AZA-JMML-001

Author: Julia Dobke, erstellt am: 2016/11/21, Last modification: 2017/01/18

Title Study With Azacitidine in Pediatric Subjects With Newly Diagnosed Advanced Myelodysplastic Syndrome (MDS) and Juvenile Myelomonocytic Leukemia (JMML)
Disease Pediatric subjects with newly diagnosed advanced myelodysplastic syndrome or juvenile myelomonocytic leukemia before hematopoietic stem cell transplantation.
Type A Phase 2, international, multicenter, open-label study
Problem / Objectives

Primary objective:
  is to assess the treatment effect on response rate (MDS: CR, PR, or marrow CR; JMML: either cCR or cPR) at Cycle 3 Day 28 and to compare against standard therapy using a matched-pairs analysis with historical data.

Secondary objective
The secondary objectives are to further evaluate safety, efficacy, pharmacodynamics (PD), and pharmacokinetics (PK) of azacitidine in this subject population.

Therapy / Study arms

Myelodysplastic Syndrome (MDS):
Azacitidin 75 mg/m2 intravenous (i.v.) or subcutaneous (s.c.) daily on day 1-7 of a 28-day cyclus for at least 3 courses and max. 6 courses.

Juvenile myelozytäre Leukämie (JMML):
Azacitidin 75 mg/m2 intravenous (i.v.) or subcutaneous (s.c.) daily on day 1-7 of a 28-days cyclus for at least 3 courses and max. 6 courses..

Inclusion Criteria
  • MDS: Patient has newly diagnosed advanced primary or secondary MDS with an amount of immature cells in blood or bone marrow or chromosomal abnormality linked to secondary MDS. Blood or bone marrow samples confirming diagnosis within 14 days prior to ICF as for the MDS.
  • JMML: Patient has newly diagnosed JMML, with samples from blood and bone marrow confirming diagnosis within the 14 days prior to informed consent/informed assent signature, with specific alteration in genes (which carry the information that determines a person characteristics) in the body
  • For both MDS and JMML:
  • Patient has a Lansky play score/ Karnofsky performance status at least equal to 60
  • Patient has a normal renal function and a normal liver function.
  • Subjects should be between 1 month to less than 18 years at time of signing ICF/ IAF
Exclusion Criteria

MDS:
Patient has an illness caused by 'genetic defects' (which cause abnormalities in the information that determine a person's characteristics).
Patient has inherited disease that cause bone marrow (the soft tissue inside of the bone) failures.

JMML:
Patient has a specific deviation in so called Germline.

Both:
Every kind of CNS-disease
Isolated extramedullary disease
Actual not controlled infection
Cardio toxicity( Shortening Fraction less than 28%).
Patient has any other organ dysfunction that will interfere with the administration of the therapy according to this protocol.
Hypersensitivity to azacitidine

Recruitment 55
Status 01.03.2014 - Oktober 2020
EudraCT
Entry Study Register ClinicalTrials.gov: NCTNCT02447666
Principal Investigator Celgene Corporation
E-Mail mailto:clinicaltrialdisclosure@celgene.com
Contact
Participants Düsseldorf, München, Hamburg, Kiel, Regensburg, Berlin, Augsburg, Jena, Tübingen, Münster, Frankfurt, Hannover, Freiburg, Essen, Dresden