juvenile chronic myeloid leukemia myelodysplasia
Conditions
Interventions
Azacitidine 75 mg/m2, either IV or SC, will be administered QD on Days 1 to 7
of a 28-day
cycle for a minimum of 3 cycles and a maximum of 6 cycles, provided that the
subject does not
have disease p
azacitidine
hematopoietic stem cell transplantation
juvenile myelomonocytic leukemia
myelodysplastic syndrome
Sponsors
Celgene Corporation
Eligibility
Age
2 Years to 17 Years
Inclusion criteria
Inclusion criteria: MDS: 1. Patient has newly diagnosed advanced primary or secondary MDS with immature cells in blood or bone marrow or chromosomal abnormality linked to secondary MDS. Blood and bone marrow samples confirming diagnosis within 14 days prior to ICF as for the MDS. JMML: 1. Patient has newly diagnosed JMML, with samples from blood and bone marrow confirming diagnosis and specific genetic changes.;Both MDS and JMML: 2.Patient has a Lansky play score/ Karnofsky performance status at least equal to 60 3.Patient has a normal renal function and a normal liver function. 4.Subjects should be between 1 month to less than 18 years at time of signing ICF/ IAF
Exclusion criteria
Exclusion criteria: MDS exclusions: 1.Patient has an illness caused by 'germline genetic defects'. 2.Patient has inherited bone marrow failure syndrome. JMML exclusion: 1.Patient has germline genetic defects. Both: 1.Patient has organ dysfunction that will interfere with the administration of the therapy according to this protocol. 2.Hypersensitivity to azacitidine
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Response Rate at Cycle 3 Day 28 | — |
Secondary
| Measure | Time frame |
|---|---|
| - Cytogenetic Response for MDS subjects; Cytogenetic and Molecular Response for JMML subjects - Duration of response - Time to response - Time to progression - Leukemia free survival - Overall survival - Deoxyribonucleic acid methylation status in BM on Days 1 and 15 of Cycle 1, Day 28 of Cycle 3, pre-HSCT, and at the time of relapse/progression - Percentage of subjects undergoing HSCT - Time to first HSCT -Safety defined by frequency and severity of treatment emergent AEs - Pharmacokinetics | — |
Countries
The Netherlands
Outcome results
None listed