NCT03602612

T Cells Expressing a Novel Fully-Human Anti-BCMA CAR for Treating Multiple Myeloma

Active, enrolled Phase 1 Results posted Last updated 25 February 2026

What this trial tests

Phase 1 trial testing Cyclophosphamide in Myeloma-Multiple in 35 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

14 September 2018

Primary endpoint
1 January 2023

21 April 2026

Quick facts

Lead sponsor	National Cancer Institute (NCI)
Phase	Phase 1
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	non randomized
Design	sequential
Masking	none
Primary purpose	treatment
Enrollment	35
Start date	14 September 2018
Primary completion	1 January 2023
Estimated completion	21 April 2026
Sites	1 location across United States

Drugs / interventions tested

• Cyclophosphamide (cyclophosphamide) — full drug profile →
• Fludarabine (FLUDARABINE) — full drug profile →
• Anti-B Cell Maturation Antigen (BCMA) chimeric antigen receptors (CARs) T cells — full drug profile →

Conditions studied

• Myeloma-Multiple — all drugs for Myeloma-Multiple →
• Myeloma, Plasma-Cell — all drugs for Myeloma, Plasma-Cell →

Sponsor

National Cancer Institute (NCI)

Who can join

Adults 18 to 73, any sex, with Myeloma-Multiple or Myeloma, Plasma-Cell. 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.

Maximum Tolerated Dose (MTD) of Anti-B Cell Maturation Antigen (BCMA) - Chimeric Antigen Receptor (CAR)-T Cells Primary · First 28 days of treatment

The MTD is the dose at which a maximum of 1 of 6 patients has a DLT. A DLT is Grade 3 toxicities possibly or probably or definitely related to the Anti-B cell maturation antigen (BCMA) - chimeric antigen receptor (CAR)-expressing T-Cells lasting more than 9 days. Grade 4 toxicities possibly or probably or definitely related to the anti-BCMA CAR T cells. Grade 3 is severe, and Grade 4 is life-threatening.

Group	Value	95% CI
All Arm 1 Dose Escalation Dose Participants	6

Number of Participants at Each Dose Level Who Experience a Dose-Limiting Toxicity (DLT) Primary · First 28 days of treatment

A DLT is Grade 3 toxicities possibly or probably or definitely related to the anti-B cell maturation antigen (BCMA) - chimeric antigen receptor (CAR)-T cells and lasting more than 9 days. Grade 4 toxicities possibly or probably or definitely related to the anti-BCMA CAR T cells. Grade 3 is severe, and Grade 4 is life-threatening.

Group	Value	95% CI
Arm 1 Dose Escalation (Esc) Dose Level 1 - 0.75x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg	1
Arm 1 Dose Escalation Dose Level 2 - 1.5x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg	0
Arm 1 Dose Escalation Dose Level 3 - 3.0x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg	0
Arm 1 Dose Escalation Dose Level 4 - 6.0x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg	0
Arm 1 Dose Escalation Dose Level 5 - 12.0x1^06 Chimeric Antigen Receptor (CAR)+ T Cells Per kg	0
Arm 2 Dose Expansion (Exp) 6.0x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg	0
Arm 1 DoseEsc DL 3-3.0x10^6 CAR+T Cells Per kg Followed by Arm 2 DoseExp 6.0x10^6 CAR+T Cells Per kg	0

Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). Secondary · Date treatment consent signed to date off study, approximately 16 months/17 days, 4 months/4 days, 4 months/18 days, 42 months/8 days, 20 months/19 days, 29 months/28 days, and 30 months/26 days for each Arm/Group respectively.

Here is the number of participants with serious and/or non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent

Group	Value	95% CI
Arm 1 Dose Escalation (Esc) Dose Level 1 - 0.75x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg	6
Arm 1 Dose Escalation Dose Level 2 - 1.5x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg	3
Arm 1 Dose Escalation Dose Level 3 - 3.0x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg	2
Arm 1 Dose Escalation Dose Level 4 - 6.0x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg	3
Arm 1 Dose Escalation Dose Level 5 - 12.0x1^06 Chimeric Antigen Receptor (CAR)+ T Cells Per kg	3
Arm 2 Dose Expansion (Exp) 6.0x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg	8
Arm 1 DoseEsc DL 3-3.0x10^6 CAR+T Cells Per kg Followed by Arm 2 DoseExp 6.0x10^6 CAR+T Cells Per kg	1

Adverse events — posted to ClinicalTrials.gov

Time frame: Date treatment consent signed to date off study, approximately 16 months/17 days, 4 months/4 days, 4 months/18 days, 42 months/8 days, 20 months/19 days, 29 months/28 days, and 30 months/26 days for each Arm/Group respectively.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Arm 1 Dose Escalation (Esc) Dose Level 1 - 0.75x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg

Serious: 6/6 (100%)

Deaths: 1/6

Arm 1 Dose Escalation Dose Level 2 - 1.5x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg

Serious: 1/3 (33%)

Deaths: 0/3

Arm 1 Dose Escalation Dose Level 3 - 3.0x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg

Serious: 2/2 (100%)

Deaths: 0/2

Arm 1 Dose Escalation Dose Level 4 - 6.0x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg

Serious: 2/3 (67%)

Deaths: 0/3

Arm 1 Dose Escalation Dose Level 5 - 12.0x1^06 Chimeric Antigen Receptor (CAR)+ T Cells Per kg

Serious: 3/3 (100%)

Deaths: 0/3

Arm 2 Dose Expansion (Exp) 6.0x10^6 Chimeric Antigen Receptor (CAR)+ T Cells Per kg

Serious: 6/8 (75%)

Deaths: 0/8

Arm 1 DoseEsc DL 3-3.0x10^6 CAR+T Cells Per kg Followed by Arm 2 DoseExp 6.0x10^6 CAR+T Cells Per kg

Serious: 1/1 (100%)

Deaths: 0/1

Serious adverse events (30 terms)

Reaction	System	Arm 1 Dose Escalation (Esc…	Arm 1 Dose Escalation Dose…	Arm 1 Dose Escalation Dose…	Arm 1 Dose Escalation Dose…	Arm 1 Dose Escalation Dose…	Arm 2 Dose Expansion (Exp)…	Arm 1 DoseEsc DL 3-3.0x10^…
Cytokine release syndrome	Immune system disorders	—	—	—	—	—	—	—
Fever	General disorders	—	—	—	—	—	—	—
Hypotension	Vascular disorders	—	—	—	—	—	—	—
Lung infection	Infections and infestations	—	—	—	—	—	—	—
Upper respiratory infection	Infections and infestations	—	—	—	—	—	—	—
Anemia	Blood and lymphatic system disorders	—	—	—	—	—	—	—
Atrial fibrillation	Cardiac disorders	—	—	—	—	—	—	—
Back pain	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—	—
CPK increased	Investigations	—	—	—	—	—	—	—
Cognitive disturbance	Nervous system disorders	—	—	—	—	—	—	—
Confusion	Psychiatric disorders	—	—	—	—	—	—	—
Cough	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—	—
Diarrhea	Gastrointestinal disorders	—	—	—	—	—	—	—
Dysarthria	Nervous system disorders	—	—	—	—	—	—	—
Dyspnea	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—	—
Ejection fraction decreased	Investigations	—	—	—	—	—	—	—
Headache	Nervous system disorders	—	—	—	—	—	—	—
Hemolysis	Blood and lymphatic system disorders	—	—	—	—	—	—	—
Hypoxia	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—	—
Nervous system disorders - Other, Saddle anesthesia with pinprick below the mid calves.	Nervous system disorders	—	—	—	—	—	—	—
Non-cardiac chest pain	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—	—
Pneumothorax	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—	—
Respiratory failure - death (due to influenza) grade 5	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—	—
Respiratory, thoracic and mediastinal disorders - Other, tachypnea	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—	—
Shingles	Infections and infestations	—	—	—	—	—	—	—

Other adverse events (137 terms — click to expand)

Reaction	System	Arm 1 Dose Escalation (Esc…	Arm 1 Dose Escalation Dose…	Arm 1 Dose Escalation Dose…	Arm 1 Dose Escalation Dose…	Arm 1 Dose Escalation Dose…	Arm 2 Dose Expansion (Exp)…	Arm 1 DoseEsc DL 3-3.0x10^…
Anemia	Blood and lymphatic system disorders	—	—	—	—	—	—	—
Lymphocyte count decreased	Investigations	—	—	—	—	—	—	—
Neutrophil count decreased	Investigations	—	—	—	—	—	—	—
White blood cell decreased	Investigations	—	—	—	—	—	—	—
Fever	General disorders	—	—	—	—	—	—	—
Hypophosphatemia	Metabolism and nutrition disorders	—	—	—	—	—	—	—
Platelet count decreased	Investigations	—	—	—	—	—	—	—
Sinus tachycardia	Cardiac disorders	—	—	—	—	—	—	—
Fatigue	General disorders	—	—	—	—	—	—	—
Hypoalbuminemia	Metabolism and nutrition disorders	—	—	—	—	—	—	—
Upper respiratory infection	Infections and infestations	—	—	—	—	—	—	—
Cough	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—	—
Diarrhea	Gastrointestinal disorders	—	—	—	—	—	—	—
Hypertension	Vascular disorders	—	—	—	—	—	—	—
Hypokalemia	Metabolism and nutrition disorders	—	—	—	—	—	—	—
Hypotension	Vascular disorders	—	—	—	—	—	—	—
Nausea	Gastrointestinal disorders	—	—	—	—	—	—	—
Anorexia	Metabolism and nutrition disorders	—	—	—	—	—	—	—
Nasal congestion	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—	—
Chills	General disorders	—	—	—	—	—	—	—
Cytokine release syndrome	Immune system disorders	—	—	—	—	—	—	—
Dyspnea	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—	—
Myalgia	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—	—
Pain in extremity	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—	—
Alanine aminotransferase increased	Investigations	—	—	—	—	—	—	—
Aspartate aminotransferase increased	Investigations	—	—	—	—	—	—	—
Back pain	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—	—
CPK increased	Investigations	—	—	—	—	—	—	—
Confusion	Psychiatric disorders	—	—	—	—	—	—	—
Edema limbs	General disorders	—	—	—	—	—	—	—
Hyponatremia	Metabolism and nutrition disorders	—	—	—	—	—	—	—
Hypoxia	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—	—
Insomnia	Psychiatric disorders	—	—	—	—	—	—	—
Non-cardiac chest pain	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—	—
Sinusitis	Infections and infestations	—	—	—	—	—	—	—
Sore throat	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—	—
Nervous system disorders - Other, Restless Leg Syndrome	Nervous system disorders	—	—	—	—	—	—	—
Abdominal infection	Infections and infestations	—	—	—	—	—	—	—
Abdominal pain	Gastrointestinal disorders	—	—	—	—	—	—	—
Activated partial thromboplastin time prolonged	Investigations	—	—	—	—	—	—	—

Most-reported serious reactions: Cytokine release syndrome, Fever, Hypotension, Lung infection, Upper respiratory infection, Anemia, Atrial fibrillation, Back pain.

Data from ClinicalTrials.gov NCT03602612 adverse events section.

Sponsor's own description

Background:

Multiple myeloma is a cancer of the blood plasma cells. It usually becomes resistant to standard treatments. Researchers have developed a procedure called gene therapy. It uses a person's own T cells, which are part of the immune system. The cells are changed in a lab and then returned to the person. Researchers hope the changed T cells will be better at recognizing and killing tumor cells.

Objective:

To test the safety of giving changed T cells to people with multiple myeloma.

Eligibility:

Adults ages 18-73 who have been diagnosed with multiple myeloma that has not been controlled with standard therapies.

Design:

Participants will be screened with:

Medical history

Physical exam

Blood tests

Heart function tests

Bone marrow sample taken by needle in a hip bone.

Scan of the chest, abdomen, and pelvis. They may have a brain scan.

Pregnancy test

Participants will have apheresis. Blood will be removed through an arm vein. The blood will be separated, and T cells removed. The rest of the blood will be returned through a vein in the other arm.

Participants will have a central line placed in a large vein in the arm or chest.

Participants will get 2 chemotherapy drugs by the central line over 3 days.

Two days later, participants will get the changed T cells by the central line. They will stay in the hospital at least 9 days.

Participants must stay near the hospital for 2 weeks.

Participants will have 8 follow-up visits over the next year for blood and urine tests. They may have scans.

Participants blood will be collected regularly over the next several years.

Publications & conference data

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

1. Safety and clinical efficacy of BCMA CAR-T-cell therapy in multiple myeloma.
   Roex G, Timmers M, Wouters K, Campillo-Davo D, et al · · 2020 · cited 137× · PMID 33272302 · DOI 10.1186/s13045-020-01001-1
2. CAR T-cell therapy in multiple myeloma: more room for improvement.
   Teoh PJ, Chng WJ. · · 2021 · cited 123× · PMID 33927192 · DOI 10.1038/s41408-021-00469-5
3. CAR T-Cells in Multiple Myeloma: State of the Art and Future Directions.
   Rodríguez-Lobato LG, Ganzetti M, Fernández de Larrea C, Hudecek M, et al · · 2020 · cited 66× · PMID 32850376 · DOI 10.3389/fonc.2020.01243
4. Patient selection for CAR T or BiTE therapy in multiple myeloma: Which treatment for each patient?
   Kegyes D, Constantinescu C, Vrancken L, Rasche L, et al · · 2022 · cited 53× · PMID 35672793 · DOI 10.1186/s13045-022-01296-2
5. CAR T and CAR NK cells in multiple myeloma: Expanding the targets.
   Shah UA, Mailankody S. · · 2020 · cited 34× · PMID 32139020 · DOI 10.1016/j.beha.2020.101141
6. CAR-T cell therapy in Multiple Myeloma: current status and future challenges.
   Swan D, Madduri D, Hocking J. · · 2024 · cited 32× · PMID 39592597 · DOI 10.1038/s41408-024-01191-8
7. B-Cell Maturation Antigen (BCMA) as a Target for New Drug Development in Relapsed and/or Refractory Multiple Myeloma.
   Abramson HN. · · 2020 · cited 30× · PMID 32707894 · DOI 10.3390/ijms21155192
8. Chimeric antigen receptor (CAR) T-cell therapy for multiple myeloma.
   Choi T, Kang Y. · · 2022 · cited 21× · PMID 34582835 · DOI 10.1016/j.pharmthera.2021.108007