CART Therapy for Multiple Myeloma

Recruiting in Palo Alto (17 mi)

Overseen byAlfred Garfall, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: University of Pennsylvania

No Placebo Group

Approved in 1 Jurisdiction

Trial Summary

What is the purpose of this trial?This is an open-label phase 1 study to assess the safety and pharmacodynamics of CART-BCMA, with or without huCART19, in patients responding to first- or second-line therapy for high-risk multiple myeloma. The regimen evaluated in this study is based on established safety of CARTBCMA demonstrated in UPCC 14415/IRB#822756 at dose of 5x108 cells, administered as split infusions, following cyclophosphamide 1.5 g/m2 in patients with relapsed/refractory myeloma. This study tests CART-BCMA (1) as consolidation of early therapy for multiple myeloma, (2) with addition of fludarabine to the lymphodepleting chemotherapy regimen, (3) in combination with huCART19, and (4) as a single rather than split-dose infusion.

Eligibility Criteria

This trial is for adults with high-risk multiple myeloma who have started treatment within the last year and seen some improvement but not a complete cure. They shouldn't have had stem cell transplants or certain chemotherapies, and their vital organs must be functioning well. Participants need to agree to birth control methods if applicable.

Inclusion Criteria

I am 18 years old or older.

My vital organs are functioning well.

Your beta-2-microglobulin level is higher than 5.5 mg/L and your LDH level is higher than the normal range.

+18 more

Exclusion Criteria

I do not have active hepatitis B, C, HIV, or any uncontrolled infection.

My myeloma has affected or is affecting my brain or spinal cord.

Any uncontrolled medical or psychiatric disorder that would preclude participation

+3 more

Participant Groups

The study tests a single-dose infusion of CART-BCMA alone or combined with huCART19 in patients after initial therapy success. It explores different chemotherapy regimens, combination treatments, and dosing strategies to improve outcomes for those at high risk.

4Treatment groups

Experimental Treatment

Group I: Phase CExperimental Treatment1 Intervention

Single-dose infusion phase to test the safety of single-dose infusion of CART-BCMA alone (Cohort 1) and CART-BCMA + huCART19 (Cohort 2) as single-dose infusions after lymphodepleting chemotherapy with cyclophosphamide + fludarabine in patients responding to first- or second-line therapy.

Group II: Phase BExperimental Treatment1 Intervention

Randomization Phase in which patients responding to first or second-line therapy will receive either CART-BCMA alone (Cohort 1) or CART-BCMA + huCART19 (Cohort 2) as split-doses after lymphodepleting chemotherapy with cyclophosphamide + fludarabine.

Group III: Phase A ExpansionExperimental Treatment1 Intervention

Once safety of CART-BCMA/huCART19 combination therapy is established in Phase A, an expansion of Phase A will occur in which the Phase A target population (patients with relapsed/refractory multiple myeloma responding to a standard salvage therapy regimen) will receive both CART-BCMA and huCART19. Enrollment into the Phase A Expansion may occur concurrently with Phase B once opened.

Group IV: Phase AExperimental Treatment1 Intervention

Safety Run-in to test the safety of CART-BCMA + huCART19 as split-dose infusions after lymphodepleting chemotherapy with cyclophosphamide + fludarabine in patients who have relapsed/refractory myeloma after two prior regimens but who are responding to their current therapy.

CART-BCMA is already approved in United States for the following indications:

🇺🇸 Approved in United States as CART-BCMA for: