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临床试验/NCT05178342
NCT05178342终止2 期

A Phase II, Open-Label, Multicenter Study of Orally Administered CA-4948 for the Treatment of Anemia in Patients With Very Low, Low or Intermediate Risk Myelodysplastic Syndromes (MDS)

University of Leipzig15 个研究点 分布在 1 个国家目标入组 38 人开始时间: 2022年1月1日最近更新:
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适应症
干预措施
相关药物

试验速览

阶段
2 期
状态
终止
发起方
入组人数
38
试验地点
15
主要终点
Erythroid response (HI-E)
研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

Anemia in LR-MDS patients

详细描述

Anemia in non-transfusion dependent (NTD) or transfusion dependent (low or high transfusion burden, LTB/HTB) patients with very low, low or intermediate risk myelodysplastic syndromes

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

  • •Diagnosis of de novo myelodysplastic syndrome (MDS) OR de novo myelodysplastic/myeloproliferative neoplasias (MDS/MPN) including MDS/MPN-RS-T, MDS/MPNu, aCML or CMML
  • •Very low/low/intermediate risk disease: IPSS-R up to 3.5 for MDS; MDS/MPN < 10% bone marrow blasts; for CMML low or intermediate risk according to CPSS-Score
  • •Symptomatic anemia (based on valid and complete hemoglobin and transfusion history):
  • •NTD (non transfusion dependent): < 3 RBC transfusions and mean hemoglobin level <10 g/dl within the last 16 weeks
  • •LTB (low transfusion burden): 3-7 RBC transfusions within the last 16 weeks in at least two transfusion episodes, maximum 3 in 8 weeks
  • •HTB (high transfusion burden): ≥ 8 RBC transfusions within the last 16 weeks, ≥ 4 in 8 weeks
  • •Defined transfusion strategy
  • •No available option of an approved MDS therapy and classification of prior erythropoiesis-stimulating agent (ESA) treatment as follows:
  • •Cohort A: ESA exposed (and refractory or intolerant)
  • •Cohort B: ESA naive AND serum erythropoietin level >200 U/L

排除标准

  • •Compliance with major study procedures
  • •Inability to swallow and retain oral medications (> 10 pills)
  • •Patient does not accept bone marrow sampling during screening and after the treatment
  • •Patient does not accept up to weekly peripheral blood sampling during screening and treatment
  • •ECOG performance status ≥ 3
  • •Inacceptable organ function
  • •Serum creatinine > 2 × ULN or calculated creatinine clearance < 30 ml/min
  • •AST > 2 × ULN or ALT > 2 × ULN
  • •total bilirubin > 2 × ULN (exception >3 × ULN in patients with documented Gilbert's syndrome)
  • •Interfering treatments
  • •Prior treatment with azacitidine or decitabine
  • •Treatment with erythropoiesis stimulating agent (ESA), G-CSF, GM-CSF, lenalidomide, luspatercept and/or another investigational drug or device up to 14 days before registration
  • •Treatment with iron chelation therapy 56 days before registration, except for subjects on a stable or decreasing dose for at least eight weeks prior to inclusion and during study treatment
  • •Major surgery within 28 days prior to registration
  • •Concomitant diseases
  • •Known human immunodeficiency virus infection (HIV)
  • •Active infectious hepatitis (HBV or HCV)
  • •Hepatitis virus detectable within 6 months before registration in patients with a history of hepatitis
  • •History of other invasive malignancy, unless definitively treated with curative intent, provided it is deemed to be at low risk for recurrence by the treating physician
  • •Presence of an acute or chronic toxicity resulting from prior anti-cancer therapy that has not resolved to Grade ≤ 1 (except anemia and alopecia)
  • •Known allergy or hypersensitivity to any component of the formulation of CA-494824
  • •Severe cardiovascular disease (e.g. myocardial infarction within 6 months registration, unstable angina within 6 months registration, NYHA Class III or greater congestive heart failure, serious arrhythmias uncontrolled on treatment, clinically significant pericardial disease, known QTc abnormality > 450 msec on ECG
  • •Formal requirements
  • •Positive serum pregnancy test in women of childbearing potential
  • •Women of childbearing potential and men who partner with a woman of childbearing potential unwilling to use highly effective contraceptive methods for the duration of the study and for 90 days after the last dose of CA-4948
  • •Age under 18 years at registration
  • •Inability to provide written informed consent
  • •Simultaneous participation in another interventional clinical trial or participation in any clinical trial involving administration of an investigational medicinal product within 28 days prior registration

研究组 & 干预措施

CA-4948 treatment

Other

Single-arm design. all patients are treated with IMP

干预措施: CA-4948 (Drug)

结局指标

主要结局

Erythroid response (HI-E)

时间窗: At the end of cycle 4 (each cycle is 28 days).

To evaluate the proportion of patients who have an erythroid response (HI-E) according to the modified IWG 2018 criteria separately for both independent substudies.

次要结局

  • Time to HI-E (erythroid response)(From the date of treatment start until first day of response, assessed up to end of cycle 4 (each cycle is 28 days).)
  • Neutrophil (HI-N) responses(At the end of cycle 4 (each cycle is 28 days).)
  • Platelet (HI-P) responses(At the end of cycle 4 (each cycle is 28 days).)
  • Number of participants with clinically significant changes of selected laborotory parameters (parameters listed in detailed description)(From the date of treatment start until the end of study, assessed up to 30 months.)
  • Impact of treatment assessed by using the validated European Organisation for Research and Treatment of Cancer Core Quality of Life questionnaire (EORTC QLQ-C30)(From the date of treatment start until the end of study, assessed up to 30 months.)
  • HI-E response (erythroid response) duration(From the date of treatment start until date of documented loss of response, assessed up to 30 months.)
  • Safety of CA-4948 (toxicities and adverse events)(From the date of treatment start until the end of study, assessed up to 30 months.)
  • Red blood cell (RBC) transfusions(From the date of treatment start until the date of end of treatment, assessed up to 30 months.)
  • Impact of treatment assessed by using the validated European Organisation for Research and Treatment of Cancer cancer related fatigue questionnaire (EORTC QLQ- FA12)(From the date of treatment start until the end of study, assessed up to 30 months.)

研究者

发起方
University of Leipzig
申办方类型
Other
责任方
Principal Investigator
主要研究者

Uwe Platzbecker

Prof. Dr.

University of Leipzig

研究点 (15)

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