Phase I, open label, study of B-cell Maturation Antigen (BCMA)-directed CAR-T cells in adult patients with multiple myeloma

Protocol # :
Multiple Myeloma
Disease Sites
Multiple Myeloma
Principal Investigator
Sperling, Adam
Site Research Nurses
Aiken, Haley
Coleman, Kimberly, C.
Gammon, Marilyn
Hogan, Sarah
Kendricken, Elizabeth
Lively, Kathleen, J.
Maciejewski, Ashley, Taylor
O'Brien, Alexandra
Smith, Caitlin
Tichon, Jennifer
Valles, Betsy, J.
Weigel, Susan

Trial Description

This is a first-in-human study to evaluate the feasibility, safety and preliminary
antitumor efficacy of autologous T cells genetically engineered with a novel B-cell
Maturation Antigen (BCMA)-specific chimeric antigen receptor (CAR) and manufactured with
a new process. CAR-T cells will be investigated as a single agent in multiple myeloma

Eligibility Requirements

Inclusion Criteria:

- Part A: Subjects with MM who are relapsed and/or refractory to at least 2 prior
treatment regimens, including an IMiD (e.g. lenalidomide or pomalidomide), a
proteasome inhibitor (e.g. bortezomib, carfilzomib), and an approved anti-CD38
antibody (e.g. daratumumab), if available, and have documented evidence of disease
progression (IMWG criteria)

- Part A: ECOG performance status that is either 0 or 1 at screening

- Part B: Subjects with newly diagnosed multiple myeloma (NDMM) who have received a
minimum of 4 and up to 6 cycles of standard induction therapy with VRd, D-VRd, or
D-Rd, and have achieved a response of PR or better. One cycle of CyBorDex is allowed
prior to induction.

- Part B: ECOG performance status that is either 0,1 or 2 at screening

- Measurable disease as defined by the protocol

- Adequate hematological values

- Must have a leukapheresis material of non-mobilized cells accepted for manufacturing

Exclusion Criteria:

- Prior administration of a genetically modified cellular product including prior BCMA
CAR-T therapy. Patients who have received prior BCMA-directed bi-specific antibodies
or antibody-drug conjugates (ADC) are not excluded.

- Autologous HSCT within 6 weeks prior to enrollment or any prior history of
allogeneic hematopoietic stem cell transplant (HSCT)

- Chemotherapy or any concomitant anti-cancer therapies (other than protocol
prescribed lymphodepletion (LD) chemotherapy) within 2 weeks prior to apheresis

- Treatment with small molecule targeted antineoplastics within 2 weeks of apheresis
collection or 5 half-lives whichever is shorter

- Have received antibodies or immunotherapies (other than daratumumab) within 4 weeks
prior to apheresis collection. Daratumumab within 3 weeks prior to apheresis