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Refractory Multiple MyelomaPhase 1NCT05577000

Anti-BCMA Chimeric Antigen Receptor T Cells for Relapsed or Refractory Multiple Myeloma

A Phase 1 study testing Manufactured Anti-BCMA CAR-T cells, Fludarabine and Cyclophosphamide in people with Refractory Multiple Myeloma and Relapsed Multiple Myeloma, run by Thomas Martin, MD at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial summary

This is an open-label study to determine the safety of anti-B-cell maturation antigen (BCMA) Chimeric antigen receptor T-cell (CAR T) therapy in participants with Relapsed or Refractory Multiple Myeloma (RRMM). Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Manufactured Anti-BCMA CAR-T cells, Fludarabine and Cyclophosphamide
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
5 participants
Where
1 siteUnited States
Study dates
18 Oct 2021to 31 Aug 2038
Phase
Phase 1
Are there results?
Not yet: the study is under way

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.
  • >=18 years of age at the time of signing informed consent.
  • Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1.
  • Diagnosis of MM with relapsed or refractory disease and have had at least 3 different prior lines of therapy including proteasome inhibitor (PI) (e.g., bortezomib or carfilzomib) immunomodulatory therapy (IMiD) (e.g., lenalidomide or pomalidomide), and anti-CD38 antibody therapy.
  • Participants must have measurable disease, including at least one of the criteria below:
  • Serum M-protein greater or equal to 0.5 g/dL.
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  • Urine M-protein greater or equal to 200 mg/24 hours (h).
  • Serum free light chain (FLC) assay: involved FLC level of >= 100 mg/L.
  • Adequate organ function, defined as:
  • Hemoglobulin >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).
  • Alanine aminotransferase (ALT)/aspartate aminotransferase (AST) =< 3 x institutional upper limit of normal (ULN).
  • Total bilirubin =< 1.5 mg/dl x institutional ULN, except with Gilbert's syndrome.
  • Serum creatinine clearance (CrCl) >= 45 mL/min using Cockcroft-Gault formula.
  • Adequate cardiac function, defined as left ventricular ejection fraction (LVEF) >= 40% as assessed by echocardiogram or multiple uptake gated acquisition (MUGA).
  • Women of childbearing potential (defined as all women physiologically capable of becoming pregnant) 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.

Who cannot join

  • Autologous transplant within 6 weeks of planned CAR-T cell infusion.
  • 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 reflecting active hepatitis B or C infection. Patients 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 patients 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.
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  • Patients 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.
  • History of autoimmune disease (e.g., rheumatoid arthritis, systemic lupus erythematosus) with requirement of immunosuppressive medication within 6 months.
  • Eligibility for Infusion of Investigational Product:
  • Participants will undergo an evaluation of eligibility on day -1 or 1 prior to infusion of anti-BCMA CAR-T cell product. This eligibility criteria will include the inclusion and exclusion criteria required for enrollment with the following exceptions and additions:
  • Hematologic function parameters will not be included as a pre-infusion eligibility criterion (because lymphodepletive chemotherapy is expected to cause pancytopenia).
  • Laboratory result abnormalities that are considered not clinically significant by the principal investigator AND are not the result of a demonstrated active infection or an active central nervous system condition.
  • Use of anti-multiple myeloma therapy, including systemic corticosteroids within 14 days prior to lymphodepletive chemotherapy.
  • Neurologic symptoms suggestive of an active central nervous system condition.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • University of California, San Francisco

    San Francisco, California, 94143

Other trials in United States

About this study

Official title: A Phase 1b Clinical Trial of Anti-BCMA Chimeric Antigen Receptor T Cells for Treatment of Relapsed or Refractory Multiple Myeloma

This is an open-label study to determine the safety of anti-B-cell maturation antigen (BCMA) Chimeric antigen receptor T-cell (CAR T) therapy in participants with Relapsed or Refractory Multiple Myeloma (RRMM).

Read the detailed description

PRIMARY OBJECTIVE:

  1. To evaluate the safety of administering chimeric antigen receptor (CAR)-T cells targeting BCMA to participants with RRMM (Dose Escalation).
  2. To determine the maximum tolerated dose (MTD) for anti-BCMA CAR-T cells (Dose Escalation).
  3. Determine whether administering chimeric antigen receptor T cells targeting BCMA to participants with RRMM increases the overall response rate (ORR) in RRMM compared with historical data for non-CAR agents per International Myeloma Working Group (IMWG) response criteria (Dose Expansion).

SECONDARY OBJECTIVES:

Dose Expansion Only:

  1. To describe the efficacy of CAR-T cells targeting BCMA in participants with RRMM defined by ORR using IMWG criteria.
  2. To evaluate the feasibility of manufacturing anti-BCMA CAR-T cells locally and ability to produce adequate quantities of vector positive T-cells.
  3. To evaluate the safety and toxicity of CAR-T cells targeting BCMA to participants with RRMM.

OUTLINE:

Participants will be enrolled sequentially to each dose level dependent on analysis of dose-limiting toxicities at the previous dose level. A dose expansion will occur at the maximum tolerated dose (MTD). Participants will undergo apheresis with collection of autologous peripheral blood mononuclear cells that will be used to generate CAR-T cells. After successful generation of the anti-BCMA CAR-T cells drug product (DP), participants will undergo lymphodepleting chemotherapy with fludarabine (and cyclophosphamide). Participants will undergo an additional evaluation of eligibility on Day -1 or 1 prior to infusion of anti-BCMA CAR-T cell product. A single infusion of anti-BCMA CAR-T cells at the starting dose will be given on Day 1. Following treatment with DP, participants will be followed up at 12 months and annually for up to 15 years.

Being tested
Manufactured Anti-BCMA CAR-T cells, Fludarabine, Cyclophosphamide
Keywords
CAR-T Therapies
Medical subject headings
Multiple Myeloma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Manufactured Anti-BCMA CAR-T cellsBiological

    Also called Anti-BCMA CAR-T cells

    Given IV

  • FludarabineDrug

    Also called Fludara

    Given IV

  • CyclophosphamideDrug

    Also called Cytoxan

    Given IV

Study groups in NCT05577000
Study groupRoleWhat they receive
Dose Escalation (150 x 10^6 CAR + T cells/ infusion)ExperimentalParticipants will undergo apheresis with collection of autologous peripheral blood mononuclear cells that will be used to generate CAR-T cells. After successful generation of the anti-BCMA CAR-T cells, participants will undergo lymphodepleting chemotherapy with fludarabine and cyclophosphamide followed by 2-5 days of rest. A single infusion of anti-BCMA CAR-T cells at the starting dose of 150 x 10^6 flat dose will then be given on Day 1.
Receives: Biological: Manufactured Anti-BCMA CAR-T cells; Drug: Fludarabine; Drug: Cyclophosphamide
Dose Escalation (450 x 10^6 CAR + T cells/ infusion)ExperimentalParticipants will undergo apheresis with collection of autologous peripheral blood mononuclear cells that will be used to generate CAR-T cells. After successful generation of the anti-BCMA CAR-T cells, and no dose limiting toxicities were reported for the previous dose level, participants will undergo lymphodepleting chemotherapy with fludarabine and cyclophosphamide followed by 2-5 days of rest. A single infusion of anti-BCMA CAR-T cells at the next highest dose of 450 x 10^6 flat dose will then be given on Day 1.
Receives: Biological: Manufactured Anti-BCMA CAR-T cells; Drug: Fludarabine; Drug: Cyclophosphamide
Dose Escalation (600 x 10^6 CAR + T cells/ infusion)ExperimentalParticipants will undergo apheresis with collection of autologous peripheral blood mononuclear cells that will be used to generate CAR-T cells. After successful generation of the anti-BCMA CAR-T cells, and no dose limiting toxicities were reported for the previous dose level, participants will undergo lymphodepleting chemotherapy with fludarabine and cyclophosphamide followed by 2-5 days of rest. A single infusion of anti-BCMA CAR-T cells at the next highest dose of 600 x 10^6 flat dose will then be given on Day 1.
Receives: Biological: Manufactured Anti-BCMA CAR-T cells; Drug: Fludarabine; Drug: Cyclophosphamide
Dose Expansion: Maximum Tolerated Dose (MTD)ExperimentalParticipants will undergo apheresis with collection of autologous peripheral blood mononuclear cells that will be used to generate CAR-T cells. After successful generation of the anti-BCMA CAR-T cells, and no dose limiting toxicities were reported for the previous dose level, participants will undergo lymphodepleting chemotherapy with fludarabine and cyclophosphamide followed by 2-5 days of rest. A single infusion of anti-BCMA CAR-T cells at the MTD will then be given on Day 1.
Receives: Biological: Manufactured Anti-BCMA CAR-T cells; Drug: Fludarabine; Drug: Cyclophosphamide

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Proportion of participants with treatment-emergent adverse events (AE) (Dose Escalation)

    Proportion of participants with treatment-emergent adverse events of CAR-T 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), and American Society of Transplantation and Cellular Therapy (ASTCT) Immune Effector Cell Associated Neurotoxicity (ICANS) Consensus Grading for Adults (for neurotoxicity grading). Analyses will be performed for all participants having received at least one dose of study drug and employ a modified criteria for hematologic adverse events.

    Measured: From initiation of study treatment (day 1) to 29 days following CAR-T infusion

  2. Proportion of participants who experience a 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 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 and will be used to determine the recommended Phase 2 dose for future studies.

    Measured: From initiation of study treatment (day 1) to 29 days following CAR-T infusion

  3. Overall Response Rate (ORR) (Dose Expansion)

    Overall response rate includes participants with a demonstrated Stringent Complete Response (sCR), Complete Response (CR), Partial Response (PR), or Very Good Partial Response (VGPR) per International Myeloma Working Group (IMWG) criteria reported as proportion with 90% binomial confidence interval for the expansion cohort including the patients on MTD in the dose escalation cohort.

    Measured: Up to 12 months following CAR- T infusion

Secondary outcomes (9)

  1. Overall Response Rate (ORR)

    Overall response for all participants who were treated with conforming product: (1) at the protocol assigned dose (2) participants treated at a lower than protocol assigned dose, and measured using IMWG criteria from the initiation of study treatment to the time of progression per IMWG criteria. Partial, and complete response rates will be reported as proportions with 90% binomial confidence intervals.

    Measured: Up to 15 years

  2. Duration of response

    This is measured, only in responders who were treated with conforming product: (1) at the protocol -assigned dose and (2) participants treated at a lower than protocol assigned dose from the documented beginning of response (sCR, CR, PR or VGPR) to the time of progression per IMWG criteria.

    Measured: Up to 12 months following CAR- T infusion

  3. Progression-free Survival (PFS)

    PFS is defined as the time from entry onto study until MM progression (by IMWG criteria) or death from any cause. PFS reflects tumor growth and, therefore, occurs prior to the endpoint of overall survival. 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.

    Measured: Up to 15 years

  4. Overall Survival

    Overall Survival is defined as the time from entry onto study until multiple myeloma progression or death from any cause and will be analyzed by Kaplan-Meier method.

    Measured: Up to 15 years

  5. Percentage of participants with minimal residual disease, negative (MRD-)

    The percentage of participants who obtain an MRD- disease will be reported for all participants treated with conforming product (1) at the protocol -assigned dose and (2) participants treated at a lower than protocol assigned dose (conforming product low-dose).

    Measured: Up to 15 years

  6. Percentage of participants with sustained MRD-

    The percentage of participants with MRD- disease status sustained over time will be reported for all participants treated with conforming product (1) at the protocol -assigned dose and (2) participants treated at a lower than protocol assigned dose (conforming product low-dose).

    Measured: Up to 15 years

  7. Proportion of participants for whom BCMA CAR T-cell therapy is manufactured successfully

    Participants who have anti-BCMA CAR T-cells manufactured locally and are able to meet pre-specified release criteria.

    Measured: From initiation of CAR T-cell manufacturing to end of infusion, up to 21 days

  8. Proportion of participants who complete study treatment

    Participants who have anti-BCMA CAR T-cells successfully manufactured locally and are able to produce adequate quantities of vector positive T-cells which meet pre-specified release criteria which are then infused with the drug product.

    Measured: From initiation of CAR T-cell manufacturing to end of infusion, up to 21 days

  9. Proportion of participants with treatment-emergent adverse events (AE)

    Proportion of participants with treatment-emergent adverse events of CAR-T 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), and American Society of Transplantation and Cellular Therapy (ASTCT) Immune Effector Cell Associated Neurotoxicity (ICANS) Consensus Grading for Adults (for neurotoxicity). Analyses will be performed for all participants having received at least one dose of study drug. The study will also employ a modified criteria for hematologic adverse events.

    Measured: From initiation of CAR T-cell manufacturing to end of long-term follow-up, approximately 15 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted13 Oct 2022
  2. Study start18 Oct 2021
  3. Primary completion30 Nov 2025
  4. Study completion31 Aug 2038 (estimated)

Resources

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
5 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05577000
Sponsor's protocol ID
22702
Other IDs
NCI-2022-08957
Status last verified
Feb 2026
First submitted
8 Oct 2022
Last updated
20 Feb 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Manufactured Anti-BCMA CAR-T cells, Fludarabine and Cyclophosphamide in people with Refractory Multiple Myeloma and Relapsed 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 18 inclusion and 14 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: under way, not looking for participants, as of the sponsor's last update on 20 Feb 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?

This study is still under way. Results are not available in the source record yet.

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 (AE) (Dose Escalation) (time frame: From initiation of study treatment (day 1) to 29 days following CAR-T infusion). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05577000.

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 20 Feb 2026.

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