A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia

Phase 1/2 Recruiting 58 enrolled Therapeutic Advances in Childhood Leukemia Consortium

Non-randomizedParallel-groupOpen-labelTreatment

Summary

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.

Timeline

Start: 2024-10-11
Primary completion: 2028-12
Completion: 2029-12

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Azacitidine	Other / unclassified	2.5 mg/kg	Intravenous
Subject	Azacitidine	Other / unclassified	75 mg/m2	Intravenous
Subject	Cytarabine	Small molecule	67 mg/kg	Intravenous
Subject	Cytarabine	Small molecule	2000 mg/m2	Intravenous
Subject	Fludarabine	Small molecule	1 mg/kg	Intravenous
Subject	Fludarabine	Small molecule	30 mg/m2	Intravenous
Subject	Trametinib	Small molecule	0.025 mg/kg	Oral
Subject	Trametinib	Small molecule	0.032 mg/kg	Oral

A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia

Summary

Timeline

Drugs

Indications