About this study
Juvenile myelomonocytic leukemia (JMML) is a rare blood cancer that mostly affects very young children. Right now, the main treatment is a stem cell (bone marrow) transplant. Even with this treatment, only about half of patients survive, and the disease can return. The transplant can also cause strong side effects. We want to find safer and better ways to treat JMML.
In the TRAZA study, health care providers use tests to see how aggressive the leukemia is. One thing they check is a methylation pattern, which shows how parts of DNA are turned on or off. Patients with only 1 gene change and a low methylation pattern usually have a milder form of the disease. Patients with more than 1 gene change, or a high methylation pattern, usually have a more aggressive form.
Based on this, each patient is placed into a risk group. A risk group shows how likely it is that the cancer will come back or get worse. Doctors use this information to choose the treatment plan that fits each patient’s needs.
This study tests 2 medicines together: trametinib and azacitidine. The treatment plan depends on the risk level of the disease:
- In the lower-risk group, patients get azacitidine for 5 days and trametinib every day for 28 days. This cycle may be repeated up to 12 times.
- In the higher-risk group, patients get trametinib and azacitidine, plus 2 chemotherapy medicines called fludarabine and cytarabine. That group can get up to 2 treatment cycles.
The study has several goals. We want to find out:
- If trametinib with azacitidine is safe for children with JMML
- If adding chemotherapy for higher-risk patients is safe and helpful
- How well the cancer responds to these treatments
We hope this new approach may work better than going directly to a stem cell transplant.
If the study shows that the medicines are safe and effective, it could give doctors new options for treating JMML. The study may also help us learn more about how JMML grows and how these medicines stop it.
Eligibility overview
- 1 month–21 years old
- Diagnosis of juvenile myelomonocytic leukemia