NCT03190915

Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia

A Phase 2 Study of the MEK Inhibitor Trametinib (NSC# 763093) in Children With Relapsed or Refractory Juvenile Myelomonocytic Leukemia

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: National Cancer Institute (NCI) · NIH
• Sex: All
• Age: 1 Month – 21 Years
• Healthy volunteers: Not accepted

Summary

This phase II trial studies how well trametinib works in treating patients with juvenile myelomonocytic leukemia that has come back (relapsed) or does not respond to treatment (refractory). Trametinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Detailed description

PRIMARY OBJECTIVE: I. To determine the objective response rate to trametinib in children with recurrent or refractory juvenile myelomonocytic leukemia (JMML). SECONDARY OBJECTIVES: I. To further define and describe the toxicities of single agent trametinib in children with recurrent or refractory JMML. II. To further characterize the pharmacokinetics of trametinib in children with recurrent or refractory JMML. III. To prospectively evaluate mutant allele burden as a marker of disease activity in JMML. IV. To measure the rate of complete responses in children with recurrent or refractory JMML. V. To measure the duration of response among responders. EXPLORATORY OBJECTIVE: I. To describe the distribution of JMML diagnostic criteria in children with recurrent or refractory JMML. OUTLINE: Patients receive trametinib orally (PO) once daily (QD) on days 1-28 of each cycle. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Patients undergo a bone marrow aspiration or biopsy at baseline, on day 28 of cycles 1 and 2, at all subsequent odd numbered cycles, and at end of treatment. After completion of study treatment, patients are followed up annually for up to 5 years.

Conditions

• Juvenile Myelomonocytic Leukemia
• Neurofibromatosis Type 1

Interventions

Type	Name	Description
PROCEDURE	Bone Marrow Aspiration and Biopsy	Undergo bone marrow aspiration or biopsy
DRUG	Trametinib	Given PO

Timeline

• Start date: 2018-09-09
• Primary completion: 2023-03-31
• Completion: 2026-10-03
• First posted: 2017-06-19
• Last updated: 2026-04-13
• Results posted: 2024-07-16

Locations

57 sites across 1 country: United States

Regulatory

• FDA-regulated drug study