SHP2 as a Therapeutic Target For Myelodysplastic Syndrome: Phase I/II Trial of Sodium Stibogluconate in Myelodysplastic Syndrome
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 1
- 试验地点
- 2
- 主要终点
- Determine the effect of SSG treatment on hematopoiesis in MDS subjects
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
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.
干预措施: sodium stibogluconate (Drug)
结局指标
主要结局
Determine the effect of SSG treatment on hematopoiesis in MDS subjects
时间窗: 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
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.
determine the effect of SSG treatment on clinical parameters of MDS
时间窗: 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
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.
次要结局
未报告次要终点
研究者
Elizabeth Eklund
Elizabeth Eklund, MD
Northwestern University