A Phase I/II study of Azacitidine (Vidaza®) in pediatric patients with relapsed high-grade pediatric MDS or JMML.

Completed

Conditions: - Pediatric myelodysplatsic syndrome (MDS)-Juvenile myelomonocytic leukemia (JMML)

Recruitment & Eligibility

Inclusion Criteria

1. Established diagnosis of relapsed MDS or JMML according to EWOG-criteria, after a prior stem cell transplantation;

2. 1 month to ≤ 18 years old;

Exclusion Criteria

1. Prior or current history:

o Other serious illnesses or medical conditions

Primary Outcome Measures

Name	Time	Method
The current study aims to establish the recommended dose, safety and preliminary efficacy of azacitidine administered IV in children with advanced newly diagnosed or relapsed/refractory MDS or JMML, in 4 different subgroups (strata) of patients. Recommended dose will be determined by:<br /><br>1. Dose-limiting toxicities;<br /><br>2. DLTs are AEs considered at least possibly drug-related and will be limited to the first course of azacitidine.

Secondary Outcome Measures

Name	Time	Method
1. To determine the safety and tolerability of azacitidine per stratum;<br /><br>2. To determine (preliminary) the hematological remission rate in these patients;<br /><br>3. To describe the durability of response and long-term follow-up, including that of patients undergoing stem-cell transplant after treatment with azacitidine;<br /><br>4. To determine the plasma pharmacokinetic parameters of azacitidine;<br /><br>5. To study the pharmacodynamic effects of azacitidine in pediatric MDS and JMML.<br><br /><br /><br>Efficacy will be determined by response definitions:<br /><br>1. For advanced MDS: Cheson et al, 2006;<br /><br>2. For JMML :Chan et al, 2009.