Risk stratified treatment for patients with newly diagnosed juvenile myelomonocytic leukemia: A Phase I/II non-randomized study of trametinib and azacitidine with or without chemotherapy

Study on Treatment for JMML

S
Sandeep Batra, MD

Primary Investigator

Enrolling By Invitation
1 month - 21 years
All
Phase 1/2
1 participants needed
2 Locations

Brief description of study

This clinical trial will test the safety and efficacy of combining trametinib and azacitidine in patients with juvenile myelomonocytic leukemia (JMML). Newly diagnosed lower-risk JMML patients will receive trametinib and azacitidine. High-risk JMML patients will receive trametinib, azacitidine, fludarabine, and cytarabine.
 
THIS STUDY IS ENROLLING BY INVITATION ONLY - Consistent with most oncology trials, patients are not actively “recruited,” but are screened by their physician for appropriate clinical trial(s) at the time of their routine clinic visit. Occasionally, a patient may be a self-referral or physician referral, but are still screened for appropriate clinical trials at the time of their routine clinic visit. PI and staff may send copies of relevant consent forms to these patients to look over prior to actually consenting or enrolling them. This may take place at the patient's visit at which the consent is presented or the patient's next visit to the outpatient hematology/oncology clinic. 
 
Interested in participating? For more information about this research study or other cancer-related clinical trials at IU Simon Comprehensive Cancer Center, please contact:
IU Clinical Trials Office 
Phone: (317) 278-5632

Detailed description of study

 

  • Patients with newly diagnosed lower-risk JMML will be treated with daily azacitidine for 5 days in combination with daily trametinib for 28 days per course for up to 12 courses.
  • Patients with newly diagnosed high-risk JMML will be treated with daily azacitidine, fludarabine, and cytarabine for 5 days in combination with daily trametinib for 28 days per course for up to 2 courses.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Juvenile Myelomonocytic Leukemia, Cancer, Riley
  • Age: 1 month - 21 years
  • Gender: All

Inclusion Criteria:

Age - Patients must be ≥ 1 month and ≤21 years of age at enrollment.

Diagnosis

- Patients must meet the World Health Organization criteria for JMML. The diagnosis is made based on the following criteria.

1 Month to 21 Years (Child,  Adult )

Updated on 10 Jul 2025. Study ID: PHO-TACL-T2020-004, 21367

This study investigates treatment options for juvenile myelomonocytic leukemia (JMML), a rare type of blood cancer that affects young children. JMML is a condition where the bone marrow makes too many white blood cells, which can lead to various health problems. The purpose of this study is to test the safety and effectiveness of combining two investigational medications, trametinib and azacitidine, with or without additional chemotherapy, for patients who have been newly diagnosed with JMML.

Participants in this study will be divided into two study arms based on their risk level. Those with lower-risk JMML will receive trametinib and azacitidine. Those with higher-risk JMML will also receive additional chemotherapy drugs, fludarabine and cytarabine. Lower-risk patients will take azacitidine for 5 days and trametinib for 28 days in each course, up to 12 courses. Higher-risk patients will take azacitidine, fludarabine, and cytarabine for 5 days, along with trametinib for 28 days, up to 2 courses.

  • Who can participate: Participants must be between 1 month and 21 years old and meet the criteria for JMML as defined by the World Health Organization.
  • Study details: Participants will receive specific treatments based on their risk level. Lower-risk patients will take trametinib and azacitidine, while higher-risk patients will also receive fludarabine and cytarabine.
Please visit our main page to search for other studies you may be interested in. If you need help finding a study or have any questions, please contact us at inhealth@iu.edu

Interested in the study?

This study is accepting only persons who receive care at a certain clinic or doctor or who are part of an invited group. Questions about this study can be directed to the study team listed in the description or contact your doctor to see if you are eligible.

Accepting Referrals by Invitation Only