High Throughput Drug Sensitivity and Genomics Data in Developing Individualized Treatment in Patients With Relapsed or Refractory Multiple Myeloma or Plasma Cell Leukemia

PRIMARY OBJECTIVES:

I. To assess the frequency of obtaining an actionable result from the assay and to estimate
feasibility as defined as a frequency of at least 50%.

II. To test patient cells in a high-throughput assay against individual drugs and drug
combinations within 21 days, to enable optimal choice of drug combinations for therapy.

SECONDARY OBJECTIVES:

I. To assess the response rate among patients treated after physicians are presented with the
testing results.

OUTLINE:

Patients undergo collection of bone marrow aspirate and blood for high-throughput drug
sensitivity assay and mutational analysis using next generation sequencing. Patients and
their treating physicians receive the results of the tests. Treatment decisions are then made
by the patients and their treating physicians.

After completion of study, patients are followed up every 3 months for 2 years.

Inclusion Criteria:

- Diagnosis of multiple myeloma with documented relapsed or refractory disease according
to International Myeloma Working Group (IMWG) criteria, or relapsed/refractory plasma
cell leukemia

- Collection of a bone marrow, fluid or tissue sample that is expected to have enough
cells to run the assay

- Measurable disease defined by one of the following:

- Serum monoclonal protein >= 0.5 g/dL by serum protein electrophoresis (SPEP)

- >= 200 mg/monoclonal protein in urine on 24 hr urine protein electrophoresis
(UPEP)

- Involved serum free light chain (FLC) >= 10 mg/dL and abnormal
involved:uninvolved ratio

- Plasma cytomas that are palpable per exam or measurable per standard radiologic
review

- Circulating plasma cells >= 2,000 if diagnosis of plasma cell leukemia

- Minimum of 3 prior lines of therapy including an immunomodulatory drug (IMiD) and a
proteasome inhibitor (PI)

- Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0-3

- Female patients of child bearing potential and non-vasectomized male patients agree to
practice appropriate methods of birth control

- Ability to understand purpose and risks of the study and provide signed and dated
informed consent, and authorization to use protected health information

- Expected survival is > 100 days

- Adequate organ function as determined by the investigator

Exclusion Criteria:

- Mucosal or internal bleeding, or platelet transfusion refractory

- Any medical conditions that would impose excessive risk to the patient, or would
adversely affect his/her participation in the study

- Known active infection requiring antibiotics within 7 days of initiation of study
treatment, unless considered controlled in the opinion of the investigator

- Other malignancy with life expectancy < 1 year due to the other malignancy

- Pregnant or breast feeding women

- Serious psychiatric illness, alcoholism, or drug addiction

- Human immunodeficiency virus (HIV), or active hepatitis B or C infection

- Previous treatments for multiple myeloma (MM) within 2 weeks of initiation of study
treatment

- Prior autologous or allogeneic stem cell transplantation (SCT) within 12 weeks of
initiation of study treatment

- Prior allogeneic hematopoietic cell transplantation (HCT) with active graft versus
host disease (GVHD) on therapeutic dosing of immunosuppression or prednisone > 20 mg
daily equivalent

- Prior major surgical procedure or radiation treatment within 2 weeks of initiation of
study treatment (not including limited radiation used for palliation of bone pain)