The Efficacy and Safety of CDA-2 for the Treatment of IPSS Lower/Intermediate-risk Myelodysplastic Syndrome Patients: a Multi-centered Prospective Open Study
试验速览
- 阶段
- 4 期
- 发起方
- 入组人数
- 800
- 主要终点
- Hematological Improvement (HI) at 12 Weeks
研究概览
简要总结
This Study aims to evaluate the efficacy and safety of CDA-2 in the treatment of International Prognostic Scoring System (IPSS) Lower/Intermediate-risk myelodysplastic syndrome (MDS) in Chinese patients.
详细描述
Patients with lower/intermediate-risk myelodysplastic syndrome (MDS) have rare therapeutic options other than supportive care. In pilot studies, CDA-2 showed promising results of hematological improvement in these patients.
To date, the optimal regimen for CDA-2 treatment is not well established. The researchers are going to make a multi-centered clinical trial to evaluate the efficacy and safety of CDA-2 in 800 patients with International Prognostic Scoring System(IPSS) Lower/Intermediate-risk myelodysplastic syndrome (MDS).
Eligible patients will be given CDA-2 intravenously, with 200 ml each day for 14 consecutive days in every four weeks (one cycle). The treatment will be repeated at least for 3 cycles. The patients will be followed up to 24 weeks.
The primary endpoint is hematological improvement (HI) at 12 weeks according to IWG criteria. Full blood counts will be done on all patients every week. Change in bone marrow function as measured by changes in bone marrow morphology and cytogenetics will be assessed before and after 3 cycles of the treatment.
The secondary endpoint is the therapy response. Complete remission (CR), partial remission (PR) and response duration, side effects, evaluation of QOL will be evaluated at the end of the treatment in every cycle.
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 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Documented diagnosis of MDS according to World Health Organization (WHO)/French American British (FAB) classification that meets IPSS-R classification of low, or intermediate-1 risk disease.
- •Subject is 18 to 85years of age the time of signing the informed consent form (ICF).
- •Able to adhere to the study visit schedule and other protocol requirements
- •Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or
- •Laboratory test results within these ranges: Serum creatinine </=1.5 mg/dL x Upper limit of the normal (ULN),Blood urine nitrogen (BUN)</=1.5 mg/dL x Upper limit of the normal (ULN),Total bilirubin </=1.5 mg/dL x Upper limit of the normal (ULN),Serum glutamic oxaloacetic transaminase/aspartate transaminase (SGOT/AST) and Serum glutamic pyruvic transaminase/alanine transaminase (SGPT/ALT)</=2 x Upper limit of the normal (ULN).
- •No prior intensive combination chemotherapy or dose Azacitidine,Decitabine,and Lenalidomide,etc.
- •Capable of understanding the investigational nature, potential risks and benefits of the study, and able to provide valid informed consent.
排除标准
- •IPSS risk group intermediate-2 or high risk
- •breast feeding and pregnant women
- •MDS associated with del 5q cytogenetic abnormality
- •Patients with history of hepatitis B, C, HIV(+), alcoholic liver disease or evidence of hepatopathy will be excluded.
- •Any significant concurrent disease, illness, or psychiatric disorder that would compromise patient safety or compliance, interfere with consent, study participation, follow up, or interpretation of study results.
研究组 & 干预措施
CDA-2 (Cell Differentiation Agent 2)
Patients will be given CDA-2 therapy.
干预措施: CDA-2 (Cell Differentiation Agent 2) (Drug)
结局指标
主要结局
Hematological Improvement (HI) at 12 Weeks
时间窗: 12 weeks
Hematologic improvement (HI) per International Working Group (IWG),HI: hemoglobin increase of \>= 1.5 g/dL, platelet increase of \>= 30,000/mL (starting with \> 20,000/mL), neutrophils increase of \>= 100% and \> 500/μL.
次要结局
- Response Rate of The Therapy at 12 Weeks(12 weeks)
- Red Blood Cell Transfusion Independence (RBC-TI) in 24 weeks(24 weeks)
- Change From Baseline to that of the 24 weeks of Scores of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire - Core 30 (EORTC QLQ C-30) Physical Functioning Scale(24 weeks)
研究者
Wu Depei
Professor
Chinese Society of Hematology