lesions or definite increase in the size of existing bone lesions, \>=50% increase in circulating PCs (minimum of 200 cells per uL) if this was the only measure of disease. |
| Progression Free Survival (PFS) | From Day 1 (post infusion) up to 18.6 months | PFS defined as the time from the date of the initial infusion of cilta-cel OOS to the date of first documented disease progression as per IMWG criteria, or death due to any cause, whichever occurs first. PD: increase of 25% from lowest response value: serum and urine M-component (absolute increase must be \>=0.5 g/dL and \>=200 mg/24 hours, respectively); only in participants without measurable serum and/or urine M-protein levels the difference between involved and uninvolved FLC levels (absolute increase must be \>10 mg/dL); only in participants without measurable serum and urine M-protein levels and without measurable disease by FLC levels, bone marrow PC percentage (absolute increase must be \>=10%), appearance of a new lesion; definite development of new bone lesions or definite increase in the size of existing bone lesions, \>=50% increase in circulating PCs (minimum of 200 cells per uL) if this was the only measure of disease. |
| Overall Survival (OS) | From Day 1 (post infusion) up to 18.6 months | OS is measured from the date of the initial infusion of cilta-cel OOS to the date of the participant's death. If the participant is alive or the vital status is unknown, then the participant's data was censored at the date the participant was last known to be alive. |
| Minimal Residual Disease (MRD)-Negative Rate | From Day 1 (post infusion) up to 18.6 months | MRD-negative rate is defined as the percentage of participants with negative MRD status (at 10\^-4, 10\^-5 and 10\^-6 cutoffs) by bone marrow aspirate. |
| Number of Participants With Presence of Replication Competent Lentivirus | Predose, 3, 6, 12 months after cilta-cel infusion on Day 1 | Number of participants with presence of replication competent lentivirus (with evaluable sample) were reported. |
| Complete Response (CR) Rate | From Day 1 (post infusion) up to 18.6 months | CR rate is defined as percentage of participants who achieved best response of CR according to IMWG response criteria. CR is defined as per IMWG criteria as negative immunofixation of serum and urine, and disappearance of any soft tissue plasmacytomas, and \<5% PCs in bone marrow. No evidence of initial monoclonal protein isotype(s) on immunofixation of the serum and urine. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) Adult participants aged 18 years or older, with multiple myeloma received the lymphodepleting chemotherapy (cyclophosphamide 300 milligrams per meter square \[mg/m\^2\] intravenous \[IV\] infusion and fludarabine 30 mg/m\^2 IV infusion daily, for 3 days \[Day -7 to Day -5\]). After 5 to 7 days of initiating lymphodepleting chemotherapy, participants received IV infusion of cilta-cel OOS at a total targeted dose of 0.75\*10\^6 chimeric antigen receptor (CAR)-positive viable T-cells/kg (dose range 0.5\*10\^6 CAR-positive viable T cells/kg to 1.0\*10\^6 CAR-positive viable T cells/kg) based on participant's body weight at apheresis or per exceptional release criteria determined alternative dose. | 82 |
| Total | 82 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Death | 14 |
| Overall Study | Disease Relapse | 2 |
| Overall Study | Enrolled (consented) but not treated | 4 |
| Overall Study | Lost to Follow-up | 2 |
| Overall Study | Other | 1 |
| Overall Study | Progressive Disease | 1 |
| Overall Study | Study Terminated by Sponsor | 49 |
| Overall Study | Withdrawal by Subject | 13 |
Baseline characteristics
| Characteristic | Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) |
|---|---|
| Age, Continuous | 65.6 years STANDARD_DEVIATION 8.58 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 2 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 76 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 4 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 3 Participants |
| Race (NIH/OMB) Black or African American | 13 Participants |
| Race (NIH/OMB) More than one race | 1 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 10 Participants |
| Race (NIH/OMB) White | 55 Participants |
| Region of Enrollment UNITED STATES | 82 Participants |
| Sex: Female, Male Female | 35 Participants |
| Sex: Female, Male Male | 47 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 16 / 82 |
| other Total, other adverse events | 81 / 82 |
| serious Total, serious adverse events | 42 / 82 |
Outcome results
Primary
Overall Response Rate (ORR)
ORR is defined as the percentage of participants who had partial response (PR) or better (sCR+CR+VGPR+PR) according to the International Myeloma Working Group (IMWG) response criteria, as assessed by the investigator. CR: Negative immunofixation on the serum and urine and disappearance of any soft tissue plasmacytomas and less than (\<) 5 percentage (%) plasma cells (PCs) in bone marrow; sCR: CR+Normal free light chain ratio and absence of clonal cells in bone marrow by immunohistochemistry or immuno fluorescence; PR: greater than equal to (\>=) 50% reduction of serum M-protein and reduction in 24-hour urinary M-protein by \>= 90 percentage (%) or to \<200 mg/24 hours; VGPR: Serum and urine M-protein detectable by immunofixation but not on electrophoresis or 90% or greater reduction in serum M-protein plus urine M-protein level \<100 mg per 24 hour.
Time frame: From Day 1 (post infusion) up to 18.6 months
Population: The all treated analysis set included all participants who received study intervention (cilta-cel OOS).
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Overall Response Rate (ORR) | 57.3 Percentage of participants |
Secondary
Clinical Benefit Rate (CBR)
Clinical benefit rate was defined as the percentage of participants who achieved a minimal response (MR) or better (including MR, PR, VGPR, CR, and sCR). MR was defined as per IMWG criteria as \>=25% but less than or equal to (\<=) 49% reduction of serum M-protein and reduction in 24-hour urine M-protein by 50% to 89%. In addition to the above criteria, if present at baseline, \>=50% reduction in the size of soft tissue plasmacytomas was also required.
Time frame: From Day 1 (post infusion) up to 18.6 months
Population: The all treated analysis set included all participants who received study intervention (cilta-cel OOS).
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Clinical Benefit Rate (CBR) | 59.8 Percentage of Participants |
Secondary
Complete Response (CR) Rate
CR rate is defined as percentage of participants who achieved best response of CR according to IMWG response criteria. CR is defined as per IMWG criteria as negative immunofixation of serum and urine, and disappearance of any soft tissue plasmacytomas, and \<5% PCs in bone marrow. No evidence of initial monoclonal protein isotype(s) on immunofixation of the serum and urine.
Time frame: From Day 1 (post infusion) up to 18.6 months
Population: The all treated analysis set included all participants who received study intervention (cilta-cel OOS).
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Complete Response (CR) Rate | 17.1 Percentage of Participants |
Secondary
Duration of Response (DOR)
DOR calculated in responders (with PR or better response) from date of initial documentation of response (PR or better) to date of first documented evidence of progressive disease (PD) as per IMWG criteria, or death due to any cause, whichever occurs first. PD: increase of 25% from lowest response value: serum and urine M-component (absolute increase must be \>=0.5 grams/deciliter \[g/dL\] and \>=200 mg/24 hours, respectively); only in participants without measurable serum and urine M-protein levels, difference between involved and uninvolved FLC levels (absolute increase must be \>10 mg/dL); only in participants without measurable serum and/or urine M-protein levels and without measurable disease by FLC levels, bone marrow PC % (absolute increase must be \>=10%), appearance of new lesion; definite development of new bone lesions or definite increase in the size of existing bone lesions, \>=50% increase in circulating PCs (minimum of 200 cells per uL) if this was the only measure of disease.
Time frame: From Day 1 (post infusion) up to 18.6 months
Population: Analysis population included responders (who achieved PR or better response) in all treated analysis set. The all treated analysis set included all participants who received study intervention (cilta-cel OOS). Here, 'N' (number of participants analyzed) signifies number of participants analyzed for this outcome measure.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Duration of Response (DOR) | NA Months |
Secondary
Minimal Residual Disease (MRD)-Negative Rate
MRD-negative rate is defined as the percentage of participants with negative MRD status (at 10\^-4, 10\^-5 and 10\^-6 cutoffs) by bone marrow aspirate.
Time frame: From Day 1 (post infusion) up to 18.6 months
Population: All participants who received cilta-cel OOS and had evaluable MRD samples in all treated analysis set. The all treated analysis set included all participants who received study intervention (cilta-cel OOS). Here, 'N' (number of participants analyzed) signifies number of participants analyzed for this outcome measure.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Minimal Residual Disease (MRD)-Negative Rate | 10^-4 cutoff | 66.7 Percentage of participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Minimal Residual Disease (MRD)-Negative Rate | 10^-5 cutoff | 66.7 Percentage of participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Minimal Residual Disease (MRD)-Negative Rate | 10^-6 cutoff | 53.3 Percentage of participants |
Secondary
Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion
Number of participants by worst grade abnormalities in clinical laboratory tests: hematology (including coagulation) and chemistry, after cilta-cel OOS infusion were reported. Grades were assessed using NCI-CTCAE (version 5.0), as: Grade 1: Mild; Grade 2: Moderate; Grade 3: Severe; Grade 4: Life-threatening. Participants with normal values or a value in the opposite direction (for laboratory tests with bidirectional toxicities defined) were assigned Grade 0. Only those categories in which at least 1 participant had data were reported.
Time frame: From Day 1 (post infusion) up to 18.6 months
Population: The all treated analysis set included all participants who received study intervention (cilta-cel OOS). Here, 'N' (number of participants analyzed) signifies number of participants analyzed for this outcome measure. Here, 'n' (number analyzed) signifies number of participants analyzed for each specified categories.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: Activated partial thromboplastin time prolonged: Grade 0 | 1 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: Activated partial thromboplastin time prolonged: Grade 2 | 1 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: Hemoglobin (Anemia): Grade 1 | 9 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: Hemoglobin (Anemia): Grade 2 | 33 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: Hemoglobin (Anemia): Grade 3 | 39 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: Lymphocyte count decreased: Grade 0 | 3 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: Lymphocyte count decreased: Grade 1 | 1 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: Lymphocyte count decreased: Grade 2 | 2 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: Lymphocyte count decreased: Grade 3 | 10 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: Lymphocyte count decreased: Grade 4 | 65 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: Neutrophil count decreased: Grade 0 | 1 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: Neutrophil count decreased: Grade 1 | 1 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: Neutrophil count decreased: Grade 2 | 4 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: Neutrophil count decreased: Grade 3 | 21 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: Neutrophil count decreased: Grade 4 | 54 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: Platelet count decreased: Grade 0 | 1 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: Platelet count decreased: Grade 1 | 16 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: Platelet count decreased: Grade 2 | 14 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: Platelet count decreased: Grade 3 | 15 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: Platelet count decreased: Grade 4 | 35 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: White blood cell decreased: Grade 2 | 4 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: White blood cell decreased: Grade 3 | 21 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Hematology: White blood cell decreased: Grade 4 | 56 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Alanine Aminotransferase increased: Grade 0 | 53 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Alanine Aminotransferase increased: Grade 1 | 20 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Alanine Aminotransferase increased: Grade 2 | 5 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Alanine Aminotransferase increased: Grade 3 | 3 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Aspartate aminotransferase increased: Grade 0 | 43 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Aspartate aminotransferase increased: Grade 1 | 28 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Aspartate aminotransferase increased: Grade 2 | 5 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Aspartate aminotransferase increased: Grade 3 | 4 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Aspartate aminotransferase increased: Grade 4 | 1 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Alkaline phosphatase high: Grade 0 | 58 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Alkaline phosphatase high: Grade 1 | 18 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Alkaline phosphatase high: Grade 2 | 4 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Alkaline phosphatase high: Grade 3 | 1 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Blood bilirubin increased: Grade 0 | 63 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Blood bilirubin increased: Grade 1 | 6 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Blood bilirubin increased: Grade 2 | 9 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Blood bilirubin increased: Grade 3 | 2 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Blood bilirubin increased: Grade 4 | 1 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Hypercalcemia: Grade 0 | 76 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Hypercalcemia: Grade 1 | 4 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Hypercalcemia: Grade 2 | 1 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Hypocalcemia: Grade 0 | 36 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Hypocalcemia: Grade 1 | 23 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Hypocalcemia: Grade 2 | 19 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Hypocalcemia: Grade 4 | 3 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Creatinine increased: Grade 0 | 53 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Creatinine increased: Grade 1 | 16 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Creatinine increased: Grade 2 | 7 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Creatinine increased: Grade 3 | 3 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants by Worst Grade Abnormalities in Clinical Laboratory Tests: Hematology (Including Coagulation) and Chemistry After Cilta-cel OOS Infusion | Biochemistry: Creatinine increased: Grade 4 | 2 Participants |
Secondary
Number of Participants With Presence of Replication Competent Lentivirus
Number of participants with presence of replication competent lentivirus (with evaluable sample) were reported.
Time frame: Predose, 3, 6, 12 months after cilta-cel infusion on Day 1
Population: The all treated analysis set included all participants who received study intervention (cilta-cel OOS). Here, 'N' (number of participants analyzed) signifies number of participants analyzed for this outcome measure. Here, 'n' (number analyzed) signifies number of participants analyzed at each specified categories.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Presence of Replication Competent Lentivirus | Predose of cilta-cel infusion | 0 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Presence of Replication Competent Lentivirus | 3 months after cilta-cel infusion | 0 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Presence of Replication Competent Lentivirus | 6 months after cilta-cel infusion | 0 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Presence of Replication Competent Lentivirus | 12 months after cilta-cel infusion | 0 Participants |
Secondary
Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Cytokine Release Syndrome (CRS), Immune Effector Cell-Associated Neurotoxicity (ICANS), Other Neurotoxicities, and Secondary Primary Malignancies
Number of participants with treatment-emergent AESIs: CRS, ICANS, other neurotoxicities, and secondary primary malignancies were reported. AESIs include the anticipated risks of treatment with study drug and events that may be related to multiple sclerosis comorbidities. Treatment-emergent AESIs were defined as any AESI that occurs on or after the start of cilta-cel OOS infusion through 100 days after the cilta-cel OOS infusion or the start of subsequent anti-myeloma therapy, whichever was earlier; or any AESI that was considered related to study intervention regardless of the start date of the event; or any AESI that was present at baseline but worsens in toxicity grade or was subsequently considered related to study intervention by the investigator.
Time frame: From Day 1 up to 100 days post cilta-cel OOS infusion (Up to 100 days)
Population: The all treated analysis set included all participants who received study intervention (cilta-cel OOS).
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Cytokine Release Syndrome (CRS), Immune Effector Cell-Associated Neurotoxicity (ICANS), Other Neurotoxicities, and Secondary Primary Malignancies | Cytokine Release Syndrome (CRS) | 61 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Cytokine Release Syndrome (CRS), Immune Effector Cell-Associated Neurotoxicity (ICANS), Other Neurotoxicities, and Secondary Primary Malignancies | Immune Effector Cell-Associated Neurotoxicity (ICANS) | 12 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Cytokine Release Syndrome (CRS), Immune Effector Cell-Associated Neurotoxicity (ICANS), Other Neurotoxicities, and Secondary Primary Malignancies | Other neurotoxicities | 8 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Cytokine Release Syndrome (CRS), Immune Effector Cell-Associated Neurotoxicity (ICANS), Other Neurotoxicities, and Secondary Primary Malignancies | Secondary primary malignancies | 5 Participants |
Secondary
Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Prolonged and Recurrent Cytopenias
Number of participants with treatment-emergent AESIs: prolonged and recurrent cytopenias were reported. AESIs include the anticipated risks of treatment with study drug and events that may be related to multiple sclerosis comorbidities. It included Thrombocytopenia, Neutropenia, Lymphopenia, and Anaemia. Treatment-emergent AESIs were defined as any AESI that occurs on or after the start of cilta-cel OOS infusion through 100 days after the cilta-cel OOS infusion or the start of subsequent anti-myeloma therapy, whichever was earlier; or any AESI that was considered related to study intervention regardless of the start date of the event; or any AESI that was present at baseline but worsens in toxicity grade or was subsequently considered related to study intervention by the investigator. Severity was assessed using NCI-CTCAE version 5.0 as: Grade 1: Mild; Grade 2: Moderate; Grade 3: Severe; Grade 4: Life-threatening; Grade 5: Death related to adverse event.
Time frame: From Day 1 up to 100 days post cilta-cel OOS infusion (Up to 100 days)
Population: The all treated analysis set included all participants who received study intervention (cilta-cel OOS).
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Prolonged and Recurrent Cytopenias | Thrombocytopenia: Grade 3/4 After Day 1 Dosing | 50 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Prolonged and Recurrent Cytopenias | Thrombocytopenia: Initial Grade 3/4 Not Recovered to <= Grade 2 by Day 30 | 42 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Prolonged and Recurrent Cytopenias | Thrombocytopenia: Initial Grade 3/4 Not Recovered to <= Grade 2 by Day 60 | 29 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Prolonged and Recurrent Cytopenias | Thrombocytopenia: Grade 3/4 > Day 60 (After Initial Recovery of Grade 3/4 within Day 60) | 1 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Prolonged and Recurrent Cytopenias | Neutropenia: Grade 3/4 After Day 1 Dosing | 75 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Prolonged and Recurrent Cytopenias | Neutropenia: Initial Grade 3/4 Not Recovered to <= Grade 2 by Day 30 | 36 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Prolonged and Recurrent Cytopenias | Neutropenia: Initial Grade 3/4 Not Recovered to <= Grade 2 by Day 60 | 18 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Prolonged and Recurrent Cytopenias | Neutropenia: Grade 3/4 > Day 60 (After Initial Recovery of Grade 3/4 within Day 60) | 1 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Prolonged and Recurrent Cytopenias | Lymphopenia: Grade 3/4 After Day 1 Dosing | 75 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Prolonged and Recurrent Cytopenias | Lymphopenia: Initial Grade 3/4 Not Recovered to <= Grade 2 by Day 30 | 32 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Prolonged and Recurrent Cytopenias | Lymphopenia: Initial Grade 3/4 Not Recovered to <= Grade 2 by Day 60 | 21 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Prolonged and Recurrent Cytopenias | Lymphopenia: Grade 3/4 > Day 60 (After Initial Recovery of Grade 3/4 within Day 60) | 4 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Prolonged and Recurrent Cytopenias | Anaemia: Grade 3/4 After Day 1 Dosing | 36 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Prolonged and Recurrent Cytopenias | Anaemia: Initial Grade 3/4 Not Recovered to <= Grade 2 by Day 30 | 12 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Prolonged and Recurrent Cytopenias | Anaemia: Initial Grade 3/4 Not Recovered to <= Grade 2 by Day 60 | 9 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Event of Special Interest (AESIs): Prolonged and Recurrent Cytopenias | Anaemia: Grade 3/4 > Day 60 (After Initial Recovery of Grade 3/4 within Day 60) | 2 Participants |
Secondary
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Number of participants with TEAEs were reported. An AE was any untoward medical occurrence in a participant participating in a clinical study that did not necessarily had a causal relationship with the pharmaceutical/biological agent under study. TEAEs are defined as any AE that occurred on or after the start of cilta-cel OOS infusion through 100 days after the cilta-cel OOS infusion or the start of subsequent anti-myeloma therapy, whichever was earlier; or any AE that was considered related to study intervention regardless of the start date of the event; or any AE that was present at baseline (Day 1, infusion of cilta-cel OOS) but worsens in toxicity grade or is subsequently considered related to study intervention by the investigator.
Time frame: From Day 1 up to 100 days post cilta-cel OOS infusion (Up to 100 days)
Population: The all treated analysis set included all participants who received study intervention (cilta-cel OOS).
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | 81 Participants |
Secondary
Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Severity
An AE was any untoward medical occurrence in a participant participating in a clinical study that did not necessarily had a causal relationship with the pharmaceutical/biological agent under study. TEAEs were defined as any AE that occurs on or after the start of cilta-cel OOS infusion through 100 days after the cilta-cel OOS infusion or the start of subsequent anti-myeloma therapy, whichever was earlier; or any AE that is considered related to study intervention regardless of the start date of the event; or any AE that was present at baseline (Day 1, pre-infusion of cilta-cel OOS) but worsens in toxicity grade or was subsequently considered related to study intervention by the investigator. Severity was assessed using National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0 as: Grade 1: Mild; Grade 2: Moderate; Grade 3: Severe; Grade 4: Life-threatening; Grade 5: Death related to adverse event.
Time frame: From Day 1 up to 100 days post cilta-cel OOS infusion (Up to 100 days)
Population: The all treated analysis set included all participants who received study intervention (cilta-cel OOS).
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Severity | Grade 1: Mild | 1 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Severity | Grade 2: Moderate | 6 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Severity | Grade 3: Severe | 14 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Severity | Grade 4: Life-threatening | 51 Participants |
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Severity | Grade 5: Death | 9 Participants |
Secondary
Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs)
Number of participants with serious TESAEs were reported. A SAE was defined as any untoward medical occurrence that results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, leads to a congenital anomaly/birth defect in the offspring of a participant, or was an important medical event. TESAEs were defined as any SAE that occurs on or after the start of cilta-cel OOS infusion through 100 days after the cilta-cel OOS infusion or the start of subsequent anti-myeloma therapy, whichever was earlier; or any SAE that was considered related to study intervention regardless of the start date of the event; or any SAE that was present at baseline but worsens in toxicity grade or was subsequently considered related to study intervention by the investigator.
Time frame: From Day 1 up to 100 days post cilta-cel OOS infusion (Up to 100 days)
Population: The all treated analysis set included all participants who received study intervention (cilta-cel OOS).
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs) | 42 Participants |
Secondary
Overall Survival (OS)
OS is measured from the date of the initial infusion of cilta-cel OOS to the date of the participant's death. If the participant is alive or the vital status is unknown, then the participant's data was censored at the date the participant was last known to be alive.
Time frame: From Day 1 (post infusion) up to 18.6 months
Population: The all treated analysis set included all participants who received study intervention (cilta-cel OOS).
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Overall Survival (OS) | NA Months |
Secondary
Partial Response (PR) Rate
PR rate was defined as percentage of participants who achieved PR according to IMWG response criteria. PR was defined as per IMWG criteria as \>= 50% reduction of serum M-protein and reduction in 24 hours urinary M-protein by \>= 90% or to \< 200 mg/24hours. If the serum and urine M-protein were unmeasurable, a \>= 50% decrease in the difference between involved and uninvolved Free light chain (FLC) levels was required in the place of the M-protein criteria. If serum and urine M-protein were not measurable, and serum free light assay was also not measurable, \>=50% reduction in bone marrow plasma cells is required in place of M-protein, provided baseline bone marrow plasma cells percentage was \>=30%. In addition to the above listed criteria, if present at baseline, a \>= 50% reduction in the size of soft tissue plasmacytomas was also required.
Time frame: From Day 1 (post infusion) up to 18.6 months
Population: The all treated analysis set included all participants who received study intervention (cilta-cel OOS).
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Partial Response (PR) Rate | 9.8 Percentage of Participants |
Secondary
Progression Free Survival (PFS)
PFS defined as the time from the date of the initial infusion of cilta-cel OOS to the date of first documented disease progression as per IMWG criteria, or death due to any cause, whichever occurs first. PD: increase of 25% from lowest response value: serum and urine M-component (absolute increase must be \>=0.5 g/dL and \>=200 mg/24 hours, respectively); only in participants without measurable serum and/or urine M-protein levels the difference between involved and uninvolved FLC levels (absolute increase must be \>10 mg/dL); only in participants without measurable serum and urine M-protein levels and without measurable disease by FLC levels, bone marrow PC percentage (absolute increase must be \>=10%), appearance of a new lesion; definite development of new bone lesions or definite increase in the size of existing bone lesions, \>=50% increase in circulating PCs (minimum of 200 cells per uL) if this was the only measure of disease.
Time frame: From Day 1 (post infusion) up to 18.6 months
Population: The all treated analysis set included all participants who received study intervention (cilta-cel OOS).
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Progression Free Survival (PFS) | NA Months |
Secondary
Stringent Complete Response (sCR) Rate
sCR rate is defined as percentage of participants who achieved sCR according to IMWG response criteria. sCR is defined per IMWG criteria as CR plus normal FLC ratio, and absence of clonal PCs by immunohistochemistry or 2- to 4-color flow cytometry. CR is defined as per IMWG as negative immunofixation of serum and urine, and disappearance of any soft tissue plasmacytomas, and \<5% PCs in bone marrow. No evidence of initial monoclonal protein isotype(s) on immunofixation of the serum and urine.
Time frame: From Day 1 (post infusion) up to 18.6 months
Population: The all treated analysis set included all participants who received study intervention (cilta-cel OOS).
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Stringent Complete Response (sCR) Rate | 15.9 Percentage of Participants |
Secondary
Very Good Partial Response (VGPR) Rate
VGPR rate is defined as percentage of participants who achieved best response of VGPR according to IMWG response criteria. As per IMWG response criteria, VPGR: serum and urine M-component detectable by immunofixation but not on electrophoresis, or \>=90% reduction in serum M-component plus urine M-component \<100 mg/24 hours.
Time frame: From Day 1 (post infusion) up to 18.6 months
Population: The all treated analysis set included all participants who received study intervention (cilta-cel OOS).
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Ciltacabtagene Autoleucel (Cilta-cel) Out-of-Specification (OOS) | Very Good Partial Response (VGPR) Rate | 14.6 Percentage of Participants |