EUCTR2010-022235-10-NL进行中（未招募）1 期
A Phase I/II study of Azacitidine (Vidaza®) in pediatric patients with relapsed high-grade pediatric MDS or JMML - Azacitidine in high grade MDS and JMML pediatric patients
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- Erasmus MC
- 入组人数
- 24
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
- 性别
- All
入选标准
- •Diagnosis of relapsed advanced primary MDS or JMML, established at initial diagnosis by the diagnostic criteria as specified in the EWOG-MDS 2006 protocol (see appendix 1), and defined as:
- •oRelapsed MDS:
- •After a documented CR or PR, this designation is defined as
- •- a reappearance of blasts in the peripheral blood,
- •- or =5% blasts in the bone marrow not attributable to any other cause (e.g. bone marrow regeneration after consolidation therapy), and confirmed with flowcytometry.
- •oRelapsed JMML:
- •After a documented CR or PR, this designation is defined as
- •- reappearance of organomegaly
- •- in combination with elevated WBC with peripheral blood monocytosis (greater than 1x109/l),
- •- and/or the reappearance of a cytogenetic or molecular lesion indicative of prior disease.
- •- In addition, clinical criteria may be used, which include objective parameters such as increase in spleen size of >50% from baseline, and/or the appearance of new skin lesions, and/or oxygen need,
- •- and/or blast crises/transformation to AML.
- •1 month to = 18 years old
- •Lansky play score = 60; or Karnofsky performance status = 60 (appendix 2)
- •Life expectancy ? 3 months
- •Normal renal function defined as less than or equal to NCI-CTCAE grade 1 (max 1.5 x ULN).
- •Normal liver function defined as less than or equal to NCI-CTCAE grade 1 (max 2.5 x ULN for transaminases and bilirubin)
- •No chemotherapy within 3 weeks of start of study medication. For 6-MP or low-dose cytarabine in JMML patients 1 week wash-out time is sufficient.
- •For JMML patients: saturation >92% without additional supply of oxygen
- •For JMML patients: peripheral blood monocyte count greater than 1.0x109/l
- •For relapsed patients following HSCT: recovery of all acute toxic effects of prior chemotherapy/stem-cell transplantation.
- •Able to comply with scheduled follow-up and with management of toxicity.
- •Reproductive Function
- •Female patients of childbearing potential must have a negative urine or serum pregnancy test confirmed prior to enrollment.
- •Female patients with infants must agree not to breastfeed their infants while on this study.
- •Male and female patients of child-bearing potential must agree to use an highly effective method of contraception approved by the investigator during the study and for 90 days after the last dose of azacitidine.
- •Highly effective methods of contraception include (but not exclusively) the following contraceptive methods:
- •combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation
- •progestogen-only hormonal contraception associated with inhibition of ovulation
- •intrauterine device (IUD)
- •intrauterine hormone-releasing system ( IUS)
- •sexual abstinence.
- •Written informed consent from patients or from parents or legal guardians for minor patients, according to local law and regulations.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 12
- •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
- •Other serious illnesses or medical conditions
- •Genetic abnormalities indicative of AML
- •JMML patients in whom a diagnosis of Noonan syndrome is suspected based on clinical history and/or presenting symptoms
- •Patients with secondary MDS with underlying bone-marrow failure syndromes or with familial MDS
- •Isolated extramedullary disease
- •Symptomatic CNS-involvement
- •Current uncontrolled infection
- •Cardiac toxicity (shortening fraction below 28%)
- •Concurrent treatment with any other anti-cancer therapy is not allowed
- •Pregnant or lactating patients
- •Patients who cannot be regularly followed up for psychological, social, familial or geographic reasons
- •Patient with expected non-compliance to toxicity management guidelines
- •Prior treatment with a demethylating agent
- •Allergy to azacitidine or mannitol.
研究者
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