Safety and Efficacy of BCMA-Targeted CAR-T Therapy for Relapsed/Refractory Multiple Myeloma

Phase 1

Recruiting

• Conditions: Multiple Myeloma; Neoplasm, Plasma Cell; Multiple Myeloma in Relapse
• Interventions: Biological: BCMA CAR-T cells

• Registration Number: NCT04272151
• Lead Sponsor: Chongqing Precision Biotech Co., Ltd

Brief Summary

This is a single arm study to evaluate the efficacy and safety of BCMA-targeted CAR-T cells therapy for patients with relapsed/refractory Multiple Myeloma.

Detailed Description

There are limited options for treatment of relapse/refractory Multiple Myeloma. BCMA is expressed on most Multiple Myeloma cells so it is an ideal target for CAR-T. In this study, investigators will evaluate the safety and efficacy of CAR-T targeting BCMA in patients with relapsed/refractory Multiple Myeloma. The primary goal is safety and efficiency assessment, including adverse events and disease status after treatment.

Study Timeline

• Study Start: 2019-12-01
• Study First Submitted: 2020-02-13
• Study First Submitted QC: 2020-02-14
• Study First Posted: 2020-02-17
• Status Verified: 2023-03
• Last Update Submitted: 2023-04-16
• Last Update Posted: 2023-04-18
• Primary Completion: 2023-12-31
• Study Completion: 2024-07-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

1. Signed written informed consent

2. Diagnose as relapsed /refractory multiple myeloma, and meet one of the following conditions:

   1. Failed to standard chemotherapy regimens;
   2. Relapse after complete remission, high-risk and / or refractory patients ;
   3. Relapse after hematopoietic stem cell transplantation;

3. Evidence for cell membrane BCMA expression；

4. All genders, ages: 18 to 75 years；

5. The expect time of survive is above 12 weeks;

6. KPS>60；

7. No serious mental disorders ;

8. Left ventricular ejection fraction ≥50%

9. Sufficient hepatic function defined by ALT/AST≤3 x ULN and bilirubin≤2 x ULN;

10. Sufficient renal function defined by creatinine clearance≤2 x ULN;

11. Sufficient pulmonary function defined by indoor oxygen saturation≥92%;

12. With single or venous blood collection standards, and no other cell collection contraindications;

13. Ability and willingness to adhere to the study visit schedule and all protocol requirements.

Exclusion Criteria

1. Have received CAR-T therapy or other genetically modified cell therapy before screening；
2. Participated in other clinical research within 1 month before screening；
3. Have received the following anti-tumor treatment before screening: Have received chemotherapy, targeted therapy or other experimental drug treatment within 4 weeks, except those who have confirmed disease progression after treatment;
4. Live attenuated vaccine within 4 weeks before screening;
5. Convulsion or stoke within past 6 months;
6. Previous history of other malignancy;
7. Presence of uncontrolled active infection;
8. Subjects with positive HBsAg or HBcAb positive and peripheral blood HBV DNA titer is higher than the lower limit of detection of the research institution; HCV antibody positive and peripheral blood HCV RNA titer is higher than the lower limit of detection of the research institution; HIV antibody positive; syphilis primary screening antibody positive;
9. Pregnant or breasting-feeding women;
10. Any situation that investigators regard not suitable for attending in this study or may affect the data analysis.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
BCMA CAR-T cells treat	BCMA CAR-T cells	Patients will be be treated with BCMA CAR-T cells

Primary Outcome Measures

Name	Time	Method
The response rate of BCMA CAR-T treatment in patients with relapse/refractory Multiple Myeloma that treatment by BCMA CAR-T cells therapy	6 months	The response rate of BCMA CAR-T treatment will be recorded and assessed according to the IMWG
Adverse events that related to treatment	2 years	Therapy-related adverse events will be recorded and assessed according to the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE, Version 5.0)

Secondary Outcome Measures

Name	Time	Method
Levels of IL-6 in Serum	3 months	In vivo (Serum) quantity of IL-6
Rate of BCMA CAR-T cells in bone marrow and peripheral blood	2 years	In vivo (bone marrow and peripheral blood) rate of BCMA CAR-T cells were determined by means of flow cytometry
Progress-free survival(PFS) of BCMA CAR-T treatment in patients with refractory/relapsed multiple myeloma	2 years	PFS will be assessed from the first CAR-T cell infusion to death from any cause or the first assessment of progression (censored)
Quantity of BCMA CAR copies in bone marrow and peripheral blood	2 years	In vivo (bone marrow and peripheral blood) quantity of BCMA CAR copies were determined by means of qPCR
Duration of Response (DOR) of BCMA CAR-T treatment in patients with refractory/relapsed Multiple Myeloma	2 years	DOR will be assessed from the first assessment of sCR/CR/VGPR/PR to the first assessment of recurrence or progression of the disease or death from any cause (censored)
Overall survival(OS) of BCMA CAR-T treatment in patients with refractory/relapsed multiple myeloma	2 years	OS will be assessed from the first CAR-T cell infusion to death from any cause (censored)
Quantity of clonal plasma cells in bone marrow	1 years	In vivo (bone marrow) quantity of clonal plasma cells

Trial Locations

Locations (1)

Chongqing University Cancer Hospital; 🇨🇳 Chongqing, Chongqing, China