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

NCT05849662 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 58

Last updated 2026-04-15

No results posted yet for this study

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.

Conditions

  • Leukemia, Juvenile Myelomonocytic
  • JMML
  • JCML
  • Neurofibromatosis 1
  • CBL Syndrome

Interventions

DRUG

Trametinib

PO or NG QD Days 1-28 For patients age \< 6 years: 0.032 mg/kg/day at max dose = 2mg/day For patients age ≥ 6 years: 0.025 mg/kg/day at max dose = 2 mg/day

DRUG

Azacitidine

IV over 30 minutes Days 1-5 Age \< 1 year or weight \<10kg: 2.5 mg/kg/day Age ≥ 1 year and weight ≥ 10kg: 75 mg/m2/day

DRUG

Fludarabine

IV over 30 minutes Days 6-10 30 mg/m2/day (1mg/kg if \<12 kg)

DRUG

Cytarabine

IV over 3 hours Days 6-10 2000 mg/m2/day (67mg/kg if \<12 kg)

Sponsors & Collaborators

  • Therapeutic Advances in Childhood Leukemia Consortium

    lead OTHER

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
1 Month
Max Age
21 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-10-11
Primary Completion
2028-12-31
Completion
2029-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05849662 on ClinicalTrials.gov