Phase I/II Trial of Sodium Stibogluconate in Myelodysplastic Syndrome

Patients receive sodium stibogluconate IV over 30 minutes on days 1-5 and 15-19. Treatment
repeats every 28 days for 4 courses in the absence of disease progression or unacceptable
toxicity. Patients who respond to treatment may continue therapy until disease progression.

Patients undergo bone marrow aspiration, biopsy, and peripheral blood sample collection
periodically for correlative laboratory studies.

After completion of study treatment, patients are followed up at 8 weeks.

Inclusion Criteria:

- Documented myelodysplastic syndromes (MDS), including therapy-related MDS

- Meets 1 of the following criteria:

- Refractory to prior azacitidine or decitabine

- Did not tolerate treatment with azacitidine or decitabine due to cytopenias or
other side effects

- Not a candidate for azacitidine or decitabine due to cytopenias or other medical
conditions that would contraindicate nucleoside analogues

- Refused treatment with azacitidine or decitabine

- Life expectancy ≥ 16 weeks

- Not pregnant or nursing

- No B12 deficiency, folate deficiency, or pyridoxine responsive anemia as confirmed by
relevant laboratory testing

- No prolongation of QTc or ventricular ectopic beats on EKG

- No evidence of cardiac disease

- No active infection AND afebrile

- More than 21 days since prior azacitidine or decitabine

- More than 21 days since other prior treatment for MDS (e.g., thalidomide, valproic
acid, or other agents as part of a clinical trial)

- Prior cytokines (e.g., erythropoietin, G-CSF, and GM-CSF) allowed

- Prior chemotherapy and/or radiotherapy for solid tumors or lymphoma allowed provided
there is no evidence of active disease from the prior malignancy

Exclusion Criteria:

- Prior treatment for leukemia (e.g., acute myeloid leukemia, chronic myelogenous
leukemia, acute lymphocytic leukemia, or chronic lymphocytic leukemia)

- Concurrent cytokines

- Concurrent antileukemic treatment, including bone marrow transplantation and
radiotherapy