Multiple MyelomaPhase 1NCT03549442
Up-front CART-BCMA With or Without huCART19 in High-risk Multiple Myeloma
A Phase 1 study testing BCMA CART + huCART19, CART BCMA or CART BCMA + huCART19 and Single-dose infusion of CART BCMA or CART BCMA + huCART19 in people with Multiple Myeloma, run by University of Pennsylvania at 1 site in United States.
Trial summary
This is an open-label, phase 1 study to assess the safety, pharmacodynamics, and anti-myeloma effects of autologous T cells expressing BCMA (B-cell maturation antigen)-specific chimeric antigen receptors with tandem TCRζ and 4-1BB (TCRζ /4-1BB) costimulatory domains (referred to as "CART-BCMA"), with or without… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Multiple Myeloma
- What is being tested?
- BCMA CART + huCART19, CART BCMA or CART BCMA + huCART19 and Single-dose infusion of CART BCMA or CART BCMA + huCART19
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteUnited States
- Study dates
- 9 May 2018to Mar 2036
- 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
- Subjects must have a diagnosis of multiple myeloma according to IMWG 2014 criteria106 with any of the following high-risk features. Subjects in the Phase A Expansion are not required to have any high-risk features.
- Beta-2-microglobulin ≥ 5.5 mg/L and LDH greater than upper limit of normal. Note: subjects in whom LDH and/or Beta-2-microglobulin were not measured prior to initiation of systemic therapy may qualify based on measurements obtained after initiation of systemic therapy.
- High-risk FISH features: at least one of the following [deletion 17p, t(14;16), t(14;20), t(4;14)] in conjunction with Beta-2-microglobulin ≥ 5.5 mg/L (i.e., revised ISS stage 3). Note: subjects in whom Beta-2-microglobulin was not measured prior to initiation of systemic therapy may qualify based on measurements obtained after initiation of systemic therapy.
- Metaphase karyotype with >3 structural abnormalities except hyperdiploidy
- Plasma cell leukemia (>20% plasma cells in peripheral blood) at any time prior to physician-investigator confirmation of eligibility.
- Failure to achieve partial response or better (by IMWG 2016 criteria1) to initial therapy with an "imid/PI" combination (thalidomide, lenalidomide, or pomalidomide in combination with bortezomib, ixazomib, or carfilzomib).
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- Early progression on first-line therapy, defined as progression (according to IMWG 2016 criteria1)
- Within one year of starting first-line therapy with an "imid/PI"combination ii. Within six months of completing first line therapy with an "imid/PI"combination (i.e. a patient who receives an "imid/PI" combination, transitions to observation or maintenance therapy, and progresses within six months of this transition) iii. Within one year of a high-dose melphalan and autologous stem cell transplantation (Phase A subjects only)
- Subjects must meet the following criteria with respect to prior myeloma therapy:
- Phase A and Phase A expansion:
- Subjects must meet the following criteria with respect to prior multiple myeloma therapy: i. have disease that has relapsed after or has been refractory to at least two regimens, including a proteasome inhibitor and thalidomide analog (thalidomide, lenalidomide, pomalidomide), OR ii. have disease that has relapsed after or has been refractory to one prior regimen if their prior/current therapy collectively has included all of the following: an "imid/PI" combination, pomalidomide, lenalidomide, daratumumab, and carfilzomib.
- Note: Refractoriness is defined as disease progression on-therapy or within 60 days of stopping therapy.
- Subjects must have achieved at least a minimal response (as defined by IMWG 2016 criteria1) to their current regimen.
- Subjects must not have received prior treatment with anti-BCMA cellular therapy. Subjects may have received treatment with other BCMA-directed agents (e.g., anti-BCMA antibody-drug conjugates or bispecific antibodies).
- Phases B and C:
- Subjects must be in their first line of multiple myeloma therapy, with the following exception: subjects who have advanced to second-line therapy due to disease progression during first-line therapy are eligible if such progression occurred within six months of beginning first-line therapy. Lines of therapy are defined by IMWG 2016 criteria1.
- Subjects must not have received cytotoxic chemotherapy (e.g., doxorubicin, cyclophosphamide, etoposide, cisplatin) with the following exceptions:
- Low-dose weekly cyclophosphamide (≤500 mg/m2/week) ii. Continuous infusion cyclophosphamide, if limited to a single cycle. c. Subjects must not have undergone autologous or allogeneic stem cell transplantation.
- Subjects must have initiated systemic therapy for multiple myeloma ≤1 year prior to physician-investigator confirmation of eligibility.
- Subjects must have achieved at least a minimal response (as defined by IMWG 2016 criteria1) to their overall systemic therapy for multiple myeloma and be clinically stable on their current regimen in the judgement of the investigator.
- Subjects must not have achieved a stringent complete response according to IMWG 2016 criteria1 at time of physician-investigator confirmation of eligibility unless clonal plasma cells are detectable in bone marrow by flow cytometry (I.e., subjects in stringent complete response are eligible if minimal residual disease can be documented by bone marrow flow cytometry) or if residual disease is detectable by imaging such as PET/CT, CT, or MRI.
- Subjects must have signed written, informed consent.
- Subjects must be ≥ 18 years of age.
- Subjects must have adequate vital organ function:
- Serum creatinine ≤ 2.5 or creatinine clearance ≥30 ml/min (measured or estimated according to CKD-EPI) and not dialysis-dependent.
- Absolute neutrophil count ≥1000/µl and platelet count ≥50,000/µl (≥30,000/µl if bone marrow plasma cells are ≥50% of cellularity).
- SGOT ≤ 3x the upper limit of normal and total bilirubin ≤ 2.0 mg/dl (except for patients in whom hyperbilirubinemia is attributed to Gilbert's syndrome).
- Left ventricular ejection fraction (LVEF) ≥ 45%. LVEF assessment must have been performed within 8 weeks of physician-investigator confirmation of eligibility.
- Toxicities from prior/ongoing therapies, with the exception of peripheral neuropathy attributable to multiple myeloma therapy, must have recovered to grade ≤ 2 according to the CTCAE 5.0 criteria or to the subject's prior baseline.
- Subjects must have an ECOG performance status of 0-2.
- Subjects must be willing to forego first-line ASCT (Phase B and C patients only).
- Subjects of reproductive potential must agree to use acceptable birth control methods, as described in the protocol.
Who cannot join
- Pregnant or lactating women
- RETIRED WITH PROTOCOL V6
- Active hepatitis B, hepatitis C, or HIV infection, or other active, uncontrolled infection.
- Any uncontrolled medical or psychiatric disorder that would preclude participation as outlined.
- NYHA Class III or IV heart failure, unstable angina, or a history of recent (within 6 months) myocardial infarction or sustained (>30 seconds) ventricular tachyarrhythmias.
- Have active auto-immune disease, including connective tissue disease, uveitis, sarcoidosis, inflammatory bowel disease, or multiple sclerosis, or have a history of severe (as judged by the physician-investigator) autoimmune disease requiring prolonged immunosuppressive therapy.
- Have prior or active central nervous system (CNS) involvement (e.g. leptomeningeal disease, parenchymal masses) with myeloma. Screening for this (e.g. with lumbar puncture) is not required unless suspicious symptoms or radiographic findings are present. Subjects with calvarial disease that extends intracranially and involves the dura will be excluded, even if CSF is negative for myeloma.
Trial locations and contacts
1 site in 1 country.
United States1 site
Univ. of Pennsylvania
Philadelphia, Pennsylvania, 19104
About this study
Official title: Phase 1 Study of CART-BCMA With or Without huCART19 as Consolidation of Standard First or Second-Line Therapy for High-Risk Multiple Myeloma
This is an open-label, phase 1 study to assess the safety, pharmacodynamics, and anti-myeloma effects of autologous T cells expressing BCMA (B-cell maturation antigen)-specific chimeric antigen receptors with tandem TCRζ and 4-1BB (TCRζ /4-1BB) costimulatory domains (referred to as "CART-BCMA"), with or without huCART19 (also known as CTL119), in patients responding to first- or second-line therapy for high-risk multiple myeloma, and in relapsed/refractory multiple myeloma patients responding to salvage therapy.
Read the detailed description
Phase A: Safety Run-in to test the safety of CART-BCMA + huCART19 as split-dose infusions after lymphodepleting chemotherapy with cyclophosphamide + fludarabine in patients who have relapsed/refractory myeloma after two prior regimens but who are responding to their current therapy. Phase A Expansion: To occur once safety is demonstrated in Phase A. - Phase B: Randomization Phase in which patients responding to first or second-line therapy will receive either CART-BCMA alone (Cohort
- or CART-BCMA + huCART19 (Cohort 2) as split-dose infusions after lymphodepleting chemotherapy with cyclophosphamide + fludarabine. Phase C: Single-dose infusion phase to test the safety of single-dose infusion of CART-BCMA alone (Cohort 1) and CART-BCMA + huCART19 (Cohort 2) as single-dose infusions after lymphodepleting chemotherapy with cyclophosphamide + fludarabine in patients responding to first- or second-line therapy.
- Conditions
- Multiple Myeloma
- Being tested
- BCMA CART + huCART19, CART BCMA or CART BCMA + huCART19, Single-dose infusion of CART BCMA or CART BCMA + huCART19
- Keywords
- Multiple Myeloma, BCMA CART, huCART19
- Medical subject headings
- Multiple Myeloma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BCMA CART + huCART19Biological
The target dose for CART-BCMA and huCART19 will be 5x10^8 CAR-expressing cell for each product. Split dose infusions will consist of a 10% dose (of one or both products) on the first infusion day, 30% dose (of one or both products) on the second infusion day, or 60% dose (of one or both products) on the third infusion day. Infusion days may be spread over 7 calendar days due to scheduling constraints or to allow observation of suspected early cytokine release syndrome or other toxicity. Infusions will begin 3 days (+/- 1 day) after completion of lymphodepleting chemotherapy with cyclophosphamide + fludarabine.
CART BCMA or CART BCMA + huCART19Biological
The target dose for CART-BCMA and huCART19 will be 5x10^8 CAR-expressing cell for each product. Cohort 1 refers to the group of subjects assigned to receive CART-BCMA alone; Cohort 2 refers to the group of subjects assigned to receive CART-BCMA + huCART19. Split dose infusions will consist of a 10% dose (of one or both products) on the first infusion day, 30% dose (of one or both products) on the second infusion day, or 60% dose (of one or both products) on the third infusion day. Infusion days may be spread over 7 calendar days due to scheduling constraints or to allow observation of suspected early cytokine release syndrome or other toxicity. Infusions will begin 3 days (+/- 1 day) after completion of lymphodepleting chemotherapy with cyclophosphamide + fludarabine.
Single-dose infusion of CART BCMA or CART BCMA + huCART19Biological
The target dose for CART-BCMA and huCART19 will be 5x10^8 CAR-expressing cell for each product. Cohort 1 refers to the group of subjects assigned to receive single dose infusions of CART-BCMA alone; Cohort 2 refers to the group of subjects assigned to receive single dose infusions of CART-BCMA + huCART19. Infusions will begin 3 days (+/- 1 day) after completion of lymphodepleting chemotherapy with cyclophosphamide + fludarabine.
BCMA CART + huCART19Biological
The target dose for CART-BCMA and huCART19 will be 5x10^8 CAR-expressing cell for each product. Split dose infusions will consist of a 10% dose (of one or both products) on the first infusion day, 30% dose (of one or both products) on the second infusion day, or 60% dose (of one or both products) on the third infusion day. Infusion days may be spread over 7 calendar days due to scheduling constraints or to allow observation of suspected early cytokine release syndrome or other toxicity. Infusions will begin 3 days (+/- 1 day) after completion of lymphodepleting chemotherapy with cyclophosphamide + fludarabine.
| Study group | Role | What they receive |
|---|---|---|
| Phase A | Experimental | Safety Run-in to test the safety of CART-BCMA + huCART19 as split-dose infusions after lymphodepleting chemotherapy with cyclophosphamide + fludarabine in patients who have relapsed/refractory myeloma after two prior regimens but who are responding to their current therapy. Receives: Biological: BCMA CART + huCART19 |
| Phase B | Experimental | Randomization Phase in which patients responding to first or second-line therapy will receive either CART-BCMA alone (Cohort 1) or CART-BCMA + huCART19 (Cohort 2) as split-doses after lymphodepleting chemotherapy with cyclophosphamide + fludarabine. Receives: Biological: CART BCMA or CART BCMA + huCART19 |
| Phase C | Experimental | Single-dose infusion phase to test the safety of single-dose infusion of CART-BCMA alone (Cohort 1) and CART-BCMA + huCART19 (Cohort 2) as single-dose infusions after lymphodepleting chemotherapy with cyclophosphamide + fludarabine in patients responding to first- or second-line therapy. Receives: Biological: Single-dose infusion of CART BCMA or CART BCMA + huCART19 |
| Phase A Expansion | Experimental | Once safety of CART-BCMA/huCART19 combination therapy is established in Phase A, an expansion of Phase A will occur in which the Phase A target population (patients with relapsed/refractory multiple myeloma responding to a standard salvage therapy regimen) will receive both CART-BCMA and huCART19. Enrollment into the Phase A Expansion may occur concurrently with Phase B once opened. Receives: Biological: BCMA CART + huCART19 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse event reporting
The occurrence of adverse events that are possibly, probably or definitely related to CAR T cells.
Measured: 90 Days
Secondary outcomes (16)
Adverse event reporting
Occurrence of adverse events that are possibly, probably, or definitely related to study interventions during the primary or long-term follow-up phase.
Measured: 15 years
Clinical outcomes after each CAR T cell regimen
Attainment of PET-negative response (absence of detectable FDG-avid disease by PET/CT).
Measured: 2 years
Duration of Response
IMWG 2016 criteria will be used to define disease progression.
Measured: 15 years
Progression-free Survival (PFS)
defined as time from initial CAR T cell until death or progression of multiple myeloma. IMWG 2016 criteria will be used to define disease progression.
Measured: 15 years
Overall Survival (OS)
Measured: 15 years
Evaluate effects of huCART19 on correlative parameters of CART BCMA resistance and clonogenic multiple myeloma cells, such as the following:
Persistence of clonal BCMAdim/neg or CD19+ plasma cells as measured by flow cytometry and immunohistochemistry
- Depletion of multiple myeloma clonogenicity as measured using in vitro colony formation assays on bone marrow samples
- Induction of anti-Sox2 and other anti-myeloma immune responses
- Depletion of clonal CD19+ B cells
Measured: 2 years
Composition of investigative products
Evaluate cellular composition of apheresis product and CARTBCMA/ huCART19 cells.
Measured: 2 years
Maintenance therapy effects on persistence
Evaluate effects of post-infusion maintenance therapy on CAR T cell persistence using quantitative molecular methods.
Measured: 28 days post infusion - 2 years
In vivo CAR T cell expansion as measured by flow cytometry
Measured: 28 days post infusion - 2 years
In vivo CAR T cell expansion as measured by qPCR
Measured: 28 days post infusion - 2 years
Duration of in vivo persistence of CAR T cells.
As measured by flow cytometry and/or qPCR for vector sequences. For each parameter, CART-BCMA and huCART19 pharmacokinetics will be analyzed separately for patients receiving both products
Measured: 28 days post infusion - 2 years
Effects of maintenance therapy on CAR T cell pharmacokinetic parameters.
As measured by flow cytometry and/or qPCR for vector sequences. For each parameter, CART-BCMA and huCART19 pharmacokinetics will be analyzed separately for patients receiving both products
Measured: 28 days post infusion - 2 years
Bioactivity by multiplex cytokine analysis
As measured by flow cytometry and/or qPCR for vector sequences. For each parameter, CART-BCMA and huCART19 pharmacokinetics will be analyzed separately for patients receiving both products
Measured: 28 days post infusion - 2 years
Cellular composition of CAR T cell products
cell-surface immunophenotype
Measured: 28 days post infusion - 2 years
Immune cell phenotyping
Characterize the cellular phenotype of multiple myeloma cells that persist after CAR T cell treatment using qualitative molecular methods.
Measured: 2 years
Impact of T Cells on systemic soluble immune factors in patients
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2018
- Study start9 May 2018
- Primary completionMar 2036 (estimated)
- Study completionMar 2036 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Multiple MyelomaStudies of the same condition, open and under way.
- More trials run by University of PennsylvaniaThe sponsor's other registered studies.
- 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
- Basic science
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Three separate phases. Phases A and C are non-randomized, Phase B is randomized
- Masking
- None (open label)
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Pennsylvania
- Sponsor type
- University, hospital or other
- Collaborators
- Novartis, National Institutes of Health (NIH)
- Responsible party
- Sponsor
- Investigators
- Alfred Garfall, MD, Principal investigator, University of Pennsylvania
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03549442
- Sponsor's protocol ID
- 828773
- Status last verified
- Jul 2026
- First submitted
- 27 Apr 2018
- Last updated
- 10 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Zhang Z, Markmann C, Yu M, Agarwal D, Rostami S, Wang W, Liu C, Zhao H, Ochoa T, Parvathaneni K, Xu X, Li E, Gonzalez V, Khadka R, Hoffmann J, Knox JJ, Scholler J, Marcellus B, Allman D, Fraietta JA, Samelson-Jones B, Milone MC, Monos D, Garfall AL, Naji A, Bhoj VG. Immunotherapy targeting B cells and long-lived plasma cells effectively eliminates pre-existing donor-specific allo-antibodies. Cell Rep Med. 2023 Dec 19;4(12):101336. doi: 10.1016/j.xcrm.2023.101336.
PubMed 38118406
Study at a glance
From the registry record, last updated by the sponsor on 10 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1 Study of CART-BCMA With or Without huCART19 as Consolidation of Standard First or Second-Line Therapy for High-Risk Multiple Myeloma
- ClinicalTrials.gov ID
- NCT03549442
- Acronym / sponsor ID
- 828773
- Conditions
- Multiple Myeloma
- Keywords
- Multiple Myeloma, BCMA CART, huCART19
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 40 participants (actual)
- Lead sponsor
- University of PennsylvaniaUniversity, hospital or other
- Collaborators
- Novartis, National Institutes of Health (NIH)
- Study start
- 9 May 2018 (actual)
- Primary completion
- Mar 2036 (estimated)
- Study completion
- Mar 2036 (estimated)
- First posted
- 8 Jun 2018 (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 is an open-label, phase 1 study to assess the safety, pharmacodynamics, and anti-myeloma effects of autologous T cells expressing BCMA (B-cell maturation antigen)-specific chimeric antigen receptors with tandem TCRζ and 4-1BB (TCRζ /4-1BB) costimulatory domains (referred to as "CART-BCMA"), with or without huCART19 (also known as CTL119), in patients responding to first- or second-line therapy for high-risk multiple myeloma, and in relapsed/refractory multiple myeloma patients responding to salvage therapy.
Detailed description
Phase A: Safety Run-in to test the safety of CART-BCMA + huCART19 as split-dose infusions after lymphodepleting chemotherapy with cyclophosphamide + fludarabine in patients who have relapsed/refractory myeloma after two prior regimens but who are responding to their current therapy. Phase A Expansion: To occur once safety is demonstrated in Phase A. - Phase B: Randomization Phase in which patients responding to first or second-line therapy will receive either CART-BCMA alone (Cohort
- or CART-BCMA + huCART19 (Cohort 2) as split-dose infusions after lymphodepleting chemotherapy with cyclophosphamide + fludarabine. Phase C: Single-dose infusion phase to test the safety of single-dose infusion of CART-BCMA alone (Cohort 1) and CART-BCMA + huCART19 (Cohort 2) as single-dose infusions after lymphodepleting chemotherapy with cyclophosphamide + fludarabine in patients responding to first- or second-line therapy.
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
- Model description
- Three separate phases. Phases A and C are non-randomized, Phase B is randomized
| Arm | Type | Receives |
|---|---|---|
| Phase A | Experimental | Biological: BCMA CART + huCART19Safety Run-in to test the safety of CART-BCMA + huCART19 as split-dose infusions after lymphodepleting chemotherapy with cyclophosphamide + fludarabine in patients who have relapsed/refractory myeloma after two prior regimens but who are responding to their current therapy. |
| Phase B | Experimental | Biological: CART BCMA or CART BCMA + huCART19Randomization Phase in which patients responding to first or second-line therapy will receive either CART-BCMA alone (Cohort 1) or CART-BCMA + huCART19 (Cohort 2) as split-doses after lymphodepleting chemotherapy with cyclophosphamide + fludarabine. |
| Phase C | Experimental | Biological: Single-dose infusion of CART BCMA or CART BCMA + huCART19Single-dose infusion phase to test the safety of single-dose infusion of CART-BCMA alone (Cohort 1) and CART-BCMA + huCART19 (Cohort 2) as single-dose infusions after lymphodepleting chemotherapy with cyclophosphamide + fludarabine in patients responding to first- or second-line therapy. |
| Phase A Expansion | Experimental | Biological: BCMA CART + huCART19Once safety of CART-BCMA/huCART19 combination therapy is established in Phase A, an expansion of Phase A will occur in which the Phase A target population (patients with relapsed/refractory multiple myeloma responding to a standard salvage therapy regimen) will receive both CART-BCMA and huCART19. Enrollment into the Phase A Expansion may occur concurrently with Phase B once opened. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (32 listed)
- Subjects must have a diagnosis of multiple myeloma according to IMWG 2014 criteria106 with any of the following high-risk features. Subjects in the Phase A Expansion are not required to have any high-risk features.
- Subjects must meet the following criteria with respect to prior myeloma therapy:
- Note: Refractoriness is defined as disease progression on-therapy or within 60 days of stopping therapy.
- Subjects must not have achieved a stringent complete response according to IMWG 2016 criteria1 at time of physician-investigator confirmation of eligibility unless clonal plasma cells are detectable in bone marrow by flow cytometry (I.e., subjects in stringent complete response are eligible if minimal residual disease can be documented by bone marrow flow cytometry) or if residual disease is detectable by imaging such as PET/CT, CT, or MRI.
- Subjects must have signed written, informed consent.
Key exclusion criteria (7 listed)
- Pregnant or lactating women
- RETIRED WITH PROTOCOL V6
- Active hepatitis B, hepatitis C, or HIV infection, or other active, uncontrolled infection.
- Any uncontrolled medical or psychiatric disorder that would preclude participation as outlined.
- NYHA Class III or IV heart failure, unstable angina, or a history of recent (within 6 months) myocardial infarction or sustained (>30 seconds) ventricular tachyarrhythmias.
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 |
|---|---|---|
| BCMA CART + huCART19The target dose for CART-BCMA and huCART19 will be 5x10^8 CAR-expressing cell for each product. Split dose infusions will consist of a 10% dose (of one or both products) on the first infusion day, 30% dose (of one or both products) on the second infusion day, or 60% dose (of one or both products) on the third infusion day. Infusion days may be spread over 7 calendar days due to scheduling constraints or to allow observation of suspected early cytokine release syndrome or other toxicity. Infusions will begin 3 days (+/- 1 day) after completion of lymphodepleting chemotherapy with cyclophosphamide + fludarabine. | Biological | Phase A |
| CART BCMA or CART BCMA + huCART19The target dose for CART-BCMA and huCART19 will be 5x10^8 CAR-expressing cell for each product. Cohort 1 refers to the group of subjects assigned to receive CART-BCMA alone; Cohort 2 refers to the group of subjects assigned to receive CART-BCMA + huCART19. Split dose infusions will consist of a 10% dose (of one or both products) on the first infusion day, 30% dose (of one or both products) on the second infusion day, or 60% dose (of one or both products) on the third infusion day. Infusion days may be spread over 7 calendar days due to scheduling constraints or to allow observation of suspected early cytokine release syndrome or other toxicity. Infusions will begin 3 days (+/- 1 day) after completion of lymphodepleting chemotherapy with cyclophosphamide + fludarabine. | Biological | Phase B |
| Single-dose infusion of CART BCMA or CART BCMA + huCART19The target dose for CART-BCMA and huCART19 will be 5x10^8 CAR-expressing cell for each product. Cohort 1 refers to the group of subjects assigned to receive single dose infusions of CART-BCMA alone; Cohort 2 refers to the group of subjects assigned to receive single dose infusions of CART-BCMA + huCART19. Infusions will begin 3 days (+/- 1 day) after completion of lymphodepleting chemotherapy with cyclophosphamide + fludarabine. | Biological | Phase C |
| BCMA CART + huCART19The target dose for CART-BCMA and huCART19 will be 5x10^8 CAR-expressing cell for each product. Split dose infusions will consist of a 10% dose (of one or both products) on the first infusion day, 30% dose (of one or both products) on the second infusion day, or 60% dose (of one or both products) on the third infusion day. Infusion days may be spread over 7 calendar days due to scheduling constraints or to allow observation of suspected early cytokine release syndrome or other toxicity. Infusions will begin 3 days (+/- 1 day) after completion of lymphodepleting chemotherapy with cyclophosphamide + fludarabine. | Biological | Phase A Expansion |
Primary outcome
Adverse event reporting
Time frame: 90 Days
The occurrence of adverse events that are possibly, probably or definitely related to CAR T cells.
16 secondary outcomes
Adverse event reporting
Time frame: 15 years
Clinical outcomes after each CAR T cell regimen
Time frame: 2 years
Duration of Response
Time frame: 15 years
Progression-free Survival (PFS)
Time frame: 15 years
Overall Survival (OS)
Time frame: 15 years
Evaluate effects of huCART19 on correlative parameters of CART BCMA resistance and clonogenic multiple myeloma cells, such as the following:
Time frame: 2 years
Composition of investigative products
Time frame: 2 years
Maintenance therapy effects on persistence
Time frame: 28 days post infusion - 2 years
In vivo CAR T cell expansion as measured by flow cytometry
Time frame: 28 days post infusion - 2 years
In vivo CAR T cell expansion as measured by qPCR
Time frame: 28 days post infusion - 2 years
Duration of in vivo persistence of CAR T cells.
Time frame: 28 days post infusion - 2 years
Effects of maintenance therapy on CAR T cell pharmacokinetic parameters.
Time frame: 28 days post infusion - 2 years
Bioactivity by multiplex cytokine analysis
Time frame: 28 days post infusion - 2 years
Cellular composition of CAR T cell products
Time frame: 28 days post infusion - 2 years
Immune cell phenotyping
Time frame: 2 years
Impact of T Cells on systemic soluble immune factors in patients
Time frame: 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jul 2026.
- Derived from this study: PubMed 38118406Zhang Z, Markmann C, Yu M, Agarwal D, Rostami S, Wang W, Liu C, Zhao H, Ochoa T, Parvathaneni K, Xu X, Li E, Gonzalez V, Khadka R, Hoffmann J, Knox JJ, Scholler J, Marcellus B, Allman D, Fraietta JA, Samelson-Jones B, Milone MC, Monos D, Garfall AL, Naji A, Bhoj VG. Immunotherapy targeting B cells and long-lived plasma cells effectively eliminates pre-existing donor-specific allo-antibodies. Cell Rep Med. 2023 Dec 19;4(12):101336. doi: 10.1016/j.xcrm.2023.101336.
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Active, not recruitingPhase 1/2
A Window of Opportunity Trial to Learn if Linvoseltamab is Safe and Well Tolerated, and How Well it Works in Adult Participants With Recently Diagnosed Multiple Myeloma Who Have Not Already Received Treatment
Multiple Myeloma
Active, not recruitingPhase 2
Testing AZD5363 as a Potential Targeted Treatment in Cancers With AKT Genetic Changes (MATCH-Subprotocol Y)
Advanced Lymphoma, Advanced Malignant Solid Neoplasm
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.
Alfred Garfall, MD principal investigator
University of Pennsylvania
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 BCMA CART + huCART19, CART BCMA or CART BCMA + huCART19 and Single-dose infusion of CART BCMA or CART BCMA + huCART19 in people with Multiple Myeloma, run by University of Pennsylvania 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 32 inclusion and 7 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 10 Jul 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?
Adverse event reporting (time frame: 90 Days). 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 NCT03549442, which also lists 1 linked publication.
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 10 Jul 2026.