INTERNATIONAL MULTICENTER, OPEN-LABEL, PHASE 2 STUDY TO TREAT MOLECULAR RELAPSE OF PEDIATRIC ACUTE MYELOID LEUKEMIA WITH AZACITIDINE

Recruitment & Eligibility

Status: Complete

Sex: All

Target Recruitment: 20

Inclusion Criteria

1.Aged 3 months to <21 years with documented diagnosis of AML according to WHO classification with at least one quantitative genetic maker, e.g. one of the following aberrations:
•t(8;21); RUNX1-RUNX1T1
•inv(16); CBFb-MYH11
•t(9;11); MLL-AF9
•t(10;11); MLL-AF10
•NPM1
•FLT3-ITD
•WT1; etc.
2.First complete remission (MRD in PB less than 5 x 10-4) confirmed at the start of last consolidation course or within 1 month after completion of consolidation treatment
3.Detection of a confirmed molecular relapse of an AML
4.Understand and voluntarily provide permission (subjects and when applicable, parental/legal representative(s)) to the ICF prior to conducting any study related assessments/procedures
5.Able to adhere to the study visit schedule and other protocol requirements
6.Lansky performance score at least equal to 50; or Karnofsky performance status at least equal to 50, whichever is applicable
7.Negative serum pregnancy tests for females of child bearing potential within 10 days prior to treatment

Exclusion Criteria

1.Concomitant treatment with any other anticancer therapy except those specified in protocol
2.HSCT within previous 3 months
3.Treated by any investigational agent in a clinical study within previous 4 weeks
4.Pregnancy or lactating
5.FAB type M3 leukemia (acute promyelocytic leukemia)
6.Therapy-related AML
7.AML of Down syndrome or other congenital syndromes giving rise to leukemia or treatment complications
8.Symptomatic cardiac disorders (CTCAE 4.0 Grade 3 or 4)
9.Evidence of invasive fungal infection or other severe systemic infection requiring treatment doses of systemic/parenteral therapy including known active viral infection with human immunodeficiency virus (HIV) or Hepatitis Type B and C
10.Any other organ dysfunction (CTCAE 4.0 Grade 3 or 4) that will interfere with the administration of the therapy according to this protocol
11.Ongoing severe toxicities (CTCAE 4.0 Grade 3 or 4) of prior chemotherapy/stem cell transplantation
12.Hypersensitivity to the active substance or other excipients contained in the investigational medical product listed in the summary of product characteristics (SmPC) or Investigators Brochure (IB).
13.Abnormal liver function
14. Symptomatic CNS-involvement or isolated extramedullary disease at initial diagnosis
15. Female and male subjects with child bearing potential who avoid using highly effective anticonceptive measure(ment)s

Study & Design

Study Type: interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method