NL-OMON47695招募中2 期
A Phase I/II study of Azacitidine (Vidaza®) in pediatric patients with relapsed high-grade pediatric MDS or JMML - Azacitidine in pediatric MDS/JMM
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 3
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 17（—）
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:
- •o Relapsed 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.
- •o Relapsed 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
- •intrauterine device (IUD)
- •intrauterine hormone-releasing system ( IUS)
- •sexual abstinence.
- 另有 2 项未显示
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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