Prospective, Multicenter, Open Label and Single-arm Study of Darbepoetin Alfa for Anemia in Myelodisplastic Syndrome Patients.
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 80
- 试验地点
- 32
- 主要终点
- Proportion of patients achieving an erythroid response during the 24-week treatment period.
研究概览
简要总结
This is an open-label, single-arm, multicentre, prospective study of darbepoetin alfa to treat anaemia in patients with low and intermediate-1 IPSS risk MDS. The study will consist of a 14-day screening period followed by a maximum 24-week treatment period and a final visit.
详细描述
This is an open-label, single-arm, multicentre, prospective study of darbepoetin alfa to treat anaemia in patients with low and intermediate-1 IPSS risk MDS. The study will consist of a 14-day screening period followed by a maximum 24-week treatment period and a final visit. Darbepoetin alfa will be initiated at a dose of 300 mcg QW SC over a period of 8 weeks. After 8 weeks, erythroid response will be evaluated, and treatment algorithm adapted to it.
The study treatment period will last for a maximum of 24 weeks. The treatment will end at the start of week 24. If the scheduled 24-week treatment period is not completed, it will end during the week of the last administration of the study drug.
The follow-up period will last for a minimum of 4 weeks and a maximum of 8 weeks after the last dose of darbepoetin alfa.
Subjects will be stratified at enrolment according to IPSS (low risk versus intermediate-1 risk).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 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
- •Age ³ 18 years
- •Low or intermediate-1 risk MDS according to IPSS, and FAB classification of RA, RARS, or RAEB with blasts £ 10%
- •Predictive variables of good response (serum erythropoietin levels < 500 IU/l and transfusion requirements < 2 packed RBC/month over the preceding 2 months)
- •Anaemia (Hb £ 10 g/dL), confirmed in the 14 days before day 1 of the study
- •Life expectancy of at least 6 months
- •ECOG Performance status score of 0, 1, or 2
- •Subject must sign and date the Informed Consent (approved by a Clinical Research Ethics Committee - CREC), before any study-specific procedure is performed
排除标准
- •Known history of convulsive disorders
- •Poorly controlled hypertension (diastolic blood pressure > 100 mmHg) at screening
- •Inadequate liver function (total bilirubin > two times the upper limit of the normal range (ULN), and liver enzymes (ALT, AST) > two times ULN)
- •Inadequate renal function (serum creatinine concentration > 2 mg/dL)
- •Ferritin < 100 ng/ml or transferrin saturation index (TSI) < 16%; Vitamin B12 deficiency (< 200 pg/ml) or folate deficiency (< 2 ng/ml)
- •Clinically-relevant haemorrhages
- •Haemolytic anaemia
- •Cardiac condition: uncontrolled angina, congestive heart failure, or uncontrolled cardiac arrhythmia
- •Clinically significant systemic infection or chronic inflammatory disease present at time of screening
- •Any concomitant therapy used to treat MDS (including other growth factors than those described as part of this protocol, chemotherapy, antibody-based cancer treatment, hormonal therapy, interferon, and interleukins)
- •Treatment with rHuEPO or darbepoetin alfa over the 4 weeks prior to Day 1 of the study
- •More than 2 RBC transfusions over the 28 days prior to Day 1 of the study
- •Pregnant or breast feeding women
- •Subjects of childbearing-potential who do not take adequate contraceptive measures, in the opinion of the investigator
- •Known hypersensitivity to any mammal-derived recombinant product
结局指标
主要结局
Proportion of patients achieving an erythroid response during the 24-week treatment period.
时间窗: weeks 8; 12; 16 and 24
次要结局
- Time to erythroid response and time it is maintained.(week 24)
- Proportion of non-responders to darbepoetin alfa who obtain an erythroid response after the addition of Filgrastim(weeks 8, 12, 16 and 24)
- Proportion of patients receiving RBC transfusions (more than 1 unit) from week 5 to 24, inclusive(weeks 8; 12; 16 and 24)
- Score changes in the FACT-Fatigue quality-of-life scale between the baseline visit, and weeks 8, 16, 24, and the end of the study.(weeks 8; 16 and 24)
- Number of morphological and cytogenetic disorders at baseline and end of treatment(week 24)
- Incidence of adverse events and serious adverse events(weeks 8; 12; 16 and 24)
- Proportion of patients with haemoglobin values over 12 g/dL at any time during the study(weeks 8; 12; 16 and 24)