Phase I/II Study of Trametinib and Azacitidine for Children and Young Adults with Newly Diagnosed Juvenile Myelomonocytic Leukemia

Sponsor: Therapeutic Advances in Childhood Leukemia Consortium

Sponsor score: 0
Inactive

ClinicalTrials ID:NCT05849662

Provider View

This clinical trial is testing the safety and effectiveness of combining two drugs, trametinib and azacitidine, for treating children and young adults with a type of blood cancer called juvenile myelomonocytic leukemia (JMML). The study involves different treatments based on the risk level of the disease.

Patient Parameters

Program Overview

The study aims to find out if using trametinib and azacitidine together is safe and effective for treating newly diagnosed juvenile myelomonocytic leukemia (JMML). Patients with lower-risk JMML will receive trametinib and azacitidine, while those with higher-risk JMML will also receive additional chemotherapy drugs, fludarabine and cytarabine.

Description

The main goals of this study are to determine if combining trametinib with azacitidine is safe for patients with newly diagnosed lower-risk JMML, and to see if adding fludarabine and cytarabine is safe for those with high-risk JMML.

For lower-risk JMML patients, the treatment involves taking azacitidine for 5 days and trametinib for 28 days in each treatment cycle, for up to 12 cycles. For high-risk JMML patients, the treatment includes azacitidine, fludarabine, and cytarabine for 5 days, along with trametinib for 28 days, for up to 2 cycles.

Eligibility Criteria

Inclusion Criteria

  • Age between 1 month and 21 years.
  • Diagnosed with juvenile myelomonocytic leukemia (JMML) based on specific medical criteria.
  • Must have certain blood and genetic markers related to JMML.
  • Must be in good general health, with a performance status score above 50%.
  • No previous leukemia treatment, except for specific medications allowed shortly before starting the study.
  • Must have adequate kidney, liver, and heart function based on medical tests.
  • Female patients of childbearing age must have a negative pregnancy test and agree not to breastfeed during the study.
  • All patients of childbearing potential must agree to use effective birth control during and for 6 months after the study.

Exclusion Criteria

  • Cannot have allergies to any study drugs.
  • Cannot have active infections that are not improving with treatment.
  • Cannot plan to receive other cancer treatments during the study.
  • Cannot have other serious health conditions that could affect participation.
  • Cannot have Noonan syndrome, but can have neurofibromatosis type 1 or CBL syndrome.
  • Cannot have used certain cancer treatments or medications affecting heart function.
  • Cannot have eye conditions like retinal vein occlusion or central serous retinopathy.
  • Cannot have used MEK inhibitors before.

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