A Phase 1, open-label, single ascending dose (SAD) study of P-BCMA-101 autologous T stem cell
memory (Tscm) CAR-T cells in patients with relapsed / refractory MM. Followed by a Phase 2
open-label efficacy and safety study. Rimiducid may be administered as indicated.
Phase 1 follows a 3 + 3 design of dose-escalating cohorts. Phase 2 of the study is an
open-label multi-center efficacy and safety study containing a two arm cycle dosing regime.
After a patient enrolls, leukapheresis will be performed to obtain peripheral blood
mononuclear cells which will be sent to a manufacturing site to produce P-BCMA-101 CAR-T
cells. The cells will then be returned to the investigational site and, after a standard
chemotherapy based conditioning regimen, will be administered to the patient across 1-3
infusions. Treated patients will undergo serial measurements of safety, tolerability and
response. Rimiducid may be administered as indicated.
- Males or females, ≥18 years of age
- Must have a confirmed diagnosis of active MM
- Must have measurable MM
- Must have relapsed / refractory MM, having received treatment with proteasome
inhibitor and IMiD [Phase 2: Must have relapsed / refractory MM, and refractory to
last line of therapy, having received treatment with proteasome inhibitor, an IMiD,
CD38 targeted therapy and undergone autologous stem cell transplant (ASCT) or not a
candidate for ASCT.]
- Must have adequate hepatic, renal, cardiac and hematopoietic function
- Is pregnant or lactating
- Has inadequate venous access and/or contraindications to leukapheresis
- Has active hemolytic anemia, plasma cell leukemia, Waldenstrom's macroglobulinemia,
POEMS syndrome, disseminated intravascular coagulation, leukostasis, amyloidosis,
significant autoimmune, CNS or other malignant disease
- Has an active second malignancy (not disease-free for at least 5 years) in addition to
MM, excluding low-risk neoplasms such as non-metastatic basal cell or squamous cell
- Has active autoimmune disease
- Has a history of significant central nervous system (CNS) disease, such as stroke,
- Has an active systemic infection
- Has hepatitis B or C virus, human immunodeficiency virus (HIV), or human
T-lymphotropic virus (HTLV) infection, or any immunodeficiency syndrome.
- Has any psychiatric or medical disorder that would preclude safe participation in
and/or adherence to the protocol
- Has receiving immunosuppressive or other contraindicated therapies within the excluded
time frame from entry
- Has CNS metastases or symptomatic CNS involvement
- Has a history of having undergone allogeneic stem cell transplantation, or any other
allogeneic or xenogeneic transplant, or has undergone autologous transplantation
within 90 days.