NCT03548207: CARTITUDE-1

A Study of JNJ-68284528, a Chimeric Antigen Receptor T Cell (CAR-T) Therapy Directed Against B-Cell Maturation Antigen (BCMA) in Participants With Relapsed or Refractory Multiple Myeloma

Completed Phase 1, PHASE2 Results posted Last updated 30 July 2025

What this trial tests

Phase 1, PHASE2 trial testing JNJ-68284528 in Multiple Myeloma in 126 participants. Completed in 23 August 2022.

Timeline

29 June 2018

Primary endpoint
23 August 2022

23 August 2022

Quick facts

Lead sponsor	Janssen Research & Development, LLC
Phase	Phase 1, PHASE2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	126
Start date	29 June 2018
Primary completion	23 August 2022
Estimated completion	23 August 2022
Sites	21 locations across Japan, United States

Drugs / interventions tested

• JNJ-68284528 — full drug profile →

Conditions studied

• Multiple Myeloma — all drugs for Multiple Myeloma →

Sponsor

Janssen Research & Development, LLC — full company profile →

Who can join

18 and older, any sex, with Multiple Myeloma. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Phase 1b: Number of Participants With Adverse Events as Per Severity Primary · Day 1 up to 45.2 months

An AE was any untoward medical occurrence in a clinical study participant administered a pharmaceutical (investigational or non-investigational) product. An AE does not necessarily have a causal relationship with the intervention. Severity was graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0. Severity scale ranges from Grade 1 (Mild) to Grade 5 (Death). Grade 1= Mild, Grade 2= Moderate, Grade 3= Severe, Grade 4= Life-threatening and Grade 5= Death related to adverse event.

Grade 1

Group	Value	95% CI
Phase 1b (US Population)	0

Grade 2

Group	Value	95% CI
Phase 1b (US Population)	0

Grade 3

Group	Value	95% CI
Phase 1b (US Population)	2

Grade 4

Group	Value	95% CI
Phase 1b (US Population)	26

Grade 5

Group	Value	95% CI
Phase 1b (US Population)	1

Phase 2: Overall Response Rate (ORR) Primary · Day 1 up to 45.2 months

ORR was defined as the percentage of participants who achieved partial response (PR) or better according to international myeloma working group (IMWG) criteria. IMWG criteria for PR: greater than or equal to (\>=) 50 percent (%) reduction of serum M-protein and reduction in 24-hour urinary M-protein by \>=90% or to less than (\<) 200 milligrams (mg) per 24 hours. If the serum and urine M-protein were not measurable, a decrease of \>=50% in the difference between involved and uninvolved free light chain (FLC) levels was required in place of the M-protein criteria. If serum and urine M-protein w

Group	Value	95% CI
Phase 2 (US Population)	97.1	89.8 – 99.6
Phase 2 (Japan Population)	100.0	66.4 – 100.0

Phase 2: Number of Participants With Adverse Events (AEs) as Per Severity Secondary · Day 1 up to 45.2 months

An AE was any untoward medical occurrence in a clinical study participant administered a pharmaceutical (investigational or non-investigational) product. An AE does not necessarily have a causal relationship with the intervention. Severity was graded according to the NCI-CTCAE version 5.0. Severity scale ranges from Grade 1 (Mild) to Grade 5 (Death). Grade 1= Mild, Grade 2= Moderate, Grade 3= Severe, Grade 4= Life-threatening and Grade 5= Death related to adverse event.

Grade 1

Group	Value	95% CI
Phase 2 (US Population)	0
Phase 2 (Japan Population)	0

Grade 2

Group	Value	95% CI
Phase 2 (US Population)	0
Phase 2 (Japan Population)	1

Grade 3

Group	Value	95% CI
Phase 2 (US Population)	5
Phase 2 (Japan Population)	0

Grade 4

Group	Value	95% CI
Phase 2 (US Population)	58
Phase 2 (Japan Population)	8

Grade 5

Group	Value	95% CI
Phase 2 (US Population)	5
Phase 2 (Japan Population)	0

Phase 1b and Phase 2: Maximum Observed Plasma Concentration (Cmax) of JNJ-68284528 Transgene Secondary · Pre and post dose on Day 1, and Days 2, 3, 7, 10, 14, 21, 28, 42, 56, 78, 100, then every 4 weeks up to 1 year, and at the time of progressive disease or at study completion for participants without progressive disease (up to approximately 2.5 years)

Maximum observed plasma concentration (Cmax) of JNJ-68284528 transgene was reported. Cmax was calculated in copies per microgram (mcg) genomic deoxyribonucleic acid (DNA).

Group	Value	95% CI
Phase 1b (US Population)	38965	± 19408
Phase 2 (US Population)	52841	± 29021
Phase 2 (Japan Population)	44077	± 39911

Phase 1b and Phase 2: Actual Sampling Time of Last Measurable Analyte Concentration (Tlast) of JNJ-68284528 Transgene Secondary · Pre and post dose on Day 1 and Days 2, 3, 7, 10, 14, 21, 28, 42, 56, 78, 100, then every 4 weeks up to 1 year, and at the time of progressive disease or at study completion for participants without progressive disease (up to approximately 2.5 years)

Tlast was defined as actual sampling time of last measurable (non-below quantification limit \[BQL\]) analyte concentration of JNJ-68284528 transgene.

Group	Value	95% CI
Phase 1b (US Population)	127.13	26.15 – 715.00
Phase 2 (US Population)	122.98	20.04 – 910.92
Phase 2 (Japan Population)	129.89	24.97 – 568.92

Phase 1b and Phase 2: Area Under the Plasma Concentration Versus Time Curve From Time 0 To Last Measurable Concentration (AUC0-last) of JNJ-68284528 Transgene Secondary · Pre and post dose on Day 1; Days 2, 3, 7, 10, 14, 21, 28, 42, 56, 78, 100, then every 4 weeks up to 1 year, and at the time of progressive disease or at study completion for participants without progressive disease (up to approximately 2.5 years)

Area under the plasma concentration versus time curve from time 0 to last measurable concentration of JNJ-68284528 transgene was reported.

Group	Value	95% CI
Phase 1b (US Population)	588162	± 466441
Phase 2 (US Population)	1356191	± 1673474
Phase 2 (Japan Population)	4062950	± 7293651

Phase 1b and Phase 2: Area Under the Plasma Concentration Versus Time Curve From Time 0 to Infinite Time (AUC0-infinity) of JNJ-68284528 Transgene Secondary · Pre and post dose on Day 1 and Days 2, 3, 7, 10, 14, 21, 28, 42, 56, 78, 100, then every 4 weeks up to 1 year, and at the time of progressive disease or at study completion for participants without progressive disease (up to approximately 2.5 years)

Area under the plasma concentration versus time curve from time 0 to infinite time of JNJ-68284528 transgene was reported.

Group	Value	95% CI
Phase 1b (US Population)	542728	± 345847
Phase 2 (US Population)	1321427	± 1513726
Phase 2 (Japan Population)	7173538	± 12191640

Phase 1b and Phase 2: Maximum Observed Plasma Concentration (Cmax) of CD3+CAR+ Cells in Blood After a Single Infusion of JNJ-68284528 Secondary · Pre and post dose on Day 1 and Days 2, 3, 7, 10, 14, 21, 28, 42, 56, 78, 100, then every 4 weeks up to 1 year, and at the time of progressive disease or at study completion for participants without progressive disease (up to approximately 2.5 years)

Maximum observed plasma concentration (Cmax) of CD3+CAR+ Cells was reported. Cmax was calculated in cells per microliter.

Group	Value	95% CI
Phase 1b (US Population)	483	± 464
Phase 2 (US Population)	1472	± 2085
Phase 2 (Japan Population)	1614	± 3718

Phase 1b and Phase 2: T Cell Persistence After a Single Infusion of JNJ-68284528 Secondary · Pre and post dose on Day 1 and Days 2, 3, 7, 10, 14, 21, 28, 42, 56, 78, 100, then every 4 weeks up to 1 year, and at the time of progressive disease or at study completion for participants without progressive disease (up to approximately 2.5 years)

T Cell persistence after a single infusion of JNJ-68284528 was reported. T Cell persistence was defined as actual sampling time of last measurable (non-below quantification limit \[BQL\]) analyte concentration.

Group	Value	95% CI
Phase 1b (US Population)	84.08	12.89 – 715.00
Phase 2 (US Population)	100.23	20.04 – 910.92
Phase 2 (Japan Population)	56.81	12.72 – 568.92

Phase 1b and Phase 2: Area Under the Plasma Concentration Versus Time Curve From Time 0 To Last Measurable Concentration (AUC0-last) of CD3+CAR+ Cells in Blood After a Single Infusion of JNJ-68284528 Secondary · Pre and post dose on Day 1 and Days 2, 3, 7, 10, 14, 21, 28, 42, 56, 78, 100, then every 4 weeks up to 1 year, and at the time of progressive disease or at study completion for participants without progressive disease (up to approximately 2.5 years)

Area under the plasma concentration versus time curve from time 0 to last measurable concentration of CD3+CAR+ Cells was reported.

Group	Value	95% CI
Phase 1b (US Population)	7758	± 7599
Phase 2 (US Population)	40073	± 79603
Phase 2 (Japan Population)	152904	± 361949

Phase 1b and Phase 2: Area Under the Plasma Concentration Versus Time Curve From Time 0 to Infinite Time (AUC0-infinity) of CD3+CAR+ Cells in Blood After a Single Infusion of JNJ-68284528 Secondary · Pre and post dose on Day 1 and Days 2, 3, 7, 10, 14, 21, 28, 42, 56, 78, 100, then every 4 weeks up to 1 year, and at the time of progressive disease or at study completion for participants without progressive disease (up to approximately 2.5 years)

Area under the plasma concentration versus time curve from time 0 to infinite time of CD3+CAR+ Cells in blood after a single infusion of JNJ-68284528 was reported.

Group	Value	95% CI
Phase 1b (US Population)	10937	± 8658
Phase 2 (US Population)	31966	± 58976
Phase 2 (Japan Population)	7144.5	± 5905

Phase 1b and Phase 2: Mean Concentration of Soluble B-cell Maturation Antigen (BCMA) Levels in Serum After Single Infusion of JNJ-68284528 Secondary · Days 352 and 1024

Mean concentration of soluble BCMA levels in serum after single infusion of JNJ-68284528 were reported.

Day 352

Group	Value	95% CI
Phase 1b (US Population)	2.25	NA – 7.65
Phase 2 (US Population)	2.01	NA – 10.40
Phase 2 (Japan Population)	5.61	NA – 23.0

Day 1024

Group	Value	95% CI
Phase 1b (US Population)	6.70	0.31 – 14.7
Phase 2 (US Population)	7.05	3.21 – 11.90

Adverse events — posted to ClinicalTrials.gov

Time frame: Day 1 up to 45.2 months. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Phase 1b (US Population)

Serious: 11/29 (38%)

Deaths: 10/29

Phase 2 (US Population)

Serious: 42/68 (62%)

Deaths: 25/68

Phase 2 (Japan Population)

Serious: 1/9 (11%)

Deaths: 3/9

Serious adverse events (59 terms)

Reaction	System	Phase 1b (US Population)	Phase 2 (US Population)	Phase 2 (Japan Population)
Cytokine Release Syndrome	Immune system disorders	—	—	—
Pneumonia	Infections and infestations	—	—	—
Sepsis	Infections and infestations	—	—	—
Immune Effector Cell-Associated Neurotoxicity Syndrome	Nervous system disorders	—	—	—
Febrile Neutropenia	Blood and lymphatic system disorders	—	—	—
Parkinsonism	Nervous system disorders	—	—	—
Mental Status Changes	Psychiatric disorders	—	—	—
Thrombocytopenia	Blood and lymphatic system disorders	—	—	—
Pyrexia	General disorders	—	—	—
Rhinovirus Infection	Infections and infestations	—	—	—
Neurotoxicity	Nervous system disorders	—	—	—
Acute Kidney Injury	Renal and urinary disorders	—	—	—
Hypoxia	Respiratory, thoracic and mediastinal disorders	—	—	—
Neutropenia	Blood and lymphatic system disorders	—	—	—
Atrial Fibrillation	Cardiac disorders	—	—	—
Pericardial Effusion	Cardiac disorders	—	—	—
Supraventricular Tachycardia	Cardiac disorders	—	—	—
Diplopia	Eye disorders	—	—	—
Haematochezia	Gastrointestinal disorders	—	—	—
Nausea	Gastrointestinal disorders	—	—	—
Asthenia	General disorders	—	—	—
Fatigue	General disorders	—	—	—
Gait Disturbance	General disorders	—	—	—
Cholecystitis Acute	Hepatobiliary disorders	—	—	—
Atypical Pneumonia	Infections and infestations	—	—	—

Other adverse events (95 terms — click to expand)

Reaction	System	Phase 1b (US Population)	Phase 2 (US Population)	Phase 2 (Japan Population)
Neutropenia	Blood and lymphatic system disorders	—	—	—
Cytokine Release Syndrome	Immune system disorders	—	—	—
Anaemia	Blood and lymphatic system disorders	—	—	—
Thrombocytopenia	Blood and lymphatic system disorders	—	—	—
Leukopenia	Blood and lymphatic system disorders	—	—	—
Lymphopenia	Blood and lymphatic system disorders	—	—	—
Fatigue	General disorders	—	—	—
Hypocalcaemia	Metabolism and nutrition disorders	—	—	—
Cough	Respiratory, thoracic and mediastinal disorders	—	—	—
Decreased Appetite	Metabolism and nutrition disorders	—	—	—
Hypophosphataemia	Metabolism and nutrition disorders	—	—	—
Nausea	Gastrointestinal disorders	—	—	—
Hypoalbuminaemia	Metabolism and nutrition disorders	—	—	—
Hyponatraemia	Metabolism and nutrition disorders	—	—	—
Diarrhoea	Gastrointestinal disorders	—	—	—
Aspartate Aminotransferase Increased	Investigations	—	—	—
Chills	General disorders	—	—	—
Pyrexia	General disorders	—	—	—
Hypokalaemia	Metabolism and nutrition disorders	—	—	—
Alanine Aminotransferase Increased	Investigations	—	—	—
Hypertension	Vascular disorders	—	—	—
Constipation	Gastrointestinal disorders	—	—	—
Vomiting	Gastrointestinal disorders	—	—	—
Oedema Peripheral	General disorders	—	—	—
Dizziness	Nervous system disorders	—	—	—
Headache	Nervous system disorders	—	—	—
Sinus Tachycardia	Cardiac disorders	—	—	—
Blood Lactate Dehydrogenase Increased	Investigations	—	—	—
Hypomagnesaemia	Metabolism and nutrition disorders	—	—	—
Hypotension	Vascular disorders	—	—	—
Hypofibrinogenaemia	Blood and lymphatic system disorders	—	—	—
Hypogammaglobulinaemia	Immune system disorders	—	—	—
Gamma-Glutamyltransferase Increased	Investigations	—	—	—
Arthralgia	Musculoskeletal and connective tissue disorders	—	—	—
Immune Effector Cell-Associated Neurotoxicity Syndrome	Nervous system disorders	—	—	—
Insomnia	Psychiatric disorders	—	—	—
Nasal Congestion	Respiratory, thoracic and mediastinal disorders	—	—	—
Blood Alkaline Phosphatase Increased	Investigations	—	—	—
Pain in Extremity	Musculoskeletal and connective tissue disorders	—	—	—
Upper Respiratory Tract Infection	Infections and infestations	—	—	—

Most-reported serious reactions: Cytokine Release Syndrome, Pneumonia, Sepsis, Immune Effector Cell-Associated Neurotoxicity Syndrome, Febrile Neutropenia, Parkinsonism, Mental Status Changes, Thrombocytopenia.

Data from ClinicalTrials.gov NCT03548207 adverse events section.

Sponsor's own description

The purpose of the study is to characterize safety of JNJ-68284528 and establish the recommended Phase 2 dose (RP2D) (Phase 1b) and to evaluate the efficacy of JNJ-68284528 (Phase 2).

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Ciltacabtagene autoleucel, a B-cell maturation antigen-directed chimeric antigen receptor T-cell therapy in patients with relapsed or refractory multiple myeloma (CARTITUDE-1): a phase 1b/2 open-label study.
   Berdeja JG, Madduri D, Usmani SZ, Jakubowiak A, et al · · 2021 · cited 1284× · PMID 34175021 · DOI 10.1016/s0140-6736(21)00933-8
2. Ciltacabtagene Autoleucel, an Anti-B-cell Maturation Antigen Chimeric Antigen Receptor T-Cell Therapy, for Relapsed/Refractory Multiple Myeloma: CARTITUDE-1 2-Year Follow-Up.
   Martin T, Usmani SZ, Berdeja JG, Agha M, et al · · 2023 · cited 489× · PMID 35658469 · DOI 10.1200/jco.22.00842
3. A phase 1, open-label study of LCAR-B38M, a chimeric antigen receptor T cell therapy directed against B cell maturation antigen, in patients with relapsed or refractory multiple myeloma.
   Zhao WH, Liu J, Wang BY, Chen YX, et al · · 2018 · cited 407× · PMID 30572922 · DOI 10.1186/s13045-018-0681-6
4. From bench to bedside: the history and progress of CAR T cell therapy.
   Mitra A, Barua A, Huang L, Ganguly S, et al · · 2023 · cited 292× · PMID 37256141 · DOI 10.3389/fimmu.2023.1188049
5. Incidence and management of CAR-T neurotoxicity in patients with multiple myeloma treated with ciltacabtagene autoleucel in CARTITUDE studies.
   Cohen AD, Parekh S, Santomasso BD, Gállego Pérez-Larraya J, et al · · 2022 · cited 219× · PMID 35210399 · DOI 10.1038/s41408-022-00629-1
6. Neurocognitive and hypokinetic movement disorder with features of parkinsonism after BCMA-targeting CAR-T cell therapy.
   Van Oekelen O, Aleman A, Upadhyaya B, Schnakenberg S, et al · · 2021 · cited 206× · PMID 34893771 · DOI 10.1038/s41591-021-01564-7
7. Chimeric antigen receptor (CAR) T therapies for the treatment of hematologic malignancies: clinical perspective and significance.
   Boyiadzis MM, Dhodapkar MV, Brentjens RJ, Kochenderfer JN, et al · · 2018 · cited 199× · PMID 30514386 · DOI 10.1186/s40425-018-0460-5
8. BCMA-targeted immunotherapy for multiple myeloma.
   Yu B, Jiang T, Liu D. · · 2020 · cited 165× · PMID 32943087 · DOI 10.1186/s13045-020-00962-7