EUCTR2014-002388-13-IE进行中（未招募）1 期
A Phase 2, multicenter, open-label study to evaluate the pharmacokinetics, pharmacodynamics, safety and activity of azacitidine and to compare azacitidine to historical controls in pediatric subjects with newly diagnosed advanced myelodysplastic syndrome or juvenile myelomonocytic leukemia before hematopoietic stem cell transplantation.
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 55
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
入选标准
- •1. Patient has newly diagnosed advanced primary or secondary MDS with an amount of immature cells in blood or bone marrow or chromosomal abnormality linked to secondary MDS. Blood or bone marrow samples confirming diagnosis within 14 days prior to ICF as for the MDS.
- •1. Patient has newly diagnosed JMML, with samples from blood and bone marrow confirming diagnosis within the 14 days prior to informed consent/informed assent signature, with specific alteration in genes (which carry the information that determines a person characteristics) in the body
- •Both MDS and JMML:
- •2.Patient has a Lansky play score/ Karnofsky performance status at least equal to 60
- •3.Patient has a normal renal function and a normal liver function.
- •4.Subjects should be between 1 month to less than 18 years at time of signing ICF/ IAF
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 55
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •MDS exclusions:
- •1.Patient has an illness caused by 'genetic defects' (which cause abnormalities in the information that determine a person's characteristics).
- •2.Patient has inherited disease that cause bone marrow (the soft tissue inside of the bone) failures.
- •JMML Exclusion:
- •1.Patient has a specific deviation in so called Germline.
- •1.Patient has any other organ dysfunction that will interfere with the administration of the therapy according to this protocol.
- •2.Hypersensitivity to azacitidine
研究者
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To determine the safety, clinical efficacy, and effect of azacitidine on the body,within the body and interactions within the body in children and young adults with newly diagnosed advanced Myelodysplastic Syndromes or Juvenile myelomonocytic leukemia before transplantation of the bone marrow and blood. At the end of the study, the data will be compared to data from an older study of patients with the same diseases.EUCTR2014-002388-13-ESCelgene Corporation55
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进行中（未招募）
1 期
To determine the safety, clinical efficacy, and effect of azacitidine on the body,within the body and interactions within the body in children and young adults with newly diagnosed advanced Myelodysplastic Syndromes or Juvenile myelomonocyctic leukemia before transplantation of the bone marrow and blood. At the end of the study, the data will be compared to data from an older study of patients with the same diseases.EUCTR2014-002388-13-SECelgene Corporation55
进行中（未招募）
不适用
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