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Multiple MyelomaPhase 1/2NCT03288493

P-BCMA-101 Tscm CAR-T Cells in the Treatment of Patients With Multiple Myeloma (MM)

A Phase 1/2 study testing P-BCMA-101 CAR-T cells and Rimiducid in people with Multiple Myeloma, run by Poseida Therapeutics, Inc. at 16 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

Phase 1 of the study is comprised of an open-label, single ascending dose (SAD), multiple cohort study; a multiple dose cycle administration cohort study; and a combination administration study of P-BCMA-101 autologous T stem cell memory (Tscm) CAR-T cells in patients with relapsed / refractory MM. 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?
P-BCMA-101 CAR-T cells and Rimiducid
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
105 participants
Where
16 sitesUnited States
Study dates
20 Sep 2017to 27 Apr 2022
Phase
Phase 1/2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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

  • Males or females, ≥18 years of age
  • Must have a confirmed diagnosis of active MM
  • Must have measurable MM
  • Must have relapsed / refractory MM, having received treatment with proteasome inhibitor and IMiD [Phase 2: Must have relapsed / refractory MM, and refractory to last line of therapy, having received treatment with proteasome inhibitor, an IMiD, CD38 targeted therapy and undergone autologous stem cell transplant (ASCT) or not a candidate for ASCT.]
  • Must have adequate hepatic, renal, cardiac and hematopoietic function

Who cannot join

  • Is pregnant or lactating
  • Has inadequate venous access and/or contraindications to leukapheresis
  • Has active hemolytic anemia, plasma cell leukemia, Waldenstrom's macroglobulinemia, POEMS syndrome, disseminated intravascular coagulation, leukostasis, amyloidosis, significant autoimmune, CNS or other malignant disease
  • Has an active second malignancy (not disease-free for at least 5 years) in addition to MM, excluding low-risk neoplasms such as non-metastatic basal cell or squamous cell skin carcinoma.
  • Has active autoimmune disease
  • Has a history of significant central nervous system (CNS) disease, such as stroke, epilepsy, etc.
Show all 14Show fewer
  • Has an active systemic infection
  • Has hepatitis B or C virus, human immunodeficiency virus (HIV), or human T-lymphotropic virus (HTLV) infection, or any immunodeficiency syndrome.
  • Has any psychiatric or medical disorder that would preclude safe participation in and/or adherence to the protocol
  • Has receiving immunosuppressive or other contraindicated therapies within the excluded time frame from entry
  • Has CNS metastases or symptomatic CNS involvement
  • Has a history of having undergone allogeneic stem cell transplantation, or any other allogeneic or xenogeneic transplant, or has undergone autologous transplantation within 90 days.
  • Unable to take acetylsalicylic acid (ASA) daily as prophylactic anticoagulation. (Cohorts R and RP only).
  • History of thromboembolic disease within the past 6 months, regardless of anticoagulation (Cohorts R and RP only).

Trial locations and contacts

16 sites in 1 country.

16 sites in 1 country

United States16 sites

  • University of Maryland Greenebaum Comprehensive Cancer Center

    Baltimore, Maryland, 21201

  • Johns Hopkins University

    Baltimore, Maryland, 21231

  • University of Chicago

    Chicago, Illinois, 60637

  • University of California Davis

    Davis, California, 95618

  • Colorado Blood Cancer Institute

    Denver, Colorado, 80218

  • Wayne State - Karmanos Cancer Institute

    Detroit, Michigan, 48201

  • Banner MD Anderson Cancer Center

    Gilbert, Arizona, 85234

  • John Theurer Cancer Center

    Hackensack, New Jersey, 07601

  • MD Anderson Cancer Center

    Houston, Texas, 77030

  • Sarah Cannon Research Institute at Tennessee Oncology

    Nashville, Tennessee, 37203

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104

  • University of California San Diego

    San Diego, California, 92093

  • University of California San Francisco

    San Francisco, California, 94143

  • Swedish Cancer Institute

    Seattle, Washington, 98104

  • University of Kansas Cancer Center

    Westwood, Kansas, 66205

Other trials in United States

About this study

Official title: Open-Label, Multicenter, Phase 1 Study to Assess the Safety of P BCMA-101 in Subjects With Relapsed / Refractory Multiple Myeloma (MM) Followed by a Phase 2 Assessment of Response and Safety (PRIME)

Phase 1 of the study is comprised of an open-label, single ascending dose (SAD), multiple cohort study; a multiple dose cycle administration cohort study; and a combination administration study of P-BCMA-101 autologous T stem cell memory (Tscm) CAR-T cells in patients with relapsed / refractory MM. Followed by a Phase 2, open-label, efficacy and safety study. Rimiducid may be administered as indicated.

Read the detailed description

Phase 1 follows a 3 + 3 design of dose-escalating cohorts. Phase 2 of the study is an open-label multi-center efficacy and safety study. After a patient enrolls, leukapheresis will be performed to obtain peripheral blood mononuclear cells which will be sent to a manufacturing site to produce P-BCMA-101 CAR-T cells. The cells will then be returned to the investigational site and, after a standard chemotherapy based conditioning regimen, will be administered to the patient across 1-3 infusions, with or without combination therapy. Treated patients will undergo serial measurements of safety, tolerability and response. Rimiducid may be administered as indicated.

Being tested
P-BCMA-101 CAR-T cells, Rimiducid
Keywords
CAR-T cells
Medical subject headings
Multiple Myeloma

What participants receive

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

  • P-BCMA-101 CAR-T cellsBiological

    P-BCMA-101 is an autologous, principally Tscm, CAR-T cell product (also called called a CARTyrin T cell product) targeting the myeloma selective protein BCMA. P-BCMA-101 cells are produced using a non-viral vector carrying the gene for an anti-BCMA Centyrin-based (small, fully human binding domain, designed to increase T cell persistence and decrease exhaustion) chimeric antigen receptor (CAR). Secondary to the large carrying capacity of the non-viral vector, P-BCMA-101 cells carry two additional genes, a selection gene used to manufacture a purified product and a "safety switch" gene to allow the cells to be eliminated if desired. Rimiducid (safety switch activator) may be administered as indicated.

  • RimiducidDrug

    Rimiducid (safety switch activator) may be administered as indicated.

Study groups in NCT03288493
Study groupRoleWhat they receive
Phase 1: P-BCMA-101 CAR-T cellsExperimentalSingle ascending dose cohorts, given in a single intravenous infusion of CAR-T cells. Rimiducid may be administered as indicated.
Receives: Biological: P-BCMA-101 CAR-T cells; Drug: Rimiducid
Phase 1 P-BCMA-101 CAR-T cells (Cohort A)ExperimentalSingle dose given across two intravenous infusions of CAR-T cells. Rimiducid may be administered as indicated.
Receives: Biological: P-BCMA-101 CAR-T cells; Drug: Rimiducid
Phase 1 P-BCMA-101 CAR-T cells (Cohort B)ExperimentalSingle dose given across three intravenous infusions of CAR-T cells. Rimiducid may be administered as indicated.
Receives: Biological: P-BCMA-101 CAR-T cells; Drug: Rimiducid
Phase 1 P-BCMA-101 CAR-T cells (Cohort C)ExperimentalSingle dose given across two intravenous infusions of CAR-T cells. Rimiducid may be administered as indicated.
Receives: Biological: P-BCMA-101 CAR-T cells; Drug: Rimiducid
Phase 1 P-BCMA-101 CAR-T cells with Comb.Therapy (Cohort R)ExperimentalSingle intravenous infusion of CAR-T cells, with combination therapy, beginning one week before CAR-T infusion. Rimiducid may be administered as indicated.
Receives: Biological: P-BCMA-101 CAR-T cells; Drug: Rimiducid
Phase 1 P-BCMA-101 CAR-T cells with Comb.Therapy (Cohort RP)ExperimentalSingle intravenous infusion of CAR-T cells, with combination therapy, beginning one week before apheresis. Rimiducid may be administered as indicated.
Receives: Biological: P-BCMA-101 CAR-T cells; Drug: Rimiducid
Phase 1 P-BCMA-101 CAR-T cells with Comb.Therapy (Cohort RIT)ExperimentalSingle intravenous infusion of CAR-T cells, with combination therapy, beginning one week before CAR-T infusion. Rimiducid may be administered as indicated.
Receives: Biological: P-BCMA-101 CAR-T cells; Drug: Rimiducid
Phase 2: P-BCMA-101 CAR-T CellsExperimentalCAR-T cells administered via intravenous infusion as a total dose
Receives: Biological: P-BCMA-101 CAR-T cells; Drug: Rimiducid

What is being measured

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

  1. Phase 1: Assess the Safety of P-BCMA-101

    Incidence and severity of treatment-emergent adverse events

    Measured: Baseline through Day 28

  2. Phase 1: Maximum Tolerated Dose of P-BCMA-101

    Rate of dose limiting toxicities (DLT)

    Measured: Baseline through Day 28

  3. Phase 2: Assess the Safety of P-BCMA-101

    Incidence and severity of treatment-emergent adverse events

    Measured: Baseline through 24 months

  4. Phase 2: Assess the Efficacy of P-BCMA-101 (ORR)

    According to the International Myeloma Working Group (IMWG) Uniform Response Criteria for Multiple Myeloma:

    Overall Response Rate (ORR)-Percentage of patients with complete response (CR), very good partial response (VGPR), or partial response (PR).

    Measured: Baseline through 24 months

  5. Phase 2: Assess the Efficacy of P-BCMA-101 (DOR)

    According to the International Myeloma Working Group (IMWG) Uniform Response Criteria for Multiple Myeloma:

    Duration of Response (DOR)-Time from complete response (CR), very good partial response (VGPR), or partial response (PR) to progressive disease.

    Measured: Baseline through 24 months

Secondary outcomes (16)

  1. Phase 1:Assess the Safety of P-BCMA-101

    Incidence and severity of treatment-emergent adverse events

    Measured: Baseline through Month 24

  2. Phase 1:Assess the Feasibility P-BCMA-101

    Ability to generate protocol-prescribed doses of P-BCMA-101.

    Measured: Baseline through Month 24

  3. Phase 1: Anti-myeloma Effect of P-BCMA-101 (ORR)

    According to the International Myeloma Working Group (IMWG) Uniform Response Criteria for Multiple Myeloma:

    Overall Response Rate (ORR)-Percentage of patients with complete response (CR), very good partial response (VGPR), or partial response (PR).

    Measured: Baseline through Month 24

  4. Phase 1: Anti-myeloma Effect of P-BCMA-101 (TTR)

    According to the International Myeloma Working Group (IMWG) Uniform Response Criteria for Multiple Myeloma:

    Time to Response (TTR)-Time to complete response (CR), very good partial response (VGPR), or partial response (PR) to progressive disease.

    Measured: Baseline through Month 24

  5. Phase 1: Anti-myeloma Effect of P-BCMA-101 (DOR)

    According to the International Myeloma Working Group (IMWG) Uniform Response Criteria for Multiple Myeloma:

    Duration of Response (DOR)-Time from complete response (CR), very good partial response (VGPR), or partial response (PR) to progressive disease.

    Measured: Baseline through Month 24

  6. Phase 1: Anti-myeloma Effect of P-BCMA-101 (PFS)

    According to the International Myeloma Working Group (IMWG) Uniform Response Criteria for Multiple Myeloma:

    Progression Free Survival (PFS)-Time from P-BCMA-101 treatment to progressive disease.

    Measured: Baseline through Month 24

  7. Phase 1: Anti-myeloma Effect of P-BCMA-101 (OS)

    According to the International Myeloma Working Group (IMWG) Uniform Response Criteria for Multiple Myeloma:

    Overall Survival (OS)-Duration of survival from time of treatment with P-BCMA-101.

    Measured: Baseline through Month 24

  8. Phase 1: The Effect of Cell Dose to Guide Selection of Doses for Further Assessment in Phase 2/3 Studies

    Incidence and severity of CRS events graded using Lee criteria (Lee, 2014)

    Measured: Baseline through Month 24

  9. Phase 2: Incidence and Severity of Cytokine Release Syndrome (CRS)

    Incidence and severity of CRS events graded using Lee criteria (Lee, 2014)

    Measured: Baseline through Month 24

  10. Phase 2: Evaluate Efficacy Endpoints (IL-6)

    Rate of IL-6 antagonist

    Measured: Baseline through Month 24

  11. Phase 2: Evaluate Efficacy Endpoints (C)

    Corticosteroid Use

    Measured: Baseline through Month 24

  12. Phase 2: Evaluate Efficacy Endpoints (R)

    Rimiducid Use

    Measured: Baseline through Month 24

  13. Phase 2: Evaluate Efficacy Endpoints (OS)

    According to the International Myeloma Working Group (IMWG) Uniform Response Criteria for Multiple Myeloma:

    Overall Survival (OS)-Duration of survival from time of treatment with P-BCMA-101.

    Measured: Baseline through Month 24

  14. Phase 2: Evaluate Efficacy Endpoints (PFS)

    According to the International Myeloma Working Group (IMWG) Uniform Response Criteria for Multiple Myeloma:

    Progression Free Survival (PFS)-Time from P-BCMA-101 treatment to progressive disease.

    Measured: Baseline through Month 24

  15. Phase 2: Evaluate Efficacy Endpoints (TTR)

    According to the International Myeloma Working Group (IMWG) Uniform Response Criteria for Multiple Myeloma:

    Time to Response (TTR)-Time to complete response (CR), very good partial response (VGPR), or partial response (PR) to progressive disease.

    Measured: Baseline through Month 24

  16. Phase 2: Evaluate Efficacy Endpoints (MRD)

    Minimum residual disease negative rate

    Measured: Baseline through Month 24

Timeline

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

  1. First posted20 Sep 2017
  2. Study start20 Sep 2017
  3. Primary completion27 Apr 2022
  4. Study completion27 Apr 2022

Why it stopped: Phase I portion of the study was completed. The phase II portion of the study was terminated early to focus on an Allogeneic BCMA CAR-T program.

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 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Model description
Phase 1: open label, 3 + 3 design of dose-escalating cohorts Phase 2: open label, administered as a total dose
Masking
None (open label)
Enrolment
105 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03288493
Sponsor's protocol ID
P-BCMA-101-001
Status last verified
Mar 2024
First submitted
13 Sep 2017
Last updated
28 Mar 2024
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Miah KM, Hyde SC, Gill DR. Emerging gene therapies for cystic fibrosis. Expert Rev Respir Med. 2019 Aug;13(8):709-725. doi: 10.1080/17476348.2019.1634547. Epub 2019 Jun 27.

    PubMed 31215818

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing P-BCMA-101 CAR-T cells and Rimiducid in people with Multiple Myeloma, run by Poseida Therapeutics, Inc. at 16 sites 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 5 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: stopped early, as of the sponsor's last update on 28 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 16 sites 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 combined 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.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 22 Jun 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Phase 1: Assess the Safety of P-BCMA-101 (time frame: Baseline through Day 28). 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?

On the ClinicalTrials.gov record for NCT03288493, 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 28 Mar 2024.

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