Azacitidine in children with MDS or JMM

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 5

Inclusion Criteria

In this study 4 subgroups of patients are eligible, which will be enrolled in 4 different strata:

?stratum 1: newly diagnosed patients with advanced MDS (RAEB or RAEB-t) in a ?pre stem cell transplantation window?.
?stratum 2: relapsed patients with advanced MDS in a ?re-transplantation window?. At relapse azacitidine may also be continued when a 2nd transplant is not feasible, as long as the patient benefits from treatment.
?stratum 3: newly diagnosed patients with JMML in a ?pre-stem cell transplantation window?.
?stratum 4: relapsed patients with JMML in a ?re-transplantation window?. Azacitidine may also be continued when a 2nd transplant is not feasible and as long as the patient benefits from treatment.
?straum 5:newly diagnosed or relapsed patients with secondary advanced MDS, occurring after
chemotherapy, radiotherapy and or stem-cell transplantation, or secondary cases after
prior treatment for aplastic anemia. Note: secondary MDS cases after bone-marrow failures or familial cases are not eligible

General conditions:
?Advanced primary or secondary MDS or JMML confirmed by the diagnostic criteria as specified in the EWOG-MDS 2006 protocol (see appendix 1)
?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 > 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.
?For patients with childbearing potential, a negative test for pregnancy is to be considered before entry on study. If applicable, use of an effective contraceptive method.
?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: 65
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

Exclusion Criteria

Prior or current history:
?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 azicitidine or mannitol.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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