GO-203-2C + Bortezomib For Relapsed Or Refractory MM
GO-203-2C + Bortezomib For Relapsed Or Refractory MM
This research study is studying a combination of targeted therapies known as GO-203-2C and
 bortezomib as a possible treatment for multiple myeloma that has either progressed or not
 responded to treatment.
This research study is a Phase I clinical trial, which tests the safety of an investigational
 intervention and also tries to define the appropriate dose of the investigational
 intervention to use for further studies. "Investigational" means that the intervention is
 being studied.

 The FDA (the U.S. Food and Drug Administration) has not approved GO-203-2C as a treatment for
 any disease.

 The FDA (the U.S. Food and Drug Administration) has approved bortezomib as a treatment option
 for your disease.

 The purpose of this research study is to test the safety of GO-203-2C with bortezomib.
 GO-203-2C is a newly discovered compound that binds to an oncoprotein (a cancer causing
 protein) called MUC1. Myeloma cells harbor an increased amount of MUC1 on its cell surface.
 By binding to MUC1, GO-203-2C has been shown to cause tumor cell death in laboratory studies.

 Bortezomib is an intravenously or subcutaneously administered medication that belongs to a
 class of drugs called proteasome inhibitors. Proteasome inhibitors disrupt the normal action
 of cells that breakdown proteins in both normal and cancer cells. This disruption can stall
 tumor growth and cause cancer cells to die. Bortezomib is currently approved for the
 treatment of multiple myeloma.

 The Investigators want to find the highest dose of GO-203-2C given in combination with
 bortezomib that can be administered safely without severe or unmanageable side effects.
Inclusion Criteria:

 -  Participants with multiple myeloma who experienced disease progression after the most
 recent treatment regimen. Patients must have had prior treatment with a proteasome
 inhibitor, immunomodulatory drug, and if eligible for transplant an autologous
 transplant.

 -  Patients must have measurable disease, defined as 1 or more of the following:

 -  serum M-protein > 0.5 g/dL. For patients with IgA myeloma where the M-protein
 cannot be quantified on SPEP, total IgA > 0.5 g/dL.

 -  Urine M-protein > 200mg/24h

 -  serum FLC assay: involved FLC level > 10 mg/dL with abnormal FLC ratio

 -  Greater than or equal to 18 years in age

 -  ECOG performance status ≤2 (see Appendix A)

 -  Life expectancy of greater than 3 months

 -  Participants must have normal organ and marrow function as defined below:

 -  leukocytes ≥2,000/mcL

 -  platelets ≥50,000/mcL

 -  total bilirubin ≤ 2.0 mg/dL (patients with Gilbert syndrome and Bilirubin ≤ 3.5
 mg/dL are eligible)

 -  AST(SGOT)/ALT(SGPT) ≤2.5 × institutional upper limit of normal

 -  creatinine ≤ 2 mg/dL

 --- OR

 -  creatinine clearance ≥60 mL/min/1.73 m2 for participants with creatinine levels
 above institutional normal.

 -  Women of child-bearing potential must have a documented negative pregnancy test.

 -  The effects of GO-203-2c on the developing human fetus are unknown. For this reason,
 women of child-bearing potential and men must agree to use adequate contraception
 (hormonal or barrier method of birth control; abstinence) prior to study entry and for
 the duration of study participation. Should a woman become pregnant or suspect she is
 pregnant while she or her partner is participating in this study, she should inform
 her treating physician immediately. Men treated or enrolled on this protocol must also
 agree to use adequate contraception prior to the study, for the duration of study
 participation, and 4 months after completion of GO-203-2c administration.

 -  Ability to understand and the willingness to sign a written informed consent document

 Exclusion Criteria:

 -  Participants who have had chemotherapy or radiotherapy within 14 days (6 weeks for
 nitrosoureas or mitomycin C) prior to entering the study or those who have not
 recovered from adverse events due to agents administered more than 14 days earlier.
 The use of steroids up the equivalent of 160 mg of dexamethasone is allowed within 14
 days of screening, but the last dose has to be given at least 1 day prior to
 initiation of treatment.

 -  Participants who are receiving any other investigational agents.

 -  Uncontrolled hypertension. This is defined as sustained blood pressure elevation >
 140/90 despite antihypertensive therapy. Patients are allowed to start
 antihypertensive therapy to meet eligibility criteria; however they have to be on a
 stable antihypertensive regimen for at least 7 days.

 -  Uncontrolled intercurrent illness including, but not limited to, uncontrolled
 infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac
 arrhythmia, or psychiatric illness/social situations that would limit compliance with
 study requirements.

 -  Pregnant women are excluded from this study because GO-203-2c is an agent with the
 potential for teratogenic or abortifacient effects. Because there is an unknown but
 potential risk for adverse events in nursing infants secondary to treatment of the
 mother with GO_203-2c, breastfeeding should be discontinued if the mother is treated
 with GO-203-2c. These potential risks may also apply to other agents used in this
 study.

 -  Individuals with a history of a different malignancy are ineligible except for the
 following circumstances: Individuals with a history of other malignancies are eligible
 if they have been disease-free for at least 5 years and are deemed by the investigator
 to be at low risk for recurrence of that malignancy. Individuals with the following
 cancers are eligible if diagnosed and treated within the past 5 years: cervical cancer
 in situ, and basal cell or squamous cell carcinoma of the skin.

 -  Hypersensitivity to bortezomib, boron or mannitol.

 -  Grade 3 or 4 peripheral neuropathy.

 -  Prior discontinuation of a bortezomib-based therapy due to toxicity attributed to
 bortezomib.

 -  Use of G-CSF administration within 7 days of screening

 -  Platelet transfusion within 7 days of screening