Phase 2 Study of Oral Ezatiostat Hydrochloride (Telintra®) in Patients With Lenalidomide (Revlimid®) Refractory or Resistant, Low to Intermediate-1 Risk, Deletion 5q Myelodysplastic Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 2
- 试验地点
- 5
- 主要终点
- Hematologic Improvement-Erythroid (HI-E) rate
研究概览
简要总结
Study TLK199.2107 is a multicenter, single arm, open-label Phase 2 study of oral ezatiostat (Telintra®) in patients with lenalidomide (Revlimid®) refractory or resistant, red blood cell (RBC) transfusion-dependent, Low to Intermediate-1 IPSS risk, del5q Myelodysplastic Syndrome (MDS).
详细描述
Study TLK199.2107 is a multicenter, single arm, open-label Phase 2 study of oral ezatiostat (Telintra®) in patients with lenalidomide (Revlimid®) refractory or resistant, red blood cell (RBC) transfusion-dependent, Low to Intermediate-1 IPSS risk, del5q Myelodysplastic Syndrome (MDS). Independence from red blood cell transfusions, improvement in the levels of red blood cells, white blood cells, and platelets, and the response of the bone marrow were evaluated. Patients received a starting dose of 2000 mg total daily dose in divided doses (1000 mg orally twice daily for three weeks (21 days) on therapy followed by a one-week (7 days) off therapy rest period in four-week (28 days) treatment cycles. Patients continued treatment until documentation of lack of MDS response, MDS progression, unacceptable toxicity, or patient withdrawal from the study.
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
- •Primary or de Novo MDS
- •Low or Intermediate-1 IPSS risk MDS
- •Deletion of the 5q chromosome [del(5q) MDS]
- •Refractory or resistant to lenalidomide (Revlimid)
- •ECOG performance score of 0 or 1
- •Documentation of significant anemia with or without additional cytopenia
- •Adequate kidney and liver function
- •Patients must have discontinued hematopoietic growth factors at least 3 weeks prior to study entry
排除标准
- •Prior allogenic bone marrow transplant for MDS
- •Known sensitivity to ezatiostat (injection or oral tablets)
- •Prior treatment with hypomethylating agent (HMA) (e.g., azacitadine, decitabine)
- •History of MDS IPSS risk score of greater than 1.0
- •Pregnant or lactating women
- •Any severe concurrent disease, infection or comorbidity that, in the judgement of the investigator, would make the patient inappropriate for study entry
- •Oral steroids greater than 10 mg per day. Exceptions: those prescribed for other conditions (such as new adrenal failure, asthma, arthritis) or brief steroid use (such as tapered dosing for an acute non-MDS condition)
- •History of hepatitis B or C, or HIV
研究组 & 干预措施
ezatiostat hydrochloride (Telintra®)
Patients received ezatiostat at a starting dose of 2000 mg total daily dose in divided doses (1000 mg PO b.i.d.) for three weeks (21 days) on therapy followed by a one-week (7 days) off therapy rest period in four-week (28 days) treatment cycles.
干预措施: ezatiostat hydrochloride (Telintra®) (Drug)
结局指标
主要结局
Hematologic Improvement-Erythroid (HI-E) rate
时间窗: At 8, 16, 24, and 32 weeks of treatment
Hematologic Improvement response will be assessed per the IWG MDS response criteria (2006)
次要结局
- Hematologic Improvement-Platelet (HI-P) rate(At 8, 16, 24, & 32 weeks of treatment)
- Unilineage, bilineage, trilineage, and overall HI response rate(2 years)
- Cytogenetic response rate(16 weeks, 48 weeks and at the time of first HI response)
- Duration of response(2 years)
- RBC Transfusion independence (TI) rate(At 4, 8, 12, 16, 20, 24, 28 & 32 weeks of treatment)
- Hematologic Improvement-Neutrophil (HI-N) rate(At 8, 16, 24, & 32 weeks of treatment)
- Safety of ezatiostat in this MDS population(At 4, 8, 12, 16, 20, 24, 28 & 32 weeks of treatment)
- Evaluation of the relationship between HI-E response, gene expression profiling and response-related variables(2 years)