A Phase 2, multicenter, open-label study to evaluate the pharmacokinetics, pharmacodynamics,<br>safety and activity of azacitidine and to compare azacitidine to historical controls in pediatric<br>subjects with newly diagnosed advanced myelodysplastic syndrome or juvenile myelomonocytic<br>leukemia before hematopoietic stem cell transplantatio
- Conditions
- juvenile chronic myeloid leukemiamyelodysplasia10024324
- Registration Number
- NL-OMON41785
- Lead Sponsor
- Celgene Corporation
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 2
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
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
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Response Rate at Cycle 3 Day 28</p><br>
- Secondary Outcome Measures
Name Time Method <p>- Cytogenetic Response for MDS subjects; Cytogenetic and Molecular Response for<br /><br>JMML subjects<br /><br>- Duration of response<br /><br>- Time to response<br /><br>- Time to progression<br /><br>- Leukemia free survival<br /><br>- Overall survival<br /><br>- Deoxyribonucleic acid methylation status in BM on Days 1 and 15 of Cycle 1,<br /><br>Day 28 of Cycle 3, pre-HSCT, and at the time of relapse/progression<br /><br>- Percentage of subjects undergoing HSCT<br /><br>- Time to first HSCT<br /><br>-Safety defined by frequency and severity of treatment emergent AEs<br /><br>- Pharmacokinetics </p><br>