To determine the safety, clinical efficacy, and effect of azacitidine on the body,within the body and interactions within the body in children and young adults with newly diagnosed advanced Myelodysplastic Syndromes or Juvenile myelomonocyctic leukemia before transplantation of the bone marrow and blood. At the end of the study, the data will be compared to data from an older study of patients with the same diseases.

Phase 1

• Conditions: newly diagnosed advanced myelodysplastic syndrome (MDS) or juvenile myelomonocytic leukemia (JMML) prior to hematopoietic stem cell transplantation (HSCT); MedDRA version: 18.0Level: PTClassification code 10023249Term: Juvenile chronic myelomonocytic leukaemiaSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps); MedDRA version: 18.0Level: LLTClassification code 10054439Term: Juvenile chronic myelomonocytic leukemiaSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps); MedDRA version: 18.0Level: HLTClassification code 10028536Term: Myelodysplastic syndromesSystem Organ Class: 100000004851; Therapeutic area: Diseases [C] - Cancer [C04]

• Registration Number: EUCTR2014-002388-13-AT
• Lead Sponsor: Celgene Corporation

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2015-02-12
• Last Update Posted: 25 November 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 55

Inclusion Criteria

MDS:

1. 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:

1. 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

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
Are the trial subjects under 18? yes
Number of subjects for this age range: 55
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

MDS exclusions:

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

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

Both:
1.Patient has any other organ dysfunction that will interfere with the administration of the therapy according to this protocol.
2.Hypersensitivity to azacitidine

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified