GO-203-2C + Bortezomib For Relapsed Or Refractory MM

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