Phase I/II Trial of Sodium Stibogluconate in Myelodysplastic Syndrome

Official Title: “SHP2 as a Therapeutic Target For Myelodysplastic Syndrome: Phase I/II Trial of Sodium Stibogluconate in Myelodysplastic Syndrome”

Sodium stibogluconate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

This phase I/II trial is studying the side effects of sodium stibogluconate and to see how well it works in treating patients with myelodysplastic syndromes.

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.

Intervention(s) in this Clinical Trial

• Drug: sodium stibogluconate
  • Sodium stibogluconate 900 mg/m2/day will be given on Monday through Friday every other week for the first 16 weeks of the study (on the 1st, 3rd, 5th, 7th, 9th, 11th, 13th and 15th weeks). On the alternate weeks patients will not receive any study treatment.

Arms, Groups and Cohorts in this Clinical Trial

• Experimental: Sodium stibogluconate
  • Sodium stibogluconate 900 mg/m2/day will be given on Monday through Friday every other week for the first 16 weeks of the study (on the 1st, 3rd, 5th, 7th, 9th, 11th, 13th and 15th weeks). On the alternate weeks patients will not receive any study treatment.

Primary Measures

• determine the effect of SSG treatment on clinical parameters of MDS
  • Time Frame: Weeks 2 and 4 of each cycle for 24 Weeks then every other month for 6 months then every 3 months for 12 months then every 6 months for 2 years
    Safety Issue?: No
• Determine the effect of SSG treatment on hematopoiesis in MDS subjects
  • Time Frame: Weeks 2 and 4 of each cycle for 24 Weeks then every other month for 6 months then every 3 months for 12 months then every 6 months for 2 years
    Safety Issue?: No

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

Gender Eligibility for this Clinical Trial: Both

Minimum Age for this Clinical Trial: 18 Years

Maximum Age for this Clinical Trial: N/A

Are Healthy Volunteers Accepted for this Clinical Trial?: No

Lead Investigator: Northwestern University Other

Overall Clinical Trial Officials and Contacts

Elizabeth Eklund, MD Principal Investigator Northwestern University  

Overall Contact: Elizabeth Eklund, MD 312-503-4625 e-eklund@northwestern.edu

Information obtained from ClinicalTrials.gov on February 09, 2012

Link to the current ClinicalTrials.gov record. http://clinicaltrials.gov/show/NCT01009502

Study ID Number: NU 08H4

ClinicalTrials.gov Identifier: NCT01009502

Health Authority: United States: Food and Drug Administration

Clinical trial summary from the National Cancer Institute's PDQ® database