Relapsed/Refractory Multiple Myeloma

2010-35 A Phase II Study of Expanded Natural Killer Cell Therapy for Multiple Myeloma

Principal Investigator: Dr. van Rhee

Primary Objective:

To determine the therapeutic efficacy of the exp-NK cell therapy in research participants with relapsed high risk MM [defined as gene expression profile (GEP) 70 gene score ≥0.66 and/or GEP 80 gene score of ≥ 2.48 and/or metaphase chromosomal abnormalities and/or high LDH ≥ 360U/L] by establishing the (near) complete response rate. Response rate will be compared to case matched historical controls (patients who relapsed on Total Therapy 2 or 3 with high-risk MM defined as above). Disease-free survival and overall survival will be captured but are not primary or secondary endpoints.

Secondary Objectives:

To monitor the persistence of exp-NK cells by molecular methods in recipients of haploidentical exp-NK cells;

To determine whether escape from exp-NK cell recognition is the mechanism for relapse in those who fail the proposed therapy

Eligibility:

Patients with high-risk disease; Previously-treated patients; Relapsed/Refractory patients


2011-07 Phase 1/2 Open-Label, Multiple-Dose, Dose-Escalation Study to Evaluate the Safety and Tolerability of SNS01-T Administered by Intravenous Infusion in Patients with Relapsed or Refractory B Cell Malignancies

Principal Investigator:  Dr. van Rhee

Primary objective:

To evaluate the safety and tolerability of multiple escalating doses of SNS01-T when administered by intravenous infusion to patients with relapsed or refractory multiple myeloma, MCL, or DLBCL by assessing the frequency, severity, and duration of treatment-related adverse events (AEs) and monitoring changes in vital signs, physical examination, and laboratory values

Secondary objectives: 

To characterize the pharmacokinetic profile of multiple doses of SNS01-T administered intravenously to patients with relapsed or refractory multiple myeloma, MCL or DLBCL by measuring pExp5A plasmid DNA and eIF5A siRNA in blood and bone marrow;

To assess the potential immunogenicity of SNS01-T administered intravenously to patients with relapsed or refractory multiple myeloma, MCL or DLBCL by measuring serum concentrations of antibodies against SNS01-T nanoparticles;

To measure the serum concentrations of select proinflammatory cytokines;

To determine the effect of SNS01-T on time to relapse or progression To determine the effect of SNS01-T on tumor response in multiple myeloma using the International Myeloma Working Group (IMWG) uniform response criteria by analyzing the following:

Changes in serum and urine values of monoclonal protein (M-protein), immunoglobulin (Ig) kappa and lambda free light chain (FLC) and ratio, β2-microglobulin, lactate dehydrogenase, hemoglobin, and C-reactive protein

Changes in bone marrow plasma cell percentage and plasma cell labeling index

To determine the preliminary response of SNS01-T in MCL and DLBCL using the Revised Response Criteria for Malignant Lymphoma, and by assessing changes in appropriate signs, symptoms and lab results