A Phase 1/2 interventional study of JCARH125 and JCARH125 + anakinra in Multiple Myeloma, sponsored by Juno Therapeutics, a Subsidiary of Celgene. Completed at 20 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-05-24.
Sponsored by Juno Therapeutics, a Subsidiary of Celgene (part of Bristol-Myers Squibb) · Phase 1/2, Interventional, and Treatment
This is an open-label, multicenter, Phase 1/2 study to determine the safety and efficacy of JCARH125, a CAR T-cell product that targets B-cell maturation antigen (BCMA), in adult subjects with relapsed and/or refractory multiple myeloma. The study will include a Phase 1 part to determine the recommended dose of JCARH125 in subjects with relapsed and/or refractory multiple myeloma, followed by a Phase 2 part to further evaluate the safety and efficacy of JCARH125 at the recommended dose. The safety and tolerability of JCARH125 in subjects who receive prophylactic treatment with anakinra will be evaluated in a separate Phase 1 cohort. The antitumor activity of JCARH125 in subjects who have been previously treated with BCMA-directed therapy will be evaluated in separate Phase 2a cohorts.
3,712 studies on the registry are indexed under Multiple Myeloma; 752 are open to participants now.
This study's enrollment of 165 is above the median of 41 across 2,965 interventional studies indexed under Multiple Myeloma.
Browse Multiple Myeloma studies →Juno Therapeutics, a Subsidiary of Celgene is the lead sponsor of 12 studies on the registry; 1 is open to participants now.
Of its 7 completed or terminated interventional studies of FDA-regulated products, 3 (43%) have results posted.
First submitted before Juno Therapeutics, Inc. became part of Bristol-Myers Squibb.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Diagnosis of multiple myeloma (MM) with relapsed and/or refractory (R/R) disease. Participants must have received at least 3 prior anti-myeloma treatment regimens. Participants must have previously received all of the following therapies and must be refractory to the last line of therapy prior to entering the study (not applicable to Phase 2a):
Subjects who have received prior allogeneic stem cell transplant or donor lymphocyte infusion at least 100 days before enrollment with no signs of acute or chronic graft-versus-host disease (GVHD) will be considered eligible. Subjects who were not candidates to receive one or more of the above treatments (ie, contraindicated) are eligible.
Phase 2a cohorts only - Subjects with R/R MM who have been previously treated with prior BCMA-directed anti-myeloma therapy, achieved at least a partial response (PR) and progressed on the following treatment:
Exclusion Criteria:
Subjects will receive a course of lymphodepleting chemotherapy with fludarabine and cyclophosphamide followed by a single dose of JCARH125
Biological: JCARH125
Subjects will receive a course of lymphodepleting chemotherapy with fludarabine and cyclophosphamide followed by prophylactic treatment with anakinra and a single dose of JCARH125
Biological: JCARH125 + anakinra
Participants will undergo leukapheresis to isolate peripheral blood mononuclear cells (PBMCs) for the production of JCARH125. During JCARH125 production, participants may receive bridging chemotherapy for disease control. Following successful generation of JCARH125 product, participants will receive a course of lymphodepleting chemotherapy followed by one dose of JCARH125 administered intravenously (IV).
Drug page: Interleukin 1 Receptor Antagonist Protein
Participants will undergo leukapheresis to isolate PBMCs for the production of JCARH125. During JCARH125 production, participants may receive bridging chemotherapy for disease control. Following successful generation of JCARH125 product, participants will receive a course of lymphodepleting chemotherapy followed by two doses of anakinra administered subcutaneously and one dose of JCARH125 administered IV. Subjects receive anakinra for 5 consecutive days following JCARH125 infusion.
Number of Participants With Dose-Limiting Toxicity (DLT) in Phase 1
DLT is defined as adverse events (AEs) that occur within 21 days following JCARH125 infusion and meet any of the following criteria: * Treatment-emergent Grade ≥3 allergic reactions related to JCARH125; * Treatment-emergent Grade 3 seizures, regardless of attribution, that do not resolve to Grade ≤2 within 3 days in participants who have no evidence of central nervous system (CNS) involvement of Multiple Myeloma or other CNS pathology; * Treatment-emergent autoimmune toxicity Grade ≥3, regardless of attribution (excluding B-cell aplasia); * Treatment-emergent Grade 3 CRS that does not resolve to Grade ≤2 within 72 hours; * Any other treatment-emergent Grade 3 AE related to JCARH125 that does not resolve to Grade ≤2 within 7 days; * Any treatment-emergent Grade 4 AE related to JCARH125 that does not resolve to Grade ≤2 within 7 days; * Treatment-emergent Grade 4 Cytokine Release Syndrome of any duration; * Any treatment-emergent Grade 5 toxicity not due to the underlying malignancy
Time frame: From day 1 to day 22 following JCARH125 infusion (Up to 21 days)
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Severity in Phase 1 and Phase 1 Anakinra
TEAE is defined as an AE that starts any time from initiation of JCARH125 administration through and including 90 days following the JCARH125 infusion graded using the NCI Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. Any AE occurring after the initiation of another anticancer treatment will not be considered a TEAE. Grade 3=Severe; Grade 4=Life-threatening; and Grade 5=death.
Time frame: From the time of JCARH125 infusion to 90 days following the infusion (Up to 90 days)
Number of Participants With Clinically Significant Laboratory Abnormalities by Severity in Phase 1 and Phase 1 Anakinra
Clinically significant laboratory abnormalities are assessed by investigator and are reported as treatment-emergent adverse event (TEAE). TEAE is defined as an AE that starts any time from initiation of JCARH125 administration through and including 90 days following the JCARH125 infusion graded by Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. Any AE occurring after the initiation of another anticancer treatment is not considered a TEAE. Grade 3=Severe; Grade 4=Life-threatening.
Time frame: From the time of JCARH125 infusion to 90 days following the infusion (Up to 90 days)
Number of Participants Receiving Prophylactic Anakinra With Grade ≥2 Cytokine Release Syndrome (CRS) in Phase 1 and Phase 1 Anakinra
Number of participants receiving prophylactic anakinra with grade ≥ 2 CRS relative to the number of participants treated at the recommended Phase 2 dose (RP2D) in the Phase 1 dose escalation portion of the trial with grade ≥ 2 CRS. CRS grade is defined by the most severe symptom (excluding fever). Grade 2=moderate; Grade 3=severe; Grade 4=life-threatening
Time frame: From first infusion to up to aproximately 61 months
Time to Onset of Grade ≥2 Cytokine Release Syndrome (CRS) in Phase 1 and Phase 1 Anakinra
Time to first onset of Grade ≥2 CRS in participants receiving prophylactic anakinra relative to onset of Grade ≥ 2 CRS in participants treated at the RP2D(s) in the Phase 1 dose escalation portion of the trial. Time to onset is calculated from the latest JCARH125 infusion prior to the first onset of Grade \>= 2 CRS. CRS grade is defined by the most severe symptom (excluding fever). Grade 2=moderate; Grade 3=severe; Grade 4=life-threatening
Time frame: From JCARH125 infusion to the first onset of Grade ≥2 CRS (Up to approximately 61 months)
Number of Participants Receiving Prophylactic Anakinra With no Cytokine Release Syndrome (CRS) Occurring on Days 1-3 in Phase 1 Anakinra
The number of participants receiving prophylactic anakinra with no CRS occurring on study days 1, 2, or 3. CRS grade is defined by the most severe symptom (excluding fever). Grade 2=moderate; Grade 3=severe; Grade 4=life-threatening
Time frame: Day 1, 2, 3
Overall Response Rate (ORR) in Phase 2 and Phase 2a
ORR is defined as stringent complete response (sCR), complete response (CR), very good partial response (VGPR), or partial response (PR), according to IMWG criteria. Participants without any reported disease response assessments will be considered non-responders. sCR=complete response plus normal free light chain ratio and absence of clonal cells in bone marrow biopsy by immunohistochemistry; CR=negative immunofixation of serum and urine and disappearance of any soft tissue plasmacytomas and \< 5% plasma cells in bone marrow aspirates. When the only method to measure disease is by serum FLC levels, CR can be defined as a normal FLC ratio of 0.26 to 1.65; VGPR=serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥ 90% reduction in serum M-protein level plus urine M-protein level \< 100 mg/24 h; PR=≥ 50% reduction of serum M protein plus reduction in 24-hour urinary M-protein by ≥ 90% or to \< 200 mg/24 h
Time frame: From the time of the JCARH125 infusion until disease progression, end of study, or the start of another anticancer therapy or stem cell transplant (Up to aproximately 61 months)
Maximum Observed Concentration (Cmax)
Cmax is the maximal concentration of JCARH125 CAR T cells in the blood after its infusion as determined by quantitative polymerase chain reaction (qPCR) to detect the JCARH125 transgene.
Time frame: From JCARH125 infusion through the day 29 visit
Time to Maximum Observed Concentration (Tmax)
Tmax is the first study day the maximum observed concetration (Cmax) of JCARH125 CAR T cells in the blood is reached as determined by quantitative polymerase chain reaction (qPCR) to detect the JCARH125 transgene.
Time frame: From JCARH125 infusion through the day 29 visit
Area Under the Concertation-Time Curve (AUC) From Time Day 1 to Day 29 [AUC (0-28 Days)]
AUC (0-28) of JCARH125 CAR T cells in the blood as determined by quantitative polymerase chain reaction (qPCR) to detect the JCARH125 transgene.
Time frame: From JCARH125 infusion through 28 days after the infusion
Number of Participants With Pharmacokinetics Persistence
Pharmacokinetics persistence of JCARH125 CAR T cells in the blood as determined by quantitative polymerase chain reaction (qPCR) over time to detect the JCARH125 transgene. Persistence is defined as a transgene count greater than or equal to the lower limit of detection (LLOD).
Time frame: Day 29, 60, 90, 180, 270, 365, 545, 730
Overall Response Rate (ORR) in Phase 1 and Phase 1 Anakinra
ORR is defined as stringent complete response (sCR), complete response (CR), very good partial response (VGPR), or partial response (PR), according to IMWG criteria. Participants without any reported disease response assessments will be considered non-responders. sCR=complete response plus normal free light chain ratio and absence of clonal cells in bone marrow biopsy by immunohistochemistry; CR=negative immunofixation of serum and urine and disappearance of any soft tissue plasmacytomas and \< 5% plasma cells in bone marrow aspirates. When the only method to measure disease is by serum FLC levels, CR can be defined as a normal FLC ratio of 0.26 to 1.65; VGPR=serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥ 90% reduction in serum M-protein level plus urine M-protein level \< 100 mg/24 h; PR=≥ 50% reduction of serum M protein plus reduction in 24-hour urinary M-protein by ≥ 90% or to \< 200 mg/24 h
Time frame: From the time of the JCARH125 infusion until disease progression, end of study, or the start of another anticancer therapy or stem cell transplant (Up to 61 approximately months)
Complete Response Rate (CRR)
CRR is defined as stringent complete response (sCR) or complete response (CR), according to IMWG criteria. Participants without any reported disease response assessments will be considered non-responders. sCR=complete response plus normal free light chain ratio and absence of clonal cells in bone marrow biopsy by immunohistochemistry; CR=negative immunofixation of serum and urine and disappearance of any soft tissue plasmacytomas and \< 5% plasma cells in bone marrow aspirates
Time frame: From the time of the JCARH125 infusion until disease progression, end of study, or the start of another anticancer therapy or stem cell transplant (Up to aproximately 61 months)
Duration of Response (DoR) in Phase 2 and 2a
DoR is defined as the time from first response (stringent complete response (sCR), complete response (CR), very good partial response (VGPR), or partial response (PR)) to the earlier date of progressive disease or death due to any cause. sCR=complete response plus normal free light chain ratio and absence of clonal cells in bone marrow biopsy by immunohistochemistry; CR=negative immunofixation of serum and urine and disappearance of any soft tissue plasmacytomas and \< 5% plasma cells in bone marrow aspirates; VGPR=serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥ 90% reduction in serum M-protein level plus urine M-protein level \< 100 mg/24 h; PR=≥ 50% reduction of serum M protein plus reduction in 24-hour urinary M-protein by ≥ 90% or to \< 200 mg/24 h. Progressive disease is defined as (1) Increase of ≥25% from lowest confirmed response; (2) Appearance of a new lesion(s); (3) ≥50% increase in circulating plasma cells.
Time frame: From first response to the date of progression or death due to any cause, whichever occurs first (Up to approixmately 61 months)
Duration of Complete Response (DoCR) in Phase 2 and 2a
DoCR is defined as the time from first response (stringent complete response (sCR), complete response (CR)) to the earlier date of progressive disease or death due to any cause. sCR=complete response plus normal free light chain ratio and absence of clonal cells in bone marrow biopsy by immunohistochemistry; CR=negative immunofixation of serum and urine and disappearance of any soft tissue plasmacytomas and \< 5% plasma cells in bone marrow aspirates. Progressive disease is defined as (1) Increase of ≥25% from lowest confirmed response; (2) Appearance of a new lesion(s); (3) ≥50% increase in circulating plasma cells.
Time frame: From first response to the date of progression or death due to any cause, whichever occurs first (Up to approixmately 61 months)
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Severity in Phase 2 and Phase 2a
TEAE is defined as an AE that starts any time from initiation of JCARH125 administration through and including 90 days following the JCARH125 infusion graded using the NCI Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. Any AE occurring after the initiation of another anticancer treatment will not be considered a TEAE. Grade 3=Severe; Grade 4=Life-threatening; and Grade 5=death.
Time frame: From the time of JCARH125 infusion to 90 days following the infusion (Up to 90 days)
Number of Participants With Clinically Significant Laboratory Abnormalities by Severity in Phase 2 and Phase 2a
Clinically significant laboratory abnormalities are assessed by investigator and are reported as treatment-emergent adverse event (TEAE). TEAE is defined as an AE that starts any time from initiation of JCARH125 administration through and including 90 days following the JCARH125 infusion graded by Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. Any AE occurring after the initiation of another anticancer treatment is not considered a TEAE. Grade 3=Severe; Grade 4=Life-threatening.
Time frame: From the time of JCARH125 infusion to 90 days following the infusion (Up to 90 days)
Overall Survival (OS) in Phase 2 and Phase 2a
OS is defined as the time from the JCARH125 infusion until death due to any cause.
Time frame: Form date of first infusion to the date of death due to any reason (Up to apprixamtely 61 months)
Progression Free Survival (PFS) in Phase 2 and Phase 2a
PFS is defined as the time from JCARH125 infusion until the earliest date of disease progression or death from any cause. Progressive disease (PD) is defined as (1) Increase of ≥25% from lowest confirmed response in one or more of the following: Serum M-protein absolute increase ≥0.5 g/dL; Serum M-protein increase ≥1 g/dL, if the lowest M component was ≥5 g/dL; Urine M protein absolute increase ≥200 mg/24 h; the difference between involved and uninvolved FLC levels absolute increase \>10 mg/dL; Bone marrow plasma-cell percentage irrespective of baseline status absolute increase ≥10%. (2) Appearance of a new lesion(s), ≥50% increase from nadir in SPDd of \>1 lesion, or ≥50% increase in the longest diameter of a previous lesion \>1 cm in short axis. (3) ≥50% increase in circulating plasma cells (minimum of 200 cells μL) if this is the only measure of disease.
Time frame: Form date of first infusion to the date of disease progression, or death, due to any reason (Up to approximately 61 months)
Time to Response (TTR) in Phase 2 and Phase 2a
TTR is defined from JCARH125 infusion to the first document of stringent complete response (sCR), complete response (CR), very good partial response (VGPR), or partial response (PR). sCR=complete response plus normal free light chain ratio and absence of clonal cells in bone marrow biopsy by immunohistochemistry; CR=negative immunofixation of serum and urine and disappearance of any soft tissue plasmacytomas and \< 5% plasma cells in bone marrow aspirates; VGPR=serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥ 90% reduction in serum M-protein level plus urine M-protein level \< 100 mg/24 h; PR=≥ 50% reduction of serum M protein plus reduction in 24-hour urinary M-protein by ≥ 90% or to \< 200 mg/24 h. Progressive disease is defined as (1) Increase of ≥25% from lowest confirmed response; (2) Appearance of a new lesion(s); (3) ≥50% increase in circulating plasma cells.
Time frame: Form date of first infusion to the date of disease progression, or death, due to any reason (Up to approximately 61 months)
Time to Complete Response (TTCR) in Phase 2 and Phase 2a
TTCR is defined from JCARH125 infusion to the first document of stringent complete response (sCR) or complete response (CR). sCR=complete response plus normal free light chain ratio and absence of clonal cells in bone marrow biopsy by immunohistochemistry; CR=negative immunofixation of serum and urine and disappearance of any soft tissue plasmacytomas and \< 5% plasma cells in bone marrow aspirates. Progressive disease is defined as (1) Increase of ≥25% from lowest confirmed response; (2) Appearance of a new lesion(s); (3) ≥50% increase in circulating plasma cells.
Time frame: Form date of first infusion to the date of disease progression, or death, due to any reason (Up to approximately 61 months)
Change From Baseline in the Total Score of European Organization for Research and Treatment of Cancer Quality-of-Life Questionnaire (EORTC QLQ-C30) in Phase 2
The EORTC QLQ-C30 is a 30-item scale composed of both multi-item scales and single-item measures such as functional scales (physical, role, cognitive, emotional, and social), global health status, symptom scales (fatigue, pain, nausea/vomiting), and other (dyspnoea, appetite loss, insomnia, constipation/diarrhea, and financial difficulties). Most questions used 4-point scale (1 'Not at All' to 4 'Very Much'); 2 questions used 7-point scale (1 'Very Poor' to 7 'Excellent'). Subscale scores are transformed to 0 to 100 scale, with higher scores on functional scales indicating better function and higher score on symptom scales indicating worse symptoms. Baseline is defined as the last non-missing measurement prior to JCARH125 infusion.
Time frame: Baseline and visit 24 month
Change From Baseline in the Total Score of European Organization for Research and Treatment of Cancer Quality-of-Life Questionnaire (QLQ-MY20) in Phase 2
QLQ-MY20 includes 20 questions across four scales: disease symptoms, treatment side-effects, future perspective, and body image. Questions used 4-point scale (1 'Not at All' to 4 'Very Much'). Scores are averaged, and transformed to 0-100 scale; higher score for the disease symptoms scale = higher level of symptomatology.
Time frame: Baseline and visit 24 month
Change From Baseline (EQ-5D-5L) Index Score in Phase 2
EQ-5D-5L is a standardized measure of health status that consists of descriptive system and Visual Analogue scale VAS). The descriptive system comprises dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression). Each dimension has 5 levels (no problems, slight problems, moderate problems, severe problems, extreme problems). Responses are coded so that a '1' indicates no problem, and '5' indicates the most serious problem. The responses for the dimensions are combined in a 5-digit number corresponding to response categories for successive dimensions using a scoring algorithm. The VAS system has endpoints labeled "the best health you can imagine" and "the worst health you can imagine." The scale is numbered from 0 to 100 with 0 corresponding to the worst imaginable health state and 100 corresponding to the best imaginable health state. A high score represents a better level of quality of life
Time frame: Baseline and visit 24 month
Duration of Hospitalization From JCARH125 Administration in Phase 2
Total length of all intensive care unit (ICU) and non-ICU stays from JCARH125 Administration. ICU inpatient and non-ICU inpatient are not exclusive. Total length includes participants who had multiple hospitalization stays.
Time frame: From JCARH125 infusion to up to approximately 61 months
Reasons for Hospitalization From JCARH125 Administration in Phase 2
Number of participants with reasons for hospitalization from JCARH125 administration
Time frame: From JCARH125 infusion to up to approximately 61 months
| Milestone | Phase 1 50 x 10^6 Cells | Phase 1 150 x 10^6 Cells | Phase 1 300 x 10^6 Cells | Phase 1 450 x 10^6 Cells | Phase 1 600 x 10^6 Cells | Phase 1 Anakinra 600 x 10^6 Cells | Phase 2 600 x 10^6 Cells | Phase 2a 600 x 10^6 Cells Anti-BCMA |
|---|---|---|---|---|---|---|---|---|
| Started | 15 | 30 | 27 | 22 | 22 | 14 | 25 | 10 |
| Received lymphodepleting chemotherapy (ldc) | 15 | 30 | 26 | 21 | 20 | 14 | 24 | 10 |
| Completed | 14 | 30 | 26 | 21 | 20 | 14 | 24 | 10 |
| Not completed | 1 | 0 | 1 | 1 | 2 | 0 | 1 | 0 |
| Withdrew: Death | 1 | 0 | 0 | 0 | 1 | 0 | 1 | 0 |
| Withdrew: Screen fail | 0 | 0 | 0 | 0 | 1 | 0 | 0 | 0 |
| Withdrew: No jcarh125 - disease related complications | 0 | 0 | 1 | 0 | 0 | 0 | 0 | 0 |
| Withdrew: Participant not eligible - other reasons | 0 | 0 | 0 | 1 | 0 | 0 | 0 | 0 |
| Milestone | Phase 1 50 x 10^6 Cells | Phase 1 150 x 10^6 Cells | Phase 1 300 x 10^6 Cells | Phase 1 450 x 10^6 Cells | Phase 1 600 x 10^6 Cells | Phase 1 Anakinra 600 x 10^6 Cells | Phase 2 600 x 10^6 Cells | Phase 2a 600 x 10^6 Cells Anti-BCMA |
|---|---|---|---|---|---|---|---|---|
| Started | 14 | 30 | 26 | 21 | 20 | 14 | 24 | 10 |
| Completed | 8 | 9 | 7 | 6 | 9 | 6 | 11 | 3 |
| Not completed | 6 | 21 | 19 | 15 | 11 | 8 | 13 | 7 |
| Withdrew: Withdrawal by subject | 1 | 1 | 5 | 5 | 3 | 4 | 5 | 3 |
| Withdrew: Lost to follow-up | 0 | 1 | 0 | 0 | 1 | 0 | 1 | 0 |
| Withdrew: Death | 4 | 19 | 10 | 8 | 5 | 3 | 4 | 2 |
| Withdrew: Other reasons | 1 | 0 | 4 | 2 | 2 | 1 | 3 | 2 |
DLT is defined as adverse events (AEs) that occur within 21 days following JCARH125 infusion and meet any of the following criteria: * Treatment-emergent Grade ≥3 allergic reactions related to JCARH125; * Treatment-emergent Grade 3 seizures, regardless of attribution, that do not resolve to Grade ≤2 within 3 days in participants who have no evidence of central nervous system (CNS) involvement of Multiple Myeloma or other CNS pathology; * Treatment-emergent autoimmune toxicity Grade ≥3, regardless of attribution (excluding B-cell aplasia); * Treatment-emergent Grade 3 CRS that does not resolve to Grade ≤2 within 72 hours; * Any other treatment-emergent Grade 3 AE related to JCARH125 that does not resolve to Grade ≤2 within 7 days; * Any treatment-emergent Grade 4 AE related to JCARH125 that does not resolve to Grade ≤2 within 7 days; * Treatment-emergent Grade 4 Cytokine Release Syndrome of any duration; * Any treatment-emergent Grade 5 toxicity not due to the underlying malignancy
| Participants | Phase 1 50 x 10^6 Cells | Phase 1 150 x 10^6 Cells | Phase 1 300 x 10^6 Cells | Phase 1 450 x 10^6 Cells | Phase 1 600 x 10^6 Cells |
|---|---|---|---|---|---|
| Number of Participants With Dose-Limiting Toxicity (DLT) in Phase 1 | 0 | 1 | 1 | 3 | 1 |
TEAE is defined as an AE that starts any time from initiation of JCARH125 administration through and including 90 days following the JCARH125 infusion graded using the NCI Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. Any AE occurring after the initiation of another anticancer treatment will not be considered a TEAE. Grade 3=Severe; Grade 4=Life-threatening; and Grade 5=death.
| Participants | Phase 1 50 x 10^6 Cells | Phase 1 150 x 10^6 Cells | Phase 1 300 x 10^6 Cells | Phase 1 450 x 10^6 Cells | Phase 1 600 x 10^6 Cells | Phase 1 Anakinra 600 x 10^6 Cells |
|---|---|---|---|---|---|---|
| TEAE | 14 | 30 | 26 | 21 | 20 | 14 |
| Grade 3-5 TEAE | 14 | 30 | 25 | 21 | 20 | 13 |
| Grade 5 TEAE | 0 | 1 | 1 | 2 | 0 | 0 |
| JCARH125-Related TEAE | 12 | 26 | 25 | 21 | 20 | 14 |
| JCARH125-Related Grade 3-5 TEAE | 5 | 10 | 15 | 16 | 16 | 7 |
| JCARH125-Related Grade 5 TEAE | 0 | 0 | 0 | 1 | 0 | 0 |
Clinically significant laboratory abnormalities are assessed by investigator and are reported as treatment-emergent adverse event (TEAE). TEAE is defined as an AE that starts any time from initiation of JCARH125 administration through and including 90 days following the JCARH125 infusion graded by Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. Any AE occurring after the initiation of another anticancer treatment is not considered a TEAE. Grade 3=Severe; Grade 4=Life-threatening.
| Participants | Phase 1 50 x 10^6 Cells | Phase 1 150 x 10^6 Cells | Phase 1 300 x 10^6 Cells | Phase 1 450 x 10^6 Cells | Phase 1 600 x 10^6 Cells | Phase 1 Anakinra 600 x 10^6 Cells |
|---|---|---|---|---|---|---|
| Neutropenia grade 3 | 4 | 1 | 2 | 1 | 3 | 1 |
| Neutropenia grade 4 | 10 | 28 | 22 | 20 | 17 | 10 |
| Anemia grade 3 | 9 | 19 | 16 | 11 | 13 | 10 |
| Thrombocytopenia grade 3 | 0 | 4 | 3 | 1 | 0 | 4 |
| Thrombocytopenia grade 4 | 4 | 15 | 11 | 12 | 11 | 5 |
| Leukopenia grade 3 | 0 | 0 | 2 | 1 | 3 | 0 |
| Leukopenia grade 4 | 4 | 8 | 12 | 7 | 3 | 4 |
| Lymphopenia grade 3 | 1 | 2 | 4 | 3 | 1 | 1 |
| Lymphopenia grade 4 | 0 | 1 | 2 | 0 | 1 | 0 |
| Febrile neutropenia grade 3 | 1 | 7 | 2 | 0 | 0 | 0 |
| Febrile neutropenia grade 4 | 0 | 0 | 0 | 1 | 0 | 0 |
| Haemolysis grade 3 | 0 | 0 | 0 | 0 | 1 | 0 |
| Hypofibrinogenaemia grade 3 | 0 | 0 | 1 | 0 | 0 | 0 |
| Hypophosphataemia grade 3 | 1 | 5 | 2 | 6 | 6 | 6 |
| Hypophosphataemia grade 4 | 0 | 1 | 0 | 0 | 0 | 0 |
| Hypocalcaemia grade 3 | 0 | 2 | 0 | 1 | 2 | 1 |
| Hypocalcaemia grade 4 | 0 | 0 | 0 | 1 | 0 | 0 |
| Hypokalaemia grade 3 | 0 | 3 | 0 | 0 | 1 | 0 |
| Hyponatraemia grade 3 | 1 | 1 | 2 | 1 | 0 | 2 |
| Hypomagnesaemia grade 3 | 0 | 1 | 0 | 0 | 0 | 0 |
| Hyperglycaemia grade 3 | 0 | 0 | 0 | 2 | 0 | 0 |
| Hyperglycaemia grade 4 | 0 | 0 | 0 | 0 | 0 | 1 |
| Hypoalbuminaemia grade 3 | 0 | 1 | 0 | 0 | 1 | 1 |
| Hypertriglyceridaemia grade 3 | 0 | 1 | 0 | 0 | 0 | 0 |
| Hypertriglyceridaemia grade 4 | 0 | 1 | 0 | 1 | 0 | 1 |
| Alanine aminotransferase increased grade 3 | 1 | 0 | 0 | 2 | 1 | 1 |
| Aspartate aminotransferase increased grade 3 | 0 | 2 | 0 | 0 | 0 | 1 |
| Aspartate aminotransferase increased grade 4 | 1 | 1 | 0 | 2 | 0 | 0 |
| Blood alkaline phosphatase increased grade 3 | 0 | 0 | 0 | 1 | 0 | 0 |
| Blood bilirubin increased grade 3 | 0 | 0 | 0 | 0 | 0 | 1 |
| Serum ferritin increased grade 3 | 0 | 1 | 1 | 0 | 0 | 0 |
Number of participants receiving prophylactic anakinra with grade ≥ 2 CRS relative to the number of participants treated at the recommended Phase 2 dose (RP2D) in the Phase 1 dose escalation portion of the trial with grade ≥ 2 CRS. CRS grade is defined by the most severe symptom (excluding fever). Grade 2=moderate; Grade 3=severe; Grade 4=life-threatening
| Participants | Phase 1 600 x 10^6 Cells | Phase 1 Anakinra 600 x 10^6 Cells |
|---|---|---|
| Number of Participants Receiving Prophylactic Anakinra With Grade ≥2 Cytokine Release Syndrome (CRS) in Phase 1 and Phase 1 Anakinra | 11 | 4 |
Time to first onset of Grade ≥2 CRS in participants receiving prophylactic anakinra relative to onset of Grade ≥ 2 CRS in participants treated at the RP2D(s) in the Phase 1 dose escalation portion of the trial. Time to onset is calculated from the latest JCARH125 infusion prior to the first onset of Grade \>= 2 CRS. CRS grade is defined by the most severe symptom (excluding fever). Grade 2=moderate; Grade 3=severe; Grade 4=life-threatening
| Days | Phase 1 600 x 10^6 Cells | Phase 1 Anakinra 600 x 10^6 Cells |
|---|---|---|
| Time to Onset of Grade ≥2 Cytokine Release Syndrome (CRS) in Phase 1 and Phase 1 Anakinra | 2.0 (1 to 3) | 2.0 (2 to 3) |
The number of participants receiving prophylactic anakinra with no CRS occurring on study days 1, 2, or 3. CRS grade is defined by the most severe symptom (excluding fever). Grade 2=moderate; Grade 3=severe; Grade 4=life-threatening
| Participants | Phase 1 Anakinra 600 x 10^6 Cells |
|---|---|
| Number of Participants Receiving Prophylactic Anakinra With no Cytokine Release Syndrome (CRS) Occurring on Days 1-3 in Phase 1 Anakinra | 2 |
ORR is defined as stringent complete response (sCR), complete response (CR), very good partial response (VGPR), or partial response (PR), according to IMWG criteria. Participants without any reported disease response assessments will be considered non-responders. sCR=complete response plus normal free light chain ratio and absence of clonal cells in bone marrow biopsy by immunohistochemistry; CR=negative immunofixation of serum and urine and disappearance of any soft tissue plasmacytomas and \< 5% plasma cells in bone marrow aspirates. When the only method to measure disease is by serum FLC levels, CR can be defined as a normal FLC ratio of 0.26 to 1.65; VGPR=serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥ 90% reduction in serum M-protein level plus urine M-protein level \< 100 mg/24 h; PR=≥ 50% reduction of serum M protein plus reduction in 24-hour urinary M-protein by ≥ 90% or to \< 200 mg/24 h
| Percent of participants | Phase 2 600 x 10^6 Cells | Phase 2a 600 x 10^6 Cells Anti-BCMA |
|---|---|---|
| Overall Response Rate (ORR) in Phase 2 and Phase 2a | 91.7 (73.0 to 99.0) | 100.0 (69.2 to 100.0) |
Cmax is the maximal concentration of JCARH125 CAR T cells in the blood after its infusion as determined by quantitative polymerase chain reaction (qPCR) to detect the JCARH125 transgene.
| Copies/UG | Phase 1 50 x 10^6 Cells | Phase 1 150 x 10^6 Cells | Phase 1 300 x 10^6 Cells | Phase 1 450 x 10^6 Cells | Phase 1 600 x 10^6 Cells | Phase 1 Anakinra 600 x 10^6 Cells | Phase 2 600 x 10^6 Cells | Phase 2a 600 x 10^6 Cells Anti-BCMA |
|---|---|---|---|---|---|---|---|---|
| Maximum Observed Concentration (Cmax) | 61037.57 ± 365.26 | 64705.82 ± 305.31 | 123460.33 ± 156.23 | 122228.99 ± 174.22 | 162706.00 ± 83.19 | 176233.64 ± 99.44 | 140331.08 ± 234.07 | 103009.98 ± 128.25 |
Tmax is the first study day the maximum observed concetration (Cmax) of JCARH125 CAR T cells in the blood is reached as determined by quantitative polymerase chain reaction (qPCR) to detect the JCARH125 transgene.
| Day | Phase 1 50 x 10^6 Cells | Phase 1 150 x 10^6 Cells | Phase 1 300 x 10^6 Cells | Phase 1 450 x 10^6 Cells | Phase 1 600 x 10^6 Cells | Phase 1 Anakinra 600 x 10^6 Cells | Phase 2 600 x 10^6 Cells | Phase 2a 600 x 10^6 Cells Anti-BCMA |
|---|---|---|---|---|---|---|---|---|
| Time to Maximum Observed Concentration (Tmax) | 13.50 (7.0 to 28.0) | 11.00 (4.0 to 22.0) | 10.00 (3.0 to 21.0) | 10.00 (7.0 to 25.0) | 10.00 (3.0 to 23.0) | 13.50 (7.0 to 28.0) | 9.50 (4.0 to 22.0) | 14.50 (7.0 to 21.0) |
AUC (0-28) of JCARH125 CAR T cells in the blood as determined by quantitative polymerase chain reaction (qPCR) to detect the JCARH125 transgene.
| Day*copies/UG | Phase 1 50 x 10^6 Cells | Phase 1 150 x 10^6 Cells | Phase 1 300 x 10^6 Cells | Phase 1 450 x 10^6 Cells | Phase 1 600 x 10^6 Cells | Phase 1 Anakinra 600 x 10^6 Cells | Phase 2 600 x 10^6 Cells | Phase 2a 600 x 10^6 Cells Anti-BCMA |
|---|---|---|---|---|---|---|---|---|
| Area Under the Concertation-Time Curve (AUC) From Time Day 1 to Day 29 [AUC (0-28 Days)] | 556080.86 ± 288.83 | 610142.64 ± 276.50 | 1307054.13 ± 159.86 | 1390556.32 ± 188.93 | 1956613.78 ± 80.39 | 2052280.38 ± 104.12 | 1427277.29 ± 226.53 | 1287032.03 ± 145.22 |
Pharmacokinetics persistence of JCARH125 CAR T cells in the blood as determined by quantitative polymerase chain reaction (qPCR) over time to detect the JCARH125 transgene. Persistence is defined as a transgene count greater than or equal to the lower limit of detection (LLOD).
| Participants | Phase 1 50 x 10^6 Cells | Phase 1 150 x 10^6 Cells | Phase 1 300 x 10^6 Cells | Phase 1 450 x 10^6 Cells | Phase 1 600 x 10^6 Cells | Phase 1 Anakinra 600 x 10^6 Cells | Phase 2 600 x 10^6 Cells | Phase 2a 600 x 10^6 Cells Anti-BCMA |
|---|---|---|---|---|---|---|---|---|
| Day 29 | 14 | 29 | 26 | 20 | 17 | 14 | 23 | 10 |
| Day 60 | 9 | 26 | 21 | 16 | 13 | 12 | 21 | 10 |
| Day 90 | 5 | 16 | 20 | 17 | 9 | 13 | 18 | 7 |
| Day 180 | 2 | 8 | 14 | 12 | 8 | 9 | 13 | 4 |
| Day 270 | 1 | 2 | 9 | 2 | 9 | 7 | 8 | 4 |
| Day 365 | 0 | 1 | 4 | 2 | 6 | 4 | 8 | 3 |
| Day 545 | 0 | 0 | 4 | 4 | 2 | 3 | 3 | 2 |
| Day 730 | 0 | 0 | 2 | 2 | 4 | 2 | 1 | 1 |
ORR is defined as stringent complete response (sCR), complete response (CR), very good partial response (VGPR), or partial response (PR), according to IMWG criteria. Participants without any reported disease response assessments will be considered non-responders. sCR=complete response plus normal free light chain ratio and absence of clonal cells in bone marrow biopsy by immunohistochemistry; CR=negative immunofixation of serum and urine and disappearance of any soft tissue plasmacytomas and \< 5% plasma cells in bone marrow aspirates. When the only method to measure disease is by serum FLC levels, CR can be defined as a normal FLC ratio of 0.26 to 1.65; VGPR=serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥ 90% reduction in serum M-protein level plus urine M-protein level \< 100 mg/24 h; PR=≥ 50% reduction of serum M protein plus reduction in 24-hour urinary M-protein by ≥ 90% or to \< 200 mg/24 h
| Percent of participants | Phase 1 50 x 10^6 Cells | Phase 1 150 x 10^6 Cells | Phase 1 300 x 10^6 Cells | Phase 1 450 x 10^6 Cells | Phase 1 600 x 10^6 Cells | Phase 1 Anakinra 600 x 10^6 Cells |
|---|---|---|---|---|---|---|
| Overall Response Rate (ORR) in Phase 1 and Phase 1 Anakinra | 78.6 (49.2 to 95.3) | 86.7 (69.3 to 96.2) | 96.2 (80.4 to 99.9) | 90.0 (68.3 to 98.8) | 100.0 (83.2 to 100.0) | 100.0 (76.8 to 100.0) |
CRR is defined as stringent complete response (sCR) or complete response (CR), according to IMWG criteria. Participants without any reported disease response assessments will be considered non-responders. sCR=complete response plus normal free light chain ratio and absence of clonal cells in bone marrow biopsy by immunohistochemistry; CR=negative immunofixation of serum and urine and disappearance of any soft tissue plasmacytomas and \< 5% plasma cells in bone marrow aspirates
| Percent of participants | Phase 1 50 x 10^6 Cells | Phase 1 150 x 10^6 Cells | Phase 1 300 x 10^6 Cells | Phase 1 450 x 10^6 Cells | Phase 1 600 x 10^6 Cells | Phase 1 Anakinra 600 x 10^6 Cells | Phase 2 600 x 10^6 Cells | Phase 2a 600 x 10^6 Cells Anti-BCMA |
|---|---|---|---|---|---|---|---|---|
| Complete Response Rate (CRR) | 42.9 (17.7 to 71.1) | 33.3 (17.3 to 52.8) | 46.2 (26.6 to 66.6) | 45.0 (23.1 to 68.5) | 65.0 (40.8 to 84.6) | 35.7 (12.8 to 64.9) | 54.2 (32.8 to 74.4) | 40.0 (12.2 to 73.8) |
DoR is defined as the time from first response (stringent complete response (sCR), complete response (CR), very good partial response (VGPR), or partial response (PR)) to the earlier date of progressive disease or death due to any cause. sCR=complete response plus normal free light chain ratio and absence of clonal cells in bone marrow biopsy by immunohistochemistry; CR=negative immunofixation of serum and urine and disappearance of any soft tissue plasmacytomas and \< 5% plasma cells in bone marrow aspirates; VGPR=serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥ 90% reduction in serum M-protein level plus urine M-protein level \< 100 mg/24 h; PR=≥ 50% reduction of serum M protein plus reduction in 24-hour urinary M-protein by ≥ 90% or to \< 200 mg/24 h. Progressive disease is defined as (1) Increase of ≥25% from lowest confirmed response; (2) Appearance of a new lesion(s); (3) ≥50% increase in circulating plasma cells.
| Months | Phase 2 600 x 10^6 Cells | Phase 2a 600 x 10^6 Cells Anti-BCMA |
|---|---|---|
| Duration of Response (DoR) in Phase 2 and 2a | 18.2 (2 to 23) | 10.1 (1 to 23) |
DoCR is defined as the time from first response (stringent complete response (sCR), complete response (CR)) to the earlier date of progressive disease or death due to any cause. sCR=complete response plus normal free light chain ratio and absence of clonal cells in bone marrow biopsy by immunohistochemistry; CR=negative immunofixation of serum and urine and disappearance of any soft tissue plasmacytomas and \< 5% plasma cells in bone marrow aspirates. Progressive disease is defined as (1) Increase of ≥25% from lowest confirmed response; (2) Appearance of a new lesion(s); (3) ≥50% increase in circulating plasma cells.
| Months | Phase 2 600 x 10^6 Cells | Phase 2a 600 x 10^6 Cells Anti-BCMA |
|---|---|---|
| Duration of Complete Response (DoCR) in Phase 2 and 2a | 18.2 (0 to 23) | 10.8 (3 to 23) |
TEAE is defined as an AE that starts any time from initiation of JCARH125 administration through and including 90 days following the JCARH125 infusion graded using the NCI Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. Any AE occurring after the initiation of another anticancer treatment will not be considered a TEAE. Grade 3=Severe; Grade 4=Life-threatening; and Grade 5=death.
| Participants | Phase 2 600 x 10^6 Cells | Phase 2a 600 x 10^6 Cells Anti-BCMA |
|---|---|---|
| TEAE | 24 | 10 |
| Grade 3-5 TEAE | 23 | 9 |
| Grade 5 TEAE | 2 | 0 |
| JCARH125-Related TEAE | 23 | 8 |
| JCARH125-Related Grade 3-5 TEAE | 16 | 7 |
| JCARH125-Related Grade 5 TEAE | 1 | 0 |
Clinically significant laboratory abnormalities are assessed by investigator and are reported as treatment-emergent adverse event (TEAE). TEAE is defined as an AE that starts any time from initiation of JCARH125 administration through and including 90 days following the JCARH125 infusion graded by Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. Any AE occurring after the initiation of another anticancer treatment is not considered a TEAE. Grade 3=Severe; Grade 4=Life-threatening.
| Participants | Phase 2 600 x 10^6 Cells | Phase 2a 600 x 10^6 Cells Anti-BCMA |
|---|---|---|
| Neutropenia grade 3 | 3 | 1 |
| Neutropenia grade 4 | 18 | 6 |
| Anemia grade 3 | 11 | 3 |
| Thrombocytopenia grade 3 | 3 | 0 |
| Thrombocytopenia grade 4 | 10 | 3 |
| Leukopenia grade 3 | 1 | 0 |
| Leukopenia grade 4 | 8 | 1 |
| Lymphopenia grade 3 | 1 | 0 |
| Lymphopenia grade 4 | 3 | 0 |
| Febrile neutropenia grade 3 | 1 | 0 |
| Hypophosphataemia grade 3 | 6 | 1 |
| Hypocalcaemia grade 3 | 3 | 0 |
| Hypocalcaemia grade 4 | 1 | 0 |
| Hypokalaemia grade 3 | 2 | 0 |
| Hyponatraemia grade 3 | 2 | 0 |
| Hyperglycaemia grade 3 | 2 | 0 |
| Hypertriglyceridaemia grade 3 | 3 | 0 |
| Lactic acidosis grade 3 | 1 | 0 |
| Alanine aminotransferase increased grade 4 | 1 | 0 |
| Aspartate aminotransferase increased grade 3 | 1 | 0 |
| Aspartate aminotransferase increased grade 4 | 1 | 0 |
| Blood fibrinogen decreased grade 3 | 1 | 0 |
| Blood fibrinogen decreased grade 4 | 1 | 0 |
| CD4 lymphocytes decreased grade 3 | 1 | 0 |
OS is defined as the time from the JCARH125 infusion until death due to any cause.
| Months | Phase 2 600 x 10^6 Cells | Phase 2a 600 x 10^6 Cells Anti-BCMA |
|---|---|---|
| Overall Survival (OS) in Phase 2 and Phase 2a | NA (8.71 to NA) | NA (7.62 to NA) |
PFS is defined as the time from JCARH125 infusion until the earliest date of disease progression or death from any cause. Progressive disease (PD) is defined as (1) Increase of ≥25% from lowest confirmed response in one or more of the following: Serum M-protein absolute increase ≥0.5 g/dL; Serum M-protein increase ≥1 g/dL, if the lowest M component was ≥5 g/dL; Urine M protein absolute increase ≥200 mg/24 h; the difference between involved and uninvolved FLC levels absolute increase \>10 mg/dL; Bone marrow plasma-cell percentage irrespective of baseline status absolute increase ≥10%. (2) Appearance of a new lesion(s), ≥50% increase from nadir in SPDd of \>1 lesion, or ≥50% increase in the longest diameter of a previous lesion \>1 cm in short axis. (3) ≥50% increase in circulating plasma cells (minimum of 200 cells μL) if this is the only measure of disease.
| Months | Phase 2 600 x 10^6 Cells | Phase 2a 600 x 10^6 Cells Anti-BCMA |
|---|---|---|
| Progression Free Survival (PFS) in Phase 2 and Phase 2a | 14.75 (5.85 to 23.82) | 12.25 (3.25 to 17.94) |
TTR is defined from JCARH125 infusion to the first document of stringent complete response (sCR), complete response (CR), very good partial response (VGPR), or partial response (PR). sCR=complete response plus normal free light chain ratio and absence of clonal cells in bone marrow biopsy by immunohistochemistry; CR=negative immunofixation of serum and urine and disappearance of any soft tissue plasmacytomas and \< 5% plasma cells in bone marrow aspirates; VGPR=serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥ 90% reduction in serum M-protein level plus urine M-protein level \< 100 mg/24 h; PR=≥ 50% reduction of serum M protein plus reduction in 24-hour urinary M-protein by ≥ 90% or to \< 200 mg/24 h. Progressive disease is defined as (1) Increase of ≥25% from lowest confirmed response; (2) Appearance of a new lesion(s); (3) ≥50% increase in circulating plasma cells.
| Months | Phase 2 600 x 10^6 Cells | Phase 2a 600 x 10^6 Cells Anti-BCMA |
|---|---|---|
| Time to Response (TTR) in Phase 2 and Phase 2a | 0.99 (0.9 to 12.5) | 0.97 (0.9 to 2.2) |
TTCR is defined from JCARH125 infusion to the first document of stringent complete response (sCR) or complete response (CR). sCR=complete response plus normal free light chain ratio and absence of clonal cells in bone marrow biopsy by immunohistochemistry; CR=negative immunofixation of serum and urine and disappearance of any soft tissue plasmacytomas and \< 5% plasma cells in bone marrow aspirates. Progressive disease is defined as (1) Increase of ≥25% from lowest confirmed response; (2) Appearance of a new lesion(s); (3) ≥50% increase in circulating plasma cells.
| Months | Phase 2 600 x 10^6 Cells | Phase 2a 600 x 10^6 Cells Anti-BCMA |
|---|---|---|
| Time to Complete Response (TTCR) in Phase 2 and Phase 2a | 2.79 (1.0 to 15.2) | 1.41 (1.0 to 2.2) |
The EORTC QLQ-C30 is a 30-item scale composed of both multi-item scales and single-item measures such as functional scales (physical, role, cognitive, emotional, and social), global health status, symptom scales (fatigue, pain, nausea/vomiting), and other (dyspnoea, appetite loss, insomnia, constipation/diarrhea, and financial difficulties). Most questions used 4-point scale (1 'Not at All' to 4 'Very Much'); 2 questions used 7-point scale (1 'Very Poor' to 7 'Excellent'). Subscale scores are transformed to 0 to 100 scale, with higher scores on functional scales indicating better function and higher score on symptom scales indicating worse symptoms. Baseline is defined as the last non-missing measurement prior to JCARH125 infusion.
| Score on a Scale | Phase 2 600 x 10^6 Cells |
|---|---|
| Global Health Status/QoL Month 24 | -2.08 ± 10.49 |
| Physical Functioning Month 24 | -6.67 ± 9.43 |
| Role Functioning Month 24 | 0.00 ± 13.61 |
| Emotional Functioning Month 24 | -6.25 ± 7.98 |
| Cognitive Functioning Month 24 | 4.17 ± 8.33 |
| Social Functioning Month 24 | 16.67 ± 13.61 |
| Fatigue Month 24 | 5.56 ± 6.42 |
| Pain Month 24 | -4.17 ± 8.33 |
| Nausea and Vomiting Month 24 | 4.17 ± 8.33 |
| Dyspnea Month 24 | -8.33 ± 16.67 |
| Insomnia Month 24 | 0.00 ± 27.22 |
| Appetite Loss Month 24 | 8.33 ± 16.67 |
| Constipation Month 24 | 8.33 ± 16.67 |
| Diarrhea Month 24 | 8.33 ± 16.67 |
| Financial Difficulties Month 24 | 8.33 ± 16.67 |
QLQ-MY20 includes 20 questions across four scales: disease symptoms, treatment side-effects, future perspective, and body image. Questions used 4-point scale (1 'Not at All' to 4 'Very Much'). Scores are averaged, and transformed to 0-100 scale; higher score for the disease symptoms scale = higher level of symptomatology.
| Score on a Scale | Phase 2 600 x 10^6 Cells |
|---|---|
| Disease Symptoms | 0.00 ± 0.00 |
| Side Effects of Treatment | 6.11 ± 3.98 |
| Future Perspectives | 11.11 ± 9.07 |
| Body Image | 16.67 ± 33.33 |
EQ-5D-5L is a standardized measure of health status that consists of descriptive system and Visual Analogue scale VAS). The descriptive system comprises dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression). Each dimension has 5 levels (no problems, slight problems, moderate problems, severe problems, extreme problems). Responses are coded so that a '1' indicates no problem, and '5' indicates the most serious problem. The responses for the dimensions are combined in a 5-digit number corresponding to response categories for successive dimensions using a scoring algorithm. The VAS system has endpoints labeled "the best health you can imagine" and "the worst health you can imagine." The scale is numbered from 0 to 100 with 0 corresponding to the worst imaginable health state and 100 corresponding to the best imaginable health state. A high score represents a better level of quality of life
| Score on a scale | Phase 2 600 x 10^6 Cells |
|---|---|
| Mobility Index Score | -0.3 ± 0.5 |
| Self Care Index Score | 0.0 ± 0.0 |
| Activity Index Score | 0.3 ± 0.5 |
| Pain Index Score | 0.0 ± 0.0 |
| Anxiety Index Score | 0.3 ± 1.0 |
| Utility Index US Index Score | -0.0035 ± 0.1193 |
Total length of all intensive care unit (ICU) and non-ICU stays from JCARH125 Administration. ICU inpatient and non-ICU inpatient are not exclusive. Total length includes participants who had multiple hospitalization stays.
| Days | Phase 2 600 x 10^6 Cells |
|---|---|
| ICU inpatient | 9.0 (3 to 32) |
| non-ICU inpatient | 11.0 (6 to 58) |
Number of participants with reasons for hospitalization from JCARH125 administration
| Participants | Phase 2 600 x 10^6 Cells |
|---|---|
| Adverse Event | 3 |
| Prophylaxis | 21 |
Collected over Participants were assessed for all-cause mortality from the first participant undergoing leukapheresis until their study completion (up to approximately 61 months). SAEs and other AEs were assessed from first JCARH125 infusion until 90 days following the JCARH125 infusion (up to approximately 41 months).. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Phase 1 50 x 10^6 Cells | 5/15 (33.3%) | 1/14 (7.1%) | 14/14 (100%) |
| Phase 1 150 x 10^6 Cells | 22/30 (73.3%) | 14/30 (46.7%) | 30/30 (100%) |
| Phase 1 300 x 10^6 Cells | 14/27 (51.9%) | 7/26 (26.9%) | 26/26 (100%) |
| Phase 1 450 x 10^6 Cells | 9/22 (40.9%) | 7/21 (33.3%) | 21/21 (100%) |
| Phase 1 600 x 10^6 Cells | 7/22 (31.8%) | 7/20 (35%) | 20/20 (100%) |
| Phase 1 Anakinra 600 x 10^6 Cells | 4/14 (28.6%) | 5/14 (35.7%) | 14/14 (100%) |
| Phase 2 600 x 10^6 Cells | 6/25 (24%) | 11/24 (45.8%) | 24/24 (100%) |
| Phase 2a 600 x 10^6 Cells Anti-BCMA | 2/10 (20%) | 1/10 (10%) | 10/10 (100%) |
| Event | Phase 1 50 x 10^6 Cells | Phase 1 150 x 10^6 Cells | Phase 1 300 x 10^6 Cells | Phase 1 450 x 10^6 Cells | Phase 1 600 x 10^6 Cells | Phase 1 Anakinra 600 x 10^6 Cells | Phase 2 600 x 10^6 Cells | Phase 2a 600 x 10^6 Cells Anti-BCMA |
|---|---|---|---|---|---|---|---|---|
| Cytokine release syndromeImmune system disorders | 0/14 | 1/30 | 3/26 | 2/21 | 2/20 | 2/14 | 3/24 | 0/10 |
| NeutropeniaBlood and lymphatic system disorders | 0/14 | 2/30 | 1/26 | 1/21 | 2/20 | 0/14 | 0/24 | 0/10 |
| ThrombocytopeniaBlood and lymphatic system disorders | 0/14 | 3/30 | 1/26 | 0/21 | 1/20 | 0/14 | 0/24 | 0/10 |
| PneumoniaInfections and infestations | 0/14 | 3/30 | 0/26 | 1/21 | 0/20 | 0/14 | 0/24 | 0/10 |
| Immune effector cell-associated neurotoxicity syndromeNervous system disorders | 0/14 | 1/30 | 2/26 | 1/21 | 0/20 | 1/14 | 1/24 | 1/10 |
| Haemophagocytic lymphohistiocytosisImmune system disorders | 0/14 | 0/30 | 1/26 | 2/21 | 1/20 | 0/14 | 1/24 | 0/10 |
| SepsisInfections and infestations | 0/14 | 0/30 | 0/26 | 2/21 | 0/20 | 0/14 | 0/24 | 0/10 |
| BacteraemiaInfections and infestations | 0/14 | 0/30 | 0/26 | 0/21 | 0/20 | 0/14 | 2/24 | 0/10 |
| MyopathyMusculoskeletal and connective tissue disorders | 0/14 | 0/30 | 2/26 | 0/21 | 0/20 | 0/14 | 0/24 | 0/10 |
| Confusional statePsychiatric disorders | 0/14 | 0/30 | 2/26 | 0/21 | 0/20 | 1/14 | 0/24 | 0/10 |
| Event | Phase 1 50 x 10^6 Cells | Phase 1 150 x 10^6 Cells | Phase 1 300 x 10^6 Cells | Phase 1 450 x 10^6 Cells | Phase 1 600 x 10^6 Cells | Phase 1 Anakinra 600 x 10^6 Cells | Phase 2 600 x 10^6 Cells | Phase 2a 600 x 10^6 Cells Anti-BCMA |
|---|---|---|---|---|---|---|---|---|
| NeutropeniaBlood and lymphatic system disorders | 14/14 | 29/30 | 24/26 | 21/21 | 20/20 | 11/14 | 21/24 | 7/10 |
| Cytokine release syndromeImmune system disorders | 9/14 | 21/30 | 21/26 | 19/21 | 16/20 | 11/14 | 19/24 | 7/10 |
| ThrombocytopeniaBlood and lymphatic system disorders | 5/14 | 21/30 | 16/26 | 14/21 | 14/20 | 9/14 | 19/24 | 4/10 |
| AnaemiaBlood and lymphatic system disorders | 9/14 | 21/30 | 17/26 | 13/21 | 15/20 | 11/14 | 18/24 | 5/10 |
| LeukopeniaBlood and lymphatic system disorders | 4/14 | 8/30 | 14/26 | 9/21 | 6/20 | 5/14 | 9/24 | 1/10 |
| HypophosphataemiaMetabolism and nutrition disorders | 2/14 | 9/30 | 7/26 | 7/21 | 9/20 | 7/14 | 8/24 | 2/10 |
| HypokalaemiaMetabolism and nutrition disorders | 3/14 | 14/30 | 5/26 | 6/21 | 6/20 | 4/14 | 4/24 | 1/10 |
| NauseaGastrointestinal disorders | 6/14 | 13/30 | 5/26 | 5/21 | 3/20 | 6/14 | 8/24 | 2/10 |
| DiarrhoeaGastrointestinal disorders | 6/14 | 9/30 | 5/26 | 8/21 | 7/20 | 3/14 | 6/24 | 1/10 |
| HypoalbuminaemiaMetabolism and nutrition disorders | 0/14 | 2/30 | 0/26 | 0/21 | 3/20 | 6/14 | 1/24 | 0/10 |
| Age, Categorical(Participants) | Phase 1 50 x 10^6 Cells | Phase 1 150 x 10^6 Cells | Phase 1 300 x 10^6 Cells | Phase 1 450 x 10^6 Cells | Phase 1 600 x 10^6 Cells | Phase 1 Anakinra 600 x 10^6 Cells | Phase 2 600 x 10^6 Cells | Phase 2a 600 x 10^6 Cells Anti-BCMA | Total |
|---|---|---|---|---|---|---|---|---|---|
| <=18 years | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Between 18 and 65 years | 12 | 19 | 16 | 15 | 14 | 9 | 17 | 5 | 107 |
| >=65 years | 3 | 11 | 11 | 7 | 8 | 5 | 8 | 5 | 58 |
| Sex: Female, Male(Participants) | Phase 1 50 x 10^6 Cells | Phase 1 150 x 10^6 Cells | Phase 1 300 x 10^6 Cells | Phase 1 450 x 10^6 Cells | Phase 1 600 x 10^6 Cells | Phase 1 Anakinra 600 x 10^6 Cells | Phase 2 600 x 10^6 Cells | Phase 2a 600 x 10^6 Cells Anti-BCMA | Total |
|---|---|---|---|---|---|---|---|---|---|
| Female | 5 | 13 | 10 | 9 | 10 | 7 | 8 | 6 | 68 |
| Male | 10 | 17 | 17 | 13 | 12 | 7 | 17 | 4 | 97 |
| Ethnicity (NIH/OMB)(Participants) | Phase 1 50 x 10^6 Cells | Phase 1 150 x 10^6 Cells | Phase 1 300 x 10^6 Cells | Phase 1 450 x 10^6 Cells | Phase 1 600 x 10^6 Cells | Phase 1 Anakinra 600 x 10^6 Cells | Phase 2 600 x 10^6 Cells | Phase 2a 600 x 10^6 Cells Anti-BCMA | Total |
|---|---|---|---|---|---|---|---|---|---|
| Hispanic or Latino | 1 | 3 | 1 | 3 | 2 | 1 | 1 | 0 | 12 |
| Not Hispanic or Latino | 13 | 25 | 25 | 19 | 20 | 12 | 24 | 7 | 145 |
| Unknown or Not Reported | 1 | 2 | 1 | 0 | 0 | 1 | 0 | 3 | 8 |
| Race (NIH/OMB)(Participants) | Phase 1 50 x 10^6 Cells | Phase 1 150 x 10^6 Cells | Phase 1 300 x 10^6 Cells | Phase 1 450 x 10^6 Cells | Phase 1 600 x 10^6 Cells | Phase 1 Anakinra 600 x 10^6 Cells | Phase 2 600 x 10^6 Cells | Phase 2a 600 x 10^6 Cells Anti-BCMA | Total |
|---|---|---|---|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Asian | 1 | 1 | 0 | 2 | 0 | 1 | 1 | 2 | 8 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Black or African American | 4 | 1 | 2 | 2 | 3 | 2 | 2 | 1 | 17 |
| White | 9 | 22 | 23 | 16 | 16 | 9 | 20 | 4 | 119 |
| More than one race | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 1 | 6 | 2 | 2 | 3 | 2 | 2 | 3 | 21 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: Undecided
Ran from Feb 2018 to primary completion in Mar 2023, 61 months.
Results were first posted in May 2024, 1 year 1 month after primary completion.
Of 99 industry-sponsored phase 1/2 interventional studies under Multiple Myeloma, half reached primary completion within 64 months of starting (the median), counting those still running; a quarter within 40 and three quarters within 86. This study's 61 months is close to that.
Counting every sponsor, half of 201 studies reached it within 64 months; a quarter within 44 and three quarters within 90.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Apr 2024. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Interleukin 1 Receptor Antagonist Protein
Juno Therapeutics, a Subsidiary of Celgene →