BCMA CAR-T Cell Therapy for Multiple Myeloma

Overseen by James N Kochenderfer, M.D.

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: National Cancer Institute (NCI)

No Placebo Group

Trial Summary

What is the purpose of this trial?

Background: Multiple myeloma is a cancer of the blood plasma cells. It usually becomes resistant to standard treatments. Researchers have developed a procedure called gene therapy. It uses a person's own T cells, which are part of the immune system. The cells are changed in a lab and then returned to the person. Researchers hope the changed T cells will be better at recognizing and killing tumor cells. Objective: To test the safety of giving changed T cells to people with multiple myeloma. Eligibility: Adults ages 18-73 who have been diagnosed with multiple myeloma that has not been controlled with standard therapies. Design: Participants will be screened with: Medical history Physical exam Blood tests Heart function tests Bone marrow sample taken by needle in a hip bone. Scan of the chest, abdomen, and pelvis. They may have a brain scan. Pregnancy test Participants will have apheresis. Blood will be removed through an arm vein. The blood will be separated, and T cells removed. The rest of the blood will be returned through a vein in the other arm. Participants will have a central line placed in a large vein in the arm or chest. Participants will get 2 chemotherapy drugs by the central line over 3 days. Two days later, participants will get the changed T cells by the central line. They will stay in the hospital at least 9 days. Participants must stay near the hospital for 2 weeks. Participants will have 8 follow-up visits over the next year for blood and urine tests. They may have scans. Participants blood will be collected regularly over the next several years.

Research Team

James N Kochenderfer, M.D.

Principal Investigator

National Cancer Institute (NCI)

Eligibility Criteria

Adults aged 18-73 with multiple myeloma resistant to standard treatments, who have tried at least three different therapies including IMiDs and proteasome inhibitors. Participants must not be HIV or hepatitis positive, should have adequate organ function, and agree to use birth control. Those with certain other health conditions or treatments are excluded.

Inclusion Criteria

My platelet count is above 55,000 without recent transfusions.

I have stored blood cells not modified by gene therapy.

Less than half of my bone marrow cells are plasma cells.

Exclusion Criteria

I need urgent treatment because my tumor is pressing on my spine or other organs.

I have multiple myeloma and no other cancer needing treatment in the last 3 years, except for certain skin cancers.

I have an active autoimmune disease like psoriasis or rheumatoid arthritis.

Treatment Details

Interventions

• Anti-BCMA CAR T cells (CAR T-cell Therapy)

Trial Overview The trial is testing a gene therapy using the patient's own T cells that are modified in the lab to target cancer cells more effectively. It includes chemotherapy drugs Cyclophosphamide and Fludarabine followed by infusion of these engineered T cells.

Participant Groups

Group I: 2/Conditioning chemotherapy plus chimeric antigen receptors (CARs) T-cells expansion phase Experimental Treatment 3 Interventions

6.0x10\^6 dose (maximum feasible dose) of CAR T Cells + Cyclophosphamide: 300 mg/m\^2 intravenous (IV) infusion over 30 minutes on days -5, -4 and -3 + Fludarabine: 30 mg/m\^2 IV infusion over 30 minutes administered immediately following the cyclophosphamide on days -5, -4, and -3

Group II: 1/Conditioning chemotherapy plus chimeric antigen receptors (CARs) T-cells dose escalation Experimental Treatment 3 Interventions

Patients will receive escalating doses (up to 5 planned) of CAR+ T cells infused on day 0 + Cyclophosphamide: 300 mg/m\^2 intravenous (IV) infusion over 30 minutes on days -5, -4 and -3 + Fludarabine: 30 mg /m\^2 IV infusion over 30 minutes administered immediately following the cyclophosphamide on days -5, -4, and -3

Who Is Running the Clinical Trial?

National Cancer Institute (NCI)

Lead Sponsor

Trials

14,080

Recruited

41,180,000+

Dr. Douglas R. Lowy

National Cancer Institute (NCI)

Chief Executive Officer since 2023

MD from New York University School of Medicine

Dr. Monica Bertagnolli

National Cancer Institute (NCI)

Chief Medical Officer since 2022

MD from Harvard Medical School