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Clinical Trial to Evaluate BCMA Car-T (CT053) in Patients With Relapsed and/or Refractory Multiple Myeloma

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.
 
ClinicalTrials.gov Identifier: NCT03975907
Recruitment Status : Recruiting
First Posted : June 5, 2019
Last Update Posted : March 25, 2020
Sponsor:
Collaborators:
Beijing Chao Yang Hospital
The First Affiliated Hospital of Soochow University
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Tianjin Medical University General Hospital
First Affiliated Hospital of Zhejiang University
Beijing Hospital
Shanghai Tongji Hospital, Tongji University School of Medicine
First Affiliated Hospital of Wenzhou Medical University
Xiangya Hospital of Central South University
Peking University People's Hospital
Qilu Hospital of Shandong University
Sun Yat-sen University
First Affiliated Hospital, Sun Yat-Sen University
Information provided by (Responsible Party):
Carsgen Therapeutics, Ltd.

Brief Summary:
This is an open-label, single arm study to evaluate the safety and tolerability of treatment with CT053 CAR-BCMA T in patients with relapsed and/or refractory multiple myeloma.

Condition or disease Intervention/treatment Phase
Multiple Myeloma Biological: CAR-BCMA T Cells Phase 1 Phase 2

Detailed Description:
The study is composed of two stages, Phase I stage is for dose escalation and recommendation of phase 2 dose, and Phase II stage is to Detailed Description: verify the efficacy and safety of the dose proposed.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 62 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description:

Phase I is an open-label, dose escalation study and 2 cohorts to evaluate the safety and tolerability of treatment with CT053 and to determine maximum tolerable dose (MTD) and the Recommended Phase 2 Dose (RP2D) for CT053.

Phase 2 is a single-arm, open, multi-center study, to evaluate the efficacy and safety of CAR-BCMA T cells (CT053) in subjects with RR/MM.

Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open Label, Phase I/II Clinical Trial to Evaluate the Safety and Efficacy of Fully Human Anti-BCMA Chimeric Antibody Receptor Autologous T Cell (CAR T) Infusion in Patients With Relapsed and/or Refractory Multiple Myeloma
Actual Study Start Date : June 10, 2019
Estimated Primary Completion Date : December 1, 2022
Estimated Study Completion Date : December 1, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Multiple Myeloma

Arm Intervention/treatment
Experimental: CAR-BCMA T Cells
Phase I: The subjects are enrolled into 2 dose level cohorts in sequence. Phase II: Single arm
Biological: CAR-BCMA T Cells
The CAR-BCMA T cells (study drug) used in this study are chimeric antigen receptor specifically expressing T cells targeting BCMA.




Primary Outcome Measures :
  1. Phase 1, Safety and tolerability: dose limiting toxicity [ Time Frame: 28days post administration of CAR-T-cells ]
    dose limiting toxicity

  2. Phase 2, efficacy of CT053 CAR-BCMA T cells: overall response rate [ Time Frame: 3 months post administration of CAR-T-cells ]
    overall response rate (ORR)=(sCR+CR+VGPR+PR)


Secondary Outcome Measures :
  1. Additional efficacy evaluation after 12 weeks of CT053 CAR-BCMA T cells infusion [ Time Frame: 3 months post administration of CAR-T-cells ]
    Minimal residual disease (MRD) negativity

  2. Additional efficacy evaluation after 12 weeks of CT053 CAR-BCMA T cells infusion [ Time Frame: 3 months post administration of CAR-T-cells ]
    TTR TIme to Response

  3. Additional efficacy evaluation after 12 weeks of CT053 CAR-BCMA T cells infusion [ Time Frame: 3 months post administration of CAR-T-cells ]
    Complete Response (CR) /stringent Complete Response (sCR)

  4. Additional efficacy evaluation after 12 weeks of CT053 CAR-BCMA T cells infusion [ Time Frame: 3 months post administration of CAR-T-cells ]
    ≥Very Good Partial Response (VGPR), including VGPR, CR, sCR

  5. Additional efficacy evaluation after 12 weeks of CT053 CAR-BCMA T cells infusion [ Time Frame: 3 months post administration of CAR-T-cells ]
    ORR at Wk12

  6. Additional efficacy evaluation after 12 weeks of CT053 CAR-BCMA T cells infusion [ Time Frame: 3 months post administration of CAR-T-cells ]
    CBR, clinical benefit rate

  7. Safety and tolerability of CAR-BCMA T cell therapy [ Time Frame: through 24 months post administration of CAR-T-cells ]
    AE&SAE

  8. Pharmacokinetics (the cell persistence duration in peripheral blood) [ Time Frame: through 24 months post administration of CAR-T-cells ]
    Copy numbers of CAR-BCMA gene in peripheral blood

  9. Pharmacokinetics (the cell persistence duration in peripheral blood) [ Time Frame: through 24 months post administration of CAR-T-cells ]
    Number of CAR positive cells in peripheral blood

  10. Safety and tolerability of CAR-BCMA T cell therapy [ Time Frame: through 24 months post administration of CAR-T-cells ]
    positivity of ADA (Anti-CAR-T antibody)

  11. Efficacy endpoint of CAR-BCMA T cells after infusion [ Time Frame: through 24 months post administration of CAR-T-cells ]
    Overal response rate (ORR)

  12. Efficacy endpoint of CAR-BCMA T cells after infusion [ Time Frame: through 24 months post administration of CAR-T-cells ]
    Complete Response (CR) /stringent Complete Response (sCR)

  13. Efficacy endpoint of CAR-BCMA T cells after infusion [ Time Frame: through 24 months post administration of CAR-T-cells ]
    ≥Very Good Partial Response (VGPR), including VGPR, CR, sCR

  14. Efficacy endpoint of CAR-BCMA T cells after infusion [ Time Frame: through 24 months post administration of CAR-T-cells ]
    Duration of Response (DOR)

  15. Efficacy endpoint of CAR-BCMA T cells after infusion [ Time Frame: through 24 months post administration of CAR-T-cells ]
    Clinical Benefit Rate (CBR)

  16. Efficacy endpoint of CAR-BCMA T cells after infusion [ Time Frame: through 24 months post administration of CAR-T-cells ]
    Time to Response (TTR)

  17. Efficacy endpoint of CAR-BCMA T cells after infusion [ Time Frame: through 24 months post administration of CAR-T-cells ]
    Minimal residual disease (MRD) negativity

  18. Efficacy endpoint of CAR-BCMA T cells after infusion [ Time Frame: through 24 months post administration of CAR-T-cells ]
    Progression Free Survival (PFS)

  19. Efficacy endpoint of CAR-BCMA T cells after infusion [ Time Frame: through 24 months post administration of CAR-T-cells ]
    Overall Survival (OS)



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Patients and legally acceptable representative must have voluntarily signed ICF and willing to complete the study procedure, after fully understanding of the study.
  2. Age ≥ 18 years and ≤ 75 years, male or female
  3. The patients have received at least 3 prior regimen for MM, (Induction therapy followed by autologous transplantation[ASCT] and maintenance therapy represents one line of therapy, those who have not been treated with ASCT should have documented rationale); For each line of therapy, the patient should have received at least one standard treatment cycle (2016 IMWG) unless the best response to the treatment line is documented as progressive diseases (PD)
  4. The patients should have received treatment with at least one proteasome inhibitor AND one immunomodulatory drug, and have ever been relapsed or deteriorated after treatment with at least one regimen consisting of above-mentioned medications (combination or single use);
  5. Patient should be relapsed within 12 months after the last line of therapy, or not achieved at least minimal response (MR) or disease progressed within 60 days after last line of therapy (IMWG criteria 2016), with documented evidence.
  6. The patients should have measurable disease based on at least one of the following parameters:

    • Serum M-protein ≥ 10 g/L;
    • Urine M-protein ≥ 200 mg/24 hrs;
    • For those whose Serum or Urine M- protein dose not meed the measurable criteria but the light chain type, serum free light chain (FLC): involved FLC level ≥ 10 mg/dL (100 mg/L) provided serum FLC ratio is abnormal
  7. Estimated life expectancy > 12 weeks
  8. ECOG performance score 0-1;
  9. Sufficient venous access for leukapheresis collection, and no other contraindications to leukapheresis
  10. Patients should maintain adequate organ function
  11. Women of childbearing age must undergo a serum pregnancy test with negative results before screening and lymphodepletion preconditioning with fludarabine and cyclophosphamide, and are willing to use effective and reliable method of contraception for at least 1 year after T cell infusion
  12. Men who actively have intercourse with child-bearing potential women must be willing to use effective and reliable method of contraception for at least 1 year after T cell infusion

Exclusion Criteria:

  1. Pregnant or lactating women;
  2. P2. Positive for any following tests: human immunodeficiency virus (HIV) antibody, Treponema Pallidum antibody, hepatitis C virus (HCV) antibody, hepatitis B virus (HBV) surface antigen (HBsAg), HBV e antigen (HBeAg), HBV e antibody, hepatitis B core antibody, HBV DNA;
  3. Patients with any uncontrolled active infection including but not limited to active tuberculosis.
  4. Patients with AEs from previous treatment that have not recovered to Common Terminology Criteria for Adverse Events (CTCAE) ≤ 1, excluding hair loss, neuropathy and other events that the treating physician can determine to be tolerable.
  5. Patients who have ever had any CAR T cell therapy;
  6. Patients who have ever had anti-BCMA therapy;
  7. Patients have received allogeneic stem cell transplantation for treating multiple myeloma;
  8. Patients have received autologous stem cell transplantation less than 12 weeks before leukapheresis;
  9. Patients have received any anti-cancer treatment within 14 days before leukapheresis including but not limited to cytotoxic therapy, proteasome inhibitors, immunomodulatory agents, targeted therapies, epigenetic therapy or experimental drug therapy. If the field of radiation covers ≤ 5% of the bone marrow, the subjects are eligible to participate in the study regardless of the radiotherapy end date;
  10. Patients have received ≥ 5 mg prednisone daily or other equivalent dose of steroids within 14 days before leukapheresis or lymphodepletion;
  11. Patients have plasma cell leukemia, Waldenström macroglobulinemia, POEMS (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes) syndrome or AL amyloidosis;
  12. Patients have been administered live attenuated vaccine 4 weeks before leukapheresis or lymphodepletion
  13. Patients allergic to component of study treatment.
  14. Patients have any of the condition as following within 6 months of ICF sign-off: New York Heart Association (NYHA) stage III or IV congestive heart failure, angina pectoris, myocardial infarction, coronary artery bypass graft, stroke (excluding lacunar stroke), history of clinically significant arrhythmia including but not limited to ventricular arrhythmia, significant QT interval prolongation, uncontrolled blood pressure as defined as systolic > 160 mmHg, diastolic > 100 mmHg, uncontrolled diabetes mellitus, pulmonary thrombolism, other conditions that investigators believe that participating in this clinical trial may endanger the health of the patients
  15. Patients are known to have active autoimmune diseases including but not limited to psoriasis, rheumatoid arthritis and other needs of long-term immunosuppressive therapy
  16. patients are oxygen dependent as defined by the blood oxygen saturation (finger oxygen detection method) can be maintained > 95% only by oxygen inhalation before leukapheresis
  17. Patients with second malignancies in addition to MM are not eligible if the second malignancy has required treatment within the past 5 years or is not in complete remission. There are two exceptions to this criterion: successfully treated cervical carcinoma in situ and non-metastatic basal cell skin carcinoma
  18. Patients have central nervous system (CNS) metastases or CNS involvement (including cranial neuropathies or mass lesions and leptomeningeal disease). Patients with history of spinal cord compression from MM are eligible provided spinal cord compression has been treated with surgery or radiation at least 28 days prior to study entry
  19. Patients are unable or unwilling to comply with the requirements of clinical trial
  20. Patients have received major surgery 2 weeks prior to leukapheresis or 4 weeks prior to lymphodepletion and after the study treatment (excluding cataract and other local anesthesia)
  21. Patients are relatives to investigator or his/her staff, or those who may have an interest in the investigator and/or his/her staff.

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 ClinicalTrials.gov identifier (NCT number): NCT03975907


Contacts
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Contact: Zonghai LI, MD 8621-64355922 zonghaili@carsgen.com
Contact: Xiaochen DONG 8621-64355922 xiaochendong@carsgen.com

Locations
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China, Beijing
Beijing Chaoyang hospital Recruiting
Beijing, Beijing, China, 100000
Contact: Wenming Chen, MD    010-85231000    13910107759@163.com   
China, Jiangsu
The First Affiliated Hospital Of Soochow University Recruiting
Suzhou, Jiangsu, China, 215006
Contact: Chengcheng Fu, MD    0512-65223637    fuzhengzheng@suda.edu.cn   
Sponsors and Collaborators
Carsgen Therapeutics, Ltd.
Beijing Chao Yang Hospital
The First Affiliated Hospital of Soochow University
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Tianjin Medical University General Hospital
First Affiliated Hospital of Zhejiang University
Beijing Hospital
Shanghai Tongji Hospital, Tongji University School of Medicine
First Affiliated Hospital of Wenzhou Medical University
Xiangya Hospital of Central South University
Peking University People's Hospital
Qilu Hospital of Shandong University
Sun Yat-sen University
First Affiliated Hospital, Sun Yat-Sen University
Investigators
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Principal Investigator: Wenming CHEN, MD Beijing Chao Yang Hospital
Principal Investigator: Zhengzheng FU, MD The First Affiliated Hospital of Soochow University
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Responsible Party: Carsgen Therapeutics, Ltd.
ClinicalTrials.gov Identifier: NCT03975907    
Other Study ID Numbers: CT053-MM-01
First Posted: June 5, 2019    Key Record Dates
Last Update Posted: March 25, 2020
Last Verified: March 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Carsgen Therapeutics, Ltd.:
multiple myeloma
car-T
Additional relevant MeSH terms:
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Multiple Myeloma
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
Neoplasms
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Paraproteinemias
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases