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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03288493
Recruitment Status : Recruiting
First Posted : September 20, 2017
Last Update Posted : August 8, 2018
Information provided by (Responsible Party):
Poseida Therapeutics, Inc.

Brief Summary:
This is a Phase 1, open-label, multi-center study of P-BCMA-101 autologous T stem cell memory (Tscm) CAR-T cells in patients with relapsed and/or refractory MM.

Condition or disease Intervention/treatment Phase
Multiple Myeloma Biological: P-BCMA-101 CAR-T cells Phase 1

Detailed Description:
The study will follow a 3 + 3 design of dose-escalating cohorts. 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. Treated patients will undergo serial measurements of safety, tolerability and response.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Intervention Model: Single Group Assignment
Intervention Model Description: 3 + 3 design of dose-escalating cohorts
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open-Label, Multicenter, Single Ascending Dose Study to Assess the Safety of P-BCMA-101 in Subjects With Relapsed and/or Refractory Multiple Myeloma (MM)
Actual Study Start Date : September 20, 2017
Estimated Primary Completion Date : December 31, 2018
Estimated Study Completion Date : June 30, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Multiple Myeloma

Arm Intervention/treatment
Experimental: P-BCMA-101 CAR-T cells
single dose, intravenous infusion, CAR-T cells
Biological: P-BCMA-101 CAR-T cells
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.

Primary Outcome Measures :
  1. Maximum tolerated dose of P-BCMA-101 based on dose limiting toxicities [ Time Frame: Baseline through Day 28 ]
    Rate of dose limiting toxicities

Secondary Outcome Measures :
  1. Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: Baseline through Month 24 ]
    Incidence and severity of treatment-emergent adverse events

  2. Anti-myeloma effect of P-BCMA-101(Response Rate) [ Time Frame: Baseline through Month 24 ]
    Percentage of patients with complete response (CR), very good partial response (VGPR), or partial response (PR) according to the International Myeloma Working Group (IMWG) Uniform Response Criteria for Multiple Myeloma.

  3. Anti-myeloma effect of P-BCMA-101(Duration of Response) [ Time Frame: Baseline through Month 24 ]
    Time from complete response (CR), very good partial response (VGPR), or partial response (PR) to progressive disease according to the International Myeloma Working Group (IMWG) Uniform Response Criteria for Multiple Myeloma.

  4. Anti-myeloma effect of P-BCMA-101(Progression Free Survival) [ Time Frame: Baseline through Month 24 ]
    Time from P-BCMA-101 treatment to progressive disease according to the International Myeloma Working Group (IMWG) Uniform Response Criteria for Multiple Myeloma.or death

  5. Anti-myeloma effect of P-BCMA-101(Overall Survival) [ Time Frame: Baseline through Month 24 ]
    Duration of survival from time of treatment with P-BCMA-101

  6. Pharmacokinetics of P-BCMA-101 [ Time Frame: Baseline through Month 24 ]
    Concentration of P-BCMA-101 cells in blood and bone marrow over time

  7. Biomarkers for P-BCMA-101 [ Time Frame: Baseline through Month 24 ]
    Levels of BCMA in blood and bone marrow cells

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Males or females, ≥18 years of age
  • Must have a confirmed diagnosis of active MM
  • Must have measurable MM
  • Must have relapsed and/or refractory MM, having received treatment with proteasome inhibitor and IMiD
  • Must have adequate hepatic, renal, cardiac and hematopoietic function

Exclusion Criteria:

  • 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, amyloidosis, significant autoimmune, CNS or other malignant disease
  • Has an active systemic infection
  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03288493

Contact: Michelle Resler 1-858-779-3100

United States, California
University of California San Diego Recruiting
San Diego, California, United States, 92121
United States, Colorado
Colorado Blood Cancer Institute Recruiting
Denver, Colorado, United States, 80218
United States, Maryland
Johns Hopkins University Recruiting
Baltimore, Maryland, United States, 21205
United States, Pennsylvania
University of Pennsylvania Recruiting
Philadelphia, Pennsylvania, United States, 19104
United States, Tennessee
Sarah Cannon Research Institute at Tennessee Oncology Recruiting
Nashville, Tennessee, United States, 37203
United States, Texas
MD Anderson Cancer Center Recruiting
Houston, Texas, United States, 77030
Sponsors and Collaborators
Poseida Therapeutics, Inc.
Study Director: Matthew A Spear, M.D. Sponsor Chief Medical Officer

Responsible Party: Poseida Therapeutics, Inc. Identifier: NCT03288493     History of Changes
Other Study ID Numbers: P-BCMA-101-001
First Posted: September 20, 2017    Key Record Dates
Last Update Posted: August 8, 2018
Last Verified: August 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Poseida Therapeutics, Inc.:
CAR-T cells

Additional relevant MeSH terms:
Multiple Myeloma
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases