NCT07061951

Testing the Effectiveness of the Anti-cancer Drug, Mirdametinib, in Treating Relapsed, Refractory Chronic Lymphocytic Leukemia

A Phase 2 Pilot Study of Mirdametinib in Relapsed Refractory Chronic Lymphocytic Leukemia

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: National Cancer Institute (NCI) · NIH
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This phase II trial tests the effect of mirdametinib in treating patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) that has come back after a period of improvement (relapsed) or that has not responded to previous treatment (refractory). Mirdametinib, a methyl ethyl ketone (MEK) inhibitor, works by blocking the action of an abnormal protein that signals cancer cells to multiply. This may help slow or stop the spread of cancer cells. Giving mirdametinib may be effective in treating patients with relapsed or refractory CLL or SLL.

Detailed description

PRIMARY OBJECTIVE: I. To evaluate the best objective response rate (ORR) with single agent mirdametinib. ORR will be composed of complete response (CR), CR with incomplete marrow recovery (CRi), and partial remission (PR) as defined by the 2018 Modified International Workshop on Chronic Lymphocytic Leukemia (iwCLL) National Cancer Institute-Working Group (NCI-WG) criteria, which includes PR with lymphocytosis (Hallek et al., 2018). SECONDARY OBJECTIVES: I. To determine the effect of mirdametinib on progression-free survival (PFS). II. To evaluate the duration of response (DOR) for mirdametinib in relapsed refractory chronic lymphocytic leukemia (R/R CLL). III. To determine the effect of mirdametinib on overall survival (OS). IV. To evaluate the safety and tolerability of mirdametinib, including the rates of high grade (3+) events, and rates of discontinuations and dose modifications. OUTLINE: Patients receive mirdametinib orally (PO) twice daily (BID) on days 1-28 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Additionally, patients undergo bone marrow biopsy, blood sample collection, echocardiography, and computed tomography (CT) throughout the study. After completion of study treatment, patients are followed up at 30 days then every 2 months until starting a new therapy followed by every 3 months for up to a total of 5 years.

Conditions

• Recurrent Chronic Lymphocytic Leukemia
• Recurrent Small Lymphocytic Lymphoma
• Refractory Chronic Lymphocytic Leukemia
• Refractory Small Lymphocytic Lymphoma

Interventions

Type	Name	Description
PROCEDURE	Biospecimen Collection	Undergo blood sample collection
PROCEDURE	Bone Marrow Biopsy	Undergo bone marrow biopsy
PROCEDURE	Computed Tomography	Undergo CT
PROCEDURE	Echocardiography Test	Undergo echocardiography
DRUG	Mirdametinib	Given PO

Timeline

• Start date: 2026-09-08
• Primary completion: 2030-02-01
• Completion: 2030-02-01
• First posted: 2025-07-14
• Last updated: 2026-04-13

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study