A Phase 2 Study of Azacitidine in Children with MDS or JMML
Research type
Research Study
Full title
A phase 2, multicenter, open-label study to evaluate the pharmacokinetics, pharmacodynamics, safety and activity of Azacitidine and to compare Azacitidine to historical controls in pediatric subjects with newly diagnosed advanced Myelodysplastic Syndrome or Juvenile myelomonocytic leukemia before hematopoietic stem cell transplantation
IRAS ID
138128
Contact name
John Grainger
Contact email
Sponsor organisation
Celgene Corporation
Eudract number
2014-002388-13
Duration of Study in the UK
4 years, 0 months, 30 days
Research summary
This study is looking at the safety and effectiveness of Azicitidine in children from 1 month to less than 18 years old with newly diagnosed Myelodysplastic Syndrome (MDS) or Juvenile myelomonocytic leukemia (JMML) prior to stem cell transplant. These are both rare childhood cancers which evolve into aggressive leukemias refractory to standard chemotherapy. The only curative treatment is a stem cell transplant. Without a sibling donor the wait for a transplant is about 3 months during which time treatment is needed. Current treatments often do not control the disease well enough during this time and in 50% disease returns post transplant. Azicitidine is an anticancer drug that belongs to a group of drugs known as demethylating drugs, which change how diseased cells in the blood and bone marrow grow. Azicitidine is already licensed in EU and US for treatment of MDS in adults. The safety profile in children is similar to that in adults and on the available adult and paediatric data, azacitidine seems to be a promising treatment for paediatric MDS and JMML
20 MDS patients and 35 JMML will be recruited from 45 sites including 2 sites in the UK recruiting 1 patient each. Eligible patients will receive up to 6 cycles (28 days)of treatment on days 1 -7 of each cycle. Study treatment will be stopped if the patient is not responding, if it is making the patient ill or if the patient is ready for transplant. All patients will be followed for 2 years after the end of treatment and assessed every 3 months.
Patients will have tests at every hospital visit including blood and bone marrow samples, ultrasound and ECG. Blood samples for drug level testing will be collected during Cycle 1.
The sponsor is Celgene Corporation.REC name
North West - Haydock Research Ethics Committee
REC reference
15/NW/0098
Date of REC Opinion
9 Mar 2015
REC opinion
Further Information Favourable Opinion