Multiple MyelomaPhase 1NCT07340853
CRISPR Delivered Anti-BCMA Car-T Therapy for Relapsed or Refractory Multiple Myeloma
A Phase 1 study testing Leukapheresis, Cyclophosphamide and Chimeric Antigen Receptor T cells (CAR-T) Targeting BCMA in people with Multiple Myeloma and Recurrent Multiple Myeloma, run by Thomas Martin, MD at 1 site in United States.
Trial summary
This phase Ib trial tests the safety, side effects and best dose of clustered regularly interspaced short palindromic repeats (CRISPR) delivered anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR)-T cells (1XX BCMA CAR-T cells) in treating patients with multiple myeloma that has come back after a… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Multiple Myeloma, Recurrent Multiple Myeloma, Refractory Multiple Myeloma
- What is being tested?
- Leukapheresis, Cyclophosphamide and Chimeric Antigen Receptor T cells (CAR-T) Targeting BCMA
- Type of treatment
- Injection or infusion, Surgery or procedure, Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- 28 Feb 2026to 25 May 2043
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Voluntarily sign informed consent form.
- Age ≥18 years.
- Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1.
- Diagnosis of multiple myeloma (per IMWG criteria) with relapsed or refractory disease and has received at least 3 prior lines of therapy including proteasome inhibitor immunomodulatory therapy, and anti-Cluster of differentiation 38 (CD38) antibody therapy.
- Participants may have received BCMA targeted therapy and must be at least 6 months from last BCMA therapy.
- Participants must have documented evidence of progressive disease within 12 months of the last line of therapy or be refractory/nonresponsive to their most recent line.
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- Participants must have measurable disease, defined as at least one of the criteria below:
- Serum M-protein greater or equal to 0.5 grams per deciliter (g/dL).
- Urine M-protein greater or equal to 200 milligrams, over a 24-hour period (mg/24 h).
- Serum free light chain (FLC) assay: involved FLC level of ≥ 100 milligrams per liter (mg/L).
- Adequate organ function, defined as:
- Adequate bone marrow function for apheresis and lymphodepleting chemotherapy.
- Hgb >8 gm/dl (transfusions allowed).
- Platelets >50,000/microliter (uL) (in the absence of platelet transfusion within 7 days of apheresis, but transfusion permitted prior to lymphodepleting chemotherapy).
- Absolute neutrophil count (ANC) > 1000/uL in the absence of growth factor support (filgrastim within 7 days or pegfilgrastim within 14 days of apheresis, but growth factor permitted prior to lymphodepleting chemotherapy). For those patients who have evidence of duffy null, ANC >750/uL is allowed.
- Absolute lymphocyte count (ALC) >300/uL.
- Alanine aminotransferase/aspartate aminotransferase (ALT/AST) < 3 x institutional upper limit of normal (ULN) and Total bilirubin < 1.5 milligrams per deciliter (mg/dl) x institutional ULN, except with Gilbert's syndrome.
- Serum creatinine clearance (CrCl) ≥ 30 milliliter per minute (mL/min) using Cockcroft-Gault formula or as measured with a 24 hour urine collection.
- Adequate cardiac function, defined as left ventricular ejection fraction (LVEF) > 40% as assessed by echocardiogram or multiple uptake gated acquisition (MUGA) and adequate pulmonary function (measured by room air pulse oximetry ≥ 92%).
- Women of childbearing potential must have a negative serum or urine pregnancy test AND agree to use highly effective methods of contraception for 1 year after the last dose of anti-BCMA CAR-T cells.
- Males who have partners of childbearing potential must agree to use an effective barrier contraceptive method for 6 months after CAR-T therapy.
Who cannot join
- Autologous transplant within 12 weeks of planned CAR-T cell infusion.
- Prior antitumor therapy as follows, prior to apheresis:
- Investigational therapy within 14 days, or at least 5 half-lives.
- Monoclonal antibody therapy within 21 days.
- Cytotoxic therapy within 14 days.
- Proteasome inhibitor therapy within 14 days.
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- Immunomodulatory therapy within 14 days.
- Radiotherapy within 14 days - with the exception that if radiotherapy (XRT) covers <5% of marrow reserve - no rest window needed.
- Toxicity from previous anticancer therapy must resolve to baseline levels or to Grade 1 or less except for alopecia, peripheral neuropathy and baseline hematologic toxicity that otherwise meets inclusion.
- Active CNS multiple myeloma, plasma cell leukemia, primary AL amyloidosis or POEMS syndrome.
- Active other malignancy, other than non-melanoma skin cancer, carcinoma in situ (e.g., cervix, bladder, or breast). Any fully treated malignancies or indolent, clinically insignificant malignancies can be discussed among the study team to determine eligibility.
- HIV seropositivity.
- Serologic status reflects active hepatitis B or C infection. Participants that are positive for hepatitis B core antibody, hepatitis B surface antigen (HBsAg), or hepatitis C antibody must have a negative polymerase chain reaction (PCR) prior to enrollment. (PCR positive participants will be excluded).
- Participants with uncontrolled intercurrent illness including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, uncontrolled cardiac arrhythmia, pulmonary abnormalities, or psychiatric illness/social situations that would limit compliance with study requirements.
- Pregnant or breastfeeding women are excluded from this study because CAR-T cell therapy may be associated with the potential for teratogenic or abortifacient effects. Because there is an unknown, but potential risk for adverse events in nursing infants secondary to treatment of the mother with CAR-T cells, breastfeeding should be discontinued. These potential risks may also apply to other agents used in this study.
- NOTE: Women of childbearing potential must have a negative serum or urine pregnancy test.
- Participants with currently symptomatic central nervous system (CNS) pathology such as epilepsy, seizure disorders, paresis, aphasia, uncontrolled cerebrovascular disease, severe brain injuries, dementia, and Parkinson's disease OR seizure or stroke within 6 months.
- History of autoimmune disease (e.g., rheumatoid arthritis, systemic lupus erythematosus) with requirement of immunosuppressive medication within 6 months.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of California, San Francisco
San Francisco, California, 94143
About this study
Official title: A Phase 1b Clinical Trial of CRISPR Delivered Anti-BCMA Chimeric Antigen Receptor T Cells for Treatment of Relapsed or Refractory Multiple Myeloma
This phase Ib trial tests the safety, side effects and best dose of clustered regularly interspaced short palindromic repeats (CRISPR) delivered anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR)-T cells (1XX BCMA CAR-T cells) in treating patients with multiple myeloma that has come back after a period of improvement (relapsed) or that has not responded to previous treatment (refractory). Anti-BCMA CAR-T cell therapy is a type of treatment in which a person's T cells (a type of immune system cell) are changed in the laboratory so they will attack cancer cells. T cells are taken from a patient's blood. Then the gene for a special receptor that binds to a certain protein, such as BCMA, on the patient's cancer cells is added to the T cells in the laboratory by a tool called clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9. The special receptor is called a CAR. Large numbers of the CAR-T cells are grown in the laboratory and given to the patient by infusion for treatment of certain cancers. Giving chemotherapy before CAR-T cells may decrease the number of lymphocytes (a type of white blood cells) in the blood and may help the 1XX BCMA CAR-T cells fight the cancer cells. Treatment with 1XX BCMA CAR-T cells may be safe, tolerable, and/or effective in treating patients with relapsed or refractory multiple myeloma (RRMM).
Read the detailed description
PRIMARY OBJECTIVES:
Dose Escalation:
- To evaluate the safety and toxicity of administering Chimeric Antigen Receptor T Cells (CAR-T) cells targeting BCMA to participants with Relapsed or Refractory Multiple Myeloma (RRMM).
II. To determine the maximum tolerated dose (MTD) for anti-BCMA CAR-T cells.
Dose Expansion:
III. Determine whether administering conforming CAR T-cell product targeting BCMA to participants with RRMM increases the overall response rate (ORR) compared with historical data for non-CAR agents per International Myeloma Working Group (IMWG) response criteria.
IV. Determine whether administering conforming CAR T-cell product targeting BCMA to participants with RRMM lowers the Grade 2 or greater neurologic events to <10% in RRMM.
SECONDARY OBJECTIVES:
Dose Expansion:
- To describe the efficacy of conforming CAR-T cell product targeting BCMA in participants with RRMM.
II. To evaluate the feasibility of manufacturing anti-BCMA CAR T-cells locally and ability to produce adequate quantities of vector positive T-cells.
III. To evaluate the safety and toxicity of conforming CAR-T cell product targeting BCMA to participants with RRMM.
EXPLORATORY OBJECTIVES:
- To determine the degree and impact of CAR-T persistence following anti-BCMA CAR-T cell infusion, on clinical outcomes and safety.
II. Describe changes in health-related quality of life (HRQoL) using the European Organization for Research and Treatment of Cancer - Quality of Life C30 questionnaire (EORTC-QLQ-C30).
III. To describe the efficacy of CAR-T cells targeting BCMA in participants with relapsed or refractory BCMA+ RRMM who were treated with product that did not meet one or more pre-specified release criteria (non-conforming product cohort).
OUTLINE:
Participants in both cohorts will undergo leukapheresis, receive lymphodepleting chemotherapy and then receive a single infusion of BCMA CAR-T therapy. After completion of study treatment, participants are followed up at 30, 60 and 90 days, 6 and 12 months, and then yearly for up to 15 years.
- Being tested
- Leukapheresis, Cyclophosphamide, Chimeric Antigen Receptor T cells (CAR-T) Targeting BCMA, Quality of Life (QoL) Questionnaires, Bone Marrow Biopsy
- Keywords
- CAR T, CRISPR
- Medical subject headings
- Multiple Myeloma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LeukapheresisProcedure or surgery
Undergo Leukapheresis
CyclophosphamideDrug
Also called Cycloblastin, Fosfaseron, Genoxal, Cytoxan
Given Intravenously (IV)
Chimeric Antigen Receptor T cells (CAR-T) Targeting BCMABiological
Also called CAR-T Infusion
Given Intravenously (IV)
Quality of Life (QoL) QuestionnairesBehavioural
Ancillary studies
Bone Marrow BiopsyProcedure or surgery
Also called Biopsy
Undergo biopsy
Biospecimen CollectionBiological
Also called Sample collection
Undergo Blood, serum and urine collection
FludarabineDrug
Also called Fluradosa
Given IV
Radiographic imagingProcedure or surgery
Also called Positron Emission Tomography (PET)/Computerized tomography (CT), PET/CT, Magnetic Resonance Imaging (MRI), MRI
Undergo radiographic imaging
| Study group | Role | What they receive |
|---|---|---|
| Dose Escalation Starting Dose: 10 × 10^6 CAR + T cells/ infusion | Experimental | Participants undergo leukapheresis and then receive lymphodepleting chemotherapy with fludarabine IV over 30 minutes and cyclophosphamide IV over 60 minutes on days -5, -4, and -3. Participants also receive 10 × 10^6 BCMA CAR-T cells/ infusion over 5-30 minutes on day 1 in the absence of disease progression or unacceptable toxicity. Additionally, participants undergo PET/CT or MRI at screening and urine and blood sample collection, bone marrow biopsy or aspiration throughout the study. Receives: Procedure: Leukapheresis; Drug: Cyclophosphamide; Biological: Chimeric Antigen Receptor T cells (CAR-T) Targeting BCMA; Behavioral: Quality of Life (QoL) Questionnaires; Procedure: Bone Marrow Biopsy; Biological: Biospecimen Collection; Drug: Fludarabine; Procedure: Radiographic imaging |
| Dose Escalation: Planned Dose: 30 × 10^6 CAR + T cells/ infusion | Experimental | Dependent on the safety profile of the starting dose, participants undergo leukapheresis and then receive lymphodepleting chemotherapy with fludarabine IV over 30 minutes and cyclophosphamide IV over 60 minutes on days -5, -4, and -3. Participants also receive 30 × 10^6 BCMA CAR-T cells/ infusion over 5-30 minutes on day 1 in the absence of disease progression or unacceptable toxicity. Additionally, participants undergo PET/CT or MRI at screening and urine and blood sample collection, bone marrow biopsy or aspiration throughout the study. Receives: Procedure: Leukapheresis; Drug: Cyclophosphamide; Biological: Chimeric Antigen Receptor T cells (CAR-T) Targeting BCMA; Behavioral: Quality of Life (QoL) Questionnaires; Procedure: Bone Marrow Biopsy; Biological: Biospecimen Collection; Drug: Fludarabine; Procedure: Radiographic imaging |
| Expansion: MTD of Anti-BCMA CAR-T | Experimental | Participants undergo leukapheresis and then receive lymphodepleting chemotherapy with fludarabine IV over 30 minutes and cyclophosphamide IV over 60 minutes on days -5, -4, and -3. Participants also receive the MTD of BCMA CAR-T cells/ infusion established in the dose escalation phase over 5-30 minutes on day 1 in the absence of disease progression or unacceptable toxicity. Additionally, participants undergo PET/CT or MRI at screening and urine and blood sample collection, bone marrow biopsy or aspiration throughout the study. Receives: Procedure: Leukapheresis; Drug: Cyclophosphamide; Biological: Chimeric Antigen Receptor T cells (CAR-T) Targeting BCMA; Behavioral: Quality of Life (QoL) Questionnaires; Procedure: Bone Marrow Biopsy; Biological: Biospecimen Collection; Drug: Fludarabine; Procedure: Radiographic imaging |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of participants with treatment-emergent adverse events
Proportion of participants with treatment-emergent adverse events of CAR-T in RRMM as graded by National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE version 5.0), revised Cytokine Release Syndrome (CRS) grading criteria (for CRS grading), and the American Society for Transplantation and Cell Transplantation (ASTCT) Immune effector cell-associated neurotoxicity syndrome (ICANS) Consensus Grading for Adults (for neurotoxicity grading).
Measured: Up to 12 months following CAR-T infusion
Proportion of participants who experience dose-limiting toxicity (DLT) (Dose Escalation)
The DLT evaluable analysis set includes all participants in the dose-finding part of the study who have received the target doses of anti-BCMA CAR-T cell product, and who have either experienced a DLT or were followed for the full DLT evaluation period. The trial observation period for dose limiting toxicities will conclude at day 30 (approximately 4 weeks after CAR-T cell infusion)
Measured: Up to 30 days
Maximum Tolerated Dose (MTD) (Dose Escalation)
The DLT evaluable analysis set includes all participants in the dose-finding part of the study who have received the target doses of anti-BCMA CAR-T cell product, and who have either experienced a DLT or were followed for the full DLT evaluation period (within 28 days following infusion of CAR-T cells targeting BCMA). The MTD is defined as the dose level immediately below that in which ≥ 2/6 participants experience a DLT.
Measured: Up to 28 days
Best Overall Response Rate (BORR) (Dose expansion + MTD dose escalation cohort)
ORR is defined as the proportion of participants with stringent complete response (sCR), complete response (CR), very good partial response (VGPR), partial response (PR), as per International Myeloma Working Group (IMWG) criteria, as best overall response over the total population. ORR will be reported as proportion with 90% binomial confidence interval for the expansion cohort including the patients on MTD in the dose escalation cohort(s).
Measured: Up to 12 months following CAR-T infusion
Proportion of participants with of severe neurologic events (Dose Expansion)
Incidence of severe neurologic events defined as grade 2 or higher will be reported as a proportion of the total participants by group.
Measured: From initiation of study treatment to 12 months following CAR-T infusion, approximately 13 months total
Secondary outcomes (9)
Overall Response Rate (ORR) - Long term
The ORR from initiation of study treatment until disease progression or death from any cause will be reported as the proportion of participants with stringent complete response (sCR), complete response (CR), very good partial response (VGPR), partial response (PR), as per International Myeloma Working Group (IMWG) criteria. ORR will be reported as proportion with 90% binomial confidence interval for each cohort
Measured: Up to 15 years
Median Duration of Response (DoR) - Beginning of response
The median duration of response measured for responders, from the documented beginning of response ( sCR, CR, PR or VGPR) (as per IMWG criteria) until progression or death from any cause along with 95% confidence intervals.
Measured: Up to 15 years
Median Progression-free Survival from Study Entry (PFS-SE)
Progression-free Survival (PFS) is defined as the time from entry onto study until multiple myeloma progression or death from any cause. Where there is missing information, censoring of the data may be defined as the last date at which progression status was adequately assessed or the first date of unscheduled new anti-MM treatment and will be analyzed by Kaplan-Meier method for the expansion cohort including the participants on MTD in the dose escalation cohort.
Measured: up to 15 years
Median DoR - First documented response
The median duration of response measured for responders is defined as the time from first response until multiple myeloma (MM) progression or death from any cause.
Measured: up to 15 years
Median Overall Survival (OS)
The median overall survival is defined as the amount of time from study treatment until death and will be analyzed by Kaplan-Meier method for the expansion cohort including the participants on MTD in the dose escalation cohort.
Measured: up to 15 years
Rates of MRD (-) and sustained MRD (-)
The rates of participants determined to be MRD negative (MRD-) or positive for MRD (MRD+) will be reported as a percentage of participants.
Measured: Up to 15 years
Proportion of participants who produced adequate quantities of product for infusion
To evaluate the feasibility of manufacturing anti-BCMA CAR T-cells locally and ability to produce adequate quantities of vector positive T-cells, the proportion of participants for whom BCMA CAR T-cell therapy is manufactured and meets pre-specified release criteria will be reported
Measured: From initiation of BCMA CAR T-cell manufacturing to end of infusion, Up to 1 month
Proportion of participants who complete Car-T infusion study treatment
To evaluate the feasibility of manufacturing anti-BCMA CAR T-cells locally and ability to produce adequate quantities of vector positive T-cells, the proportion of participants successfully completed the BCMA CAR T-cell therapy is manufactured and meets pre-specified release criteria will be reported
Measured: From initiation of BCMA CAR T-cell manufacturing to end of infusion, Up to 1 month
Proportion of participants with treatment- emergent adverse events - Long Term
The proportion of participants with treatment-emergent adverse events of CAR-T in RRMM, as graded by National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE version 5.0), revised CRS grading criteria (for CRS grading), and ASTCT ICANS Consensus Grading for Adults (for neurotoxicity grading) From initiation of BCMA CAR T-cell manufacturing to end of long-term follow-up
Measured: Up to 15 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2026
- Study start28 Feb 2026
- Primary completion25 May 2028 (estimated)
- Study completion25 May 2043 (estimated)
Why it stopped: Safety Review
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multiple MyelomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Thomas Martin, MD
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Thomas Martin, MD, University of California, San Francisco
- Investigators
- Anupama Kumar, MD, Principal investigator, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07340853
- Sponsor's protocol ID
- 25707
- Other IDs
- NCI-2025-08425
- Status last verified
- Jun 2026
- First submitted
- 5 Jan 2026
- Last updated
- 11 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b Clinical Trial of CRISPR Delivered Anti-BCMA Chimeric Antigen Receptor T Cells for Treatment of Relapsed or Refractory Multiple Myeloma
- ClinicalTrials.gov ID
- NCT07340853
- Acronym / sponsor ID
- 25707
- Keywords
- CAR T, CRISPR
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- SuspendedReason stopped: Safety Review
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Thomas Martin, MDUniversity, hospital or other
- Study start
- 28 Feb 2026 (actual)
- Primary completion
- 25 May 2028 (estimated)
- Study completion
- 25 May 2043 (estimated)
- First posted
- 14 Jan 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase Ib trial tests the safety, side effects and best dose of clustered regularly interspaced short palindromic repeats (CRISPR) delivered anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR)-T cells (1XX BCMA CAR-T cells) in treating patients with multiple myeloma that has come back after a period of improvement (relapsed) or that has not responded to previous treatment (refractory). Anti-BCMA CAR-T cell therapy is a type of treatment in which a person's T cells (a type of immune system cell) are changed in the laboratory so they will attack cancer cells. T cells are taken from a patient's blood. Then the gene for a special receptor that binds to a certain protein, such as BCMA, on the patient's cancer cells is added to the T cells in the laboratory by a tool called clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9. The special receptor is called a CAR. Large numbers of the CAR-T cells are grown in the laboratory and given to the patient by infusion for treatment of certain cancers. Giving chemotherapy before CAR-T cells may decrease the number of lymphocytes (a type of white blood cells) in the blood and may help the 1XX BCMA CAR-T cells fight the cancer cells. Treatment with 1XX BCMA CAR-T cells may be safe, tolerable, and/or effective in treating patients with relapsed or refractory multiple myeloma (RRMM).
Detailed description
PRIMARY OBJECTIVES:
Dose Escalation:
- To evaluate the safety and toxicity of administering Chimeric Antigen Receptor T Cells (CAR-T) cells targeting BCMA to participants with Relapsed or Refractory Multiple Myeloma (RRMM).
II. To determine the maximum tolerated dose (MTD) for anti-BCMA CAR-T cells.
Dose Expansion:
III. Determine whether administering conforming CAR T-cell product targeting BCMA to participants with RRMM increases the overall response rate (ORR) compared with historical data for non-CAR agents per International Myeloma Working Group (IMWG) response criteria.
IV. Determine whether administering conforming CAR T-cell product targeting BCMA to participants with RRMM lowers the Grade 2 or greater neurologic events to <10% in RRMM.
SECONDARY OBJECTIVES:
Dose Expansion:
- To describe the efficacy of conforming CAR-T cell product targeting BCMA in participants with RRMM.
II. To evaluate the feasibility of manufacturing anti-BCMA CAR T-cells locally and ability to produce adequate quantities of vector positive T-cells.
III. To evaluate the safety and toxicity of conforming CAR-T cell product targeting BCMA to participants with RRMM.
EXPLORATORY OBJECTIVES:
- To determine the degree and impact of CAR-T persistence following anti-BCMA CAR-T cell infusion, on clinical outcomes and safety.
II. Describe changes in health-related quality of life (HRQoL) using the European Organization for Research and Treatment of Cancer - Quality of Life C30 questionnaire (EORTC-QLQ-C30).
III. To describe the efficacy of CAR-T cells targeting BCMA in participants with relapsed or refractory BCMA+ RRMM who were treated with product that did not meet one or more pre-specified release criteria (non-conforming product cohort).
OUTLINE:
Participants in both cohorts will undergo leukapheresis, receive lymphodepleting chemotherapy and then receive a single infusion of BCMA CAR-T therapy. After completion of study treatment, participants are followed up at 30, 60 and 90 days, 6 and 12 months, and then yearly for up to 15 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Dose Escalation Starting Dose: 10 × 10^6 CAR + T cells/ infusion | Experimental | Procedure: Leukapheresis, Drug: Cyclophosphamide, Biological: Chimeric Antigen Receptor T cells (CAR-T) Targeting BCMA, Behavioral: Quality of Life (QoL) Questionnaires, Procedure: Bone Marrow Biopsy, Biological: Biospecimen Collection, Drug: Fludarabine, Procedure: Radiographic imagingParticipants undergo leukapheresis and then receive lymphodepleting chemotherapy with fludarabine IV over 30 minutes and cyclophosphamide IV over 60 minutes on days -5, -4, and -3. Participants also receive 10 × 10^6 BCMA CAR-T cells/ infusion over 5-30 minutes on day 1 in the absence of disease progression or unacceptable toxicity. Additionally, participants undergo PET/CT or MRI at screening and urine and blood sample collection, bone marrow biopsy or aspiration throughout the study. |
| Dose Escalation: Planned Dose: 30 × 10^6 CAR + T cells/ infusion | Experimental | Procedure: Leukapheresis, Drug: Cyclophosphamide, Biological: Chimeric Antigen Receptor T cells (CAR-T) Targeting BCMA, Behavioral: Quality of Life (QoL) Questionnaires, Procedure: Bone Marrow Biopsy, Biological: Biospecimen Collection, Drug: Fludarabine, Procedure: Radiographic imagingDependent on the safety profile of the starting dose, participants undergo leukapheresis and then receive lymphodepleting chemotherapy with fludarabine IV over 30 minutes and cyclophosphamide IV over 60 minutes on days -5, -4, and -3. Participants also receive 30 × 10^6 BCMA CAR-T cells/ infusion over 5-30 minutes on day 1 in the absence of disease progression or unacceptable toxicity. Additionally, participants undergo PET/CT or MRI at screening and urine and blood sample collection, bone marrow biopsy or aspiration throughout the study. |
| Expansion: MTD of Anti-BCMA CAR-T | Experimental | Procedure: Leukapheresis, Drug: Cyclophosphamide, Biological: Chimeric Antigen Receptor T cells (CAR-T) Targeting BCMA, Behavioral: Quality of Life (QoL) Questionnaires, Procedure: Bone Marrow Biopsy, Biological: Biospecimen Collection, Drug: Fludarabine, Procedure: Radiographic imagingParticipants undergo leukapheresis and then receive lymphodepleting chemotherapy with fludarabine IV over 30 minutes and cyclophosphamide IV over 60 minutes on days -5, -4, and -3. Participants also receive the MTD of BCMA CAR-T cells/ infusion established in the dose escalation phase over 5-30 minutes on day 1 in the absence of disease progression or unacceptable toxicity. Additionally, participants undergo PET/CT or MRI at screening and urine and blood sample collection, bone marrow biopsy or aspiration throughout the study. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (21 listed)
- Voluntarily sign informed consent form.
- Age ≥18 years.
- Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1.
- Diagnosis of multiple myeloma (per IMWG criteria) with relapsed or refractory disease and has received at least 3 prior lines of therapy including proteasome inhibitor immunomodulatory therapy, and anti-Cluster of differentiation 38 (CD38) antibody therapy.
- Participants may have received BCMA targeted therapy and must be at least 6 months from last BCMA therapy.
Key exclusion criteria (18 listed)
- Autologous transplant within 12 weeks of planned CAR-T cell infusion.
- Prior antitumor therapy as follows, prior to apheresis:
- Toxicity from previous anticancer therapy must resolve to baseline levels or to Grade 1 or less except for alopecia, peripheral neuropathy and baseline hematologic toxicity that otherwise meets inclusion.
- Active CNS multiple myeloma, plasma cell leukemia, primary AL amyloidosis or POEMS syndrome.
- Active other malignancy, other than non-melanoma skin cancer, carcinoma in situ (e.g., cervix, bladder, or breast). Any fully treated malignancies or indolent, clinically insignificant malignancies can be discussed among the study team to determine eligibility.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LeukapheresisUndergo Leukapheresis | Procedure or surgery | Dose Escalation Starting Dose: 10 × 10^6 CAR + T cells/ infusion, Dose Escalation: Planned Dose: 30 × 10^6 CAR + T cells/ infusion, Expansion: MTD of Anti-BCMA CAR-T |
| CyclophosphamideAlso: Cycloblastin, Fosfaseron, Genoxal, CytoxanGiven Intravenously (IV) | Drug | Dose Escalation Starting Dose: 10 × 10^6 CAR + T cells/ infusion, Dose Escalation: Planned Dose: 30 × 10^6 CAR + T cells/ infusion, Expansion: MTD of Anti-BCMA CAR-T |
| Chimeric Antigen Receptor T cells (CAR-T) Targeting BCMAAlso: CAR-T InfusionGiven Intravenously (IV) | Biological | Dose Escalation Starting Dose: 10 × 10^6 CAR + T cells/ infusion, Dose Escalation: Planned Dose: 30 × 10^6 CAR + T cells/ infusion, Expansion: MTD of Anti-BCMA CAR-T |
| Quality of Life (QoL) QuestionnairesAncillary studies | Behavioural | Dose Escalation Starting Dose: 10 × 10^6 CAR + T cells/ infusion, Dose Escalation: Planned Dose: 30 × 10^6 CAR + T cells/ infusion, Expansion: MTD of Anti-BCMA CAR-T |
| Bone Marrow BiopsyAlso: BiopsyUndergo biopsy | Procedure or surgery | Dose Escalation Starting Dose: 10 × 10^6 CAR + T cells/ infusion, Dose Escalation: Planned Dose: 30 × 10^6 CAR + T cells/ infusion, Expansion: MTD of Anti-BCMA CAR-T |
| Biospecimen CollectionAlso: Sample collectionUndergo Blood, serum and urine collection | Biological | Dose Escalation Starting Dose: 10 × 10^6 CAR + T cells/ infusion, Dose Escalation: Planned Dose: 30 × 10^6 CAR + T cells/ infusion, Expansion: MTD of Anti-BCMA CAR-T |
| FludarabineAlso: FluradosaGiven IV | Drug | Dose Escalation Starting Dose: 10 × 10^6 CAR + T cells/ infusion, Dose Escalation: Planned Dose: 30 × 10^6 CAR + T cells/ infusion, Expansion: MTD of Anti-BCMA CAR-T |
| Radiographic imagingAlso: Positron Emission Tomography (PET)/Computerized tomography (CT), PET/CT, Magnetic Resonance Imaging (MRI), MRIUndergo radiographic imaging | Procedure or surgery | Dose Escalation Starting Dose: 10 × 10^6 CAR + T cells/ infusion, Dose Escalation: Planned Dose: 30 × 10^6 CAR + T cells/ infusion, Expansion: MTD of Anti-BCMA CAR-T |
Primary outcome
Proportion of participants with treatment-emergent adverse events
Time frame: Up to 12 months following CAR-T infusion
Proportion of participants with treatment-emergent adverse events of CAR-T in RRMM as graded by National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE version 5.0), revised Cytokine Release Syndrome (CRS) grading criteria (for CRS grading), and the American Society for Transplantation and Cell Transplantation (ASTCT) Immune effector cell-associated neurotoxicity syndrome (ICANS) Consensus Grading for Adults (for neurotoxicity grading).
Proportion of participants who experience dose-limiting toxicity (DLT) (Dose Escalation)
Time frame: Up to 30 days
The DLT evaluable analysis set includes all participants in the dose-finding part of the study who have received the target doses of anti-BCMA CAR-T cell product, and who have either experienced a DLT or were followed for the full DLT evaluation period. The trial observation period for dose limiting toxicities will conclude at day 30 (approximately 4 weeks after CAR-T cell infusion)
Maximum Tolerated Dose (MTD) (Dose Escalation)
Time frame: Up to 28 days
The DLT evaluable analysis set includes all participants in the dose-finding part of the study who have received the target doses of anti-BCMA CAR-T cell product, and who have either experienced a DLT or were followed for the full DLT evaluation period (within 28 days following infusion of CAR-T cells targeting BCMA). The MTD is defined as the dose level immediately below that in which ≥ 2/6 participants experience a DLT.
Best Overall Response Rate (BORR) (Dose expansion + MTD dose escalation cohort)
Time frame: Up to 12 months following CAR-T infusion
ORR is defined as the proportion of participants with stringent complete response (sCR), complete response (CR), very good partial response (VGPR), partial response (PR), as per International Myeloma Working Group (IMWG) criteria, as best overall response over the total population. ORR will be reported as proportion with 90% binomial confidence interval for the expansion cohort including the patients on MTD in the dose escalation cohort(s).
Proportion of participants with of severe neurologic events (Dose Expansion)
Time frame: From initiation of study treatment to 12 months following CAR-T infusion, approximately 13 months total
Incidence of severe neurologic events defined as grade 2 or higher will be reported as a proportion of the total participants by group.
9 secondary outcomes
Overall Response Rate (ORR) - Long term
Time frame: Up to 15 years
Median Duration of Response (DoR) - Beginning of response
Time frame: Up to 15 years
Median Progression-free Survival from Study Entry (PFS-SE)
Time frame: up to 15 years
Median DoR - First documented response
Time frame: up to 15 years
Median Overall Survival (OS)
Time frame: up to 15 years
Rates of MRD (-) and sustained MRD (-)
Time frame: Up to 15 years
Proportion of participants who produced adequate quantities of product for infusion
Time frame: From initiation of BCMA CAR T-cell manufacturing to end of infusion, Up to 1 month
Proportion of participants who complete Car-T infusion study treatment
Time frame: From initiation of BCMA CAR T-cell manufacturing to end of infusion, Up to 1 month
Proportion of participants with treatment- emergent adverse events - Long Term
Time frame: Up to 15 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jun 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Anupama Kumar, MD principal investigator
University of California, San Francisco
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Leukapheresis, Cyclophosphamide and Chimeric Antigen Receptor T cells (CAR-T) Targeting BCMA in people with Multiple Myeloma and Recurrent Multiple Myeloma, run by Thomas Martin, MD at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 21 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: paused, as of the sponsor's last update on 11 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of participants with treatment-emergent adverse events (time frame: Up to 12 months following CAR-T infusion). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jun 2026.