Clinical trial NCT01009502: 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
Conditions: Myelodysplastic Syndromes
Lead sponsor: Northwestern University
Sponsor class: OTHER
Collaborators: name: Robert H. Lurie Cancer Center
First posted: 2009-11-06
Enrollment: 1
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: determine the effect of SSG treatment on clinical parameters of MDS
timeFrame: 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
measure: Determine the effect of SSG treatment on hematopoiesis in MDS subjects
timeFrame: 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
Secondary outcomes:
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Chicago
country: United States
facility: Jesse Brown VHA Medical Center
state: Illinois
Study officials:
affiliation: Northwestern University
name: Elizabeth Eklund, MD
role: PRINCIPAL_INVESTIGATOR
References: