Phase I/II Trial of Sodium Stibogluconate in Myelodysplastic Syndrome

Myelodysplastic Syndromes Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01009502
• Other study ID #: NU 08H4
• Secondary ID: 2008-0807STU0000
• Status: Terminated
• Phase: Phase 1
• First received: November 5, 2009
• Last updated: November 6, 2013
• Start date: July 2009
• Est. completion date: May 2013

Study information

• Verified date: November 2013
• Source: Northwestern University
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationUnited States: Institutional Review Board
• Study type: Interventional

Clinical Trial Summary

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

This was originally designed as a phase I/II trial studying the side effects of sodium stibogluconate and how well it works in treating patients with myelodysplastic syndromes. Unfortunately, due to funding issues, the phase II portion was never conducted.

Description:

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.

The phase II portion of this trial was never conducted due to lack of funding.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 1
• Est. completion date: May 2013
• Est. primary completion date: March 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Study Design

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Myelodysplastic Syndromes
• Preleukemia
• Syndrome

Intervention

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.

Locations

Country	Name	City	State
United States	Jesse Brown VHA Medical Center	Chicago	Illinois
United States	Northwestern University	Chicago	Illinois

Sponsors (2)

Lead Sponsor	Collaborator
Northwestern University	Robert H. Lurie Cancer Center

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	determine the effect of SSG treatment on clinical parameters of MDS	To determine the effect of SSG treatment on clinical parameters of MDS. This will include determination of cytopenias, bone marrow dysplasia, % myeloid blasts, transfusion frequency, incidence of infection and phagocyte function in MDS subjects pre and during treatment. Serum Sb levels will also be determined as an early indication of toxicity.	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	No
Primary	Determine the effect of SSG treatment on hematopoiesis in MDS subjects	To determine the effect of SSG treatment on hematopoiesis in MDS subjects. This will include determination of cytokine hypersensitivity, apoptosis resistance, and altered expression of key HoxA10 and ICSBP target genes in the bone marrow of subjects pre and during treatment.	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	No