NCT05592015

Ruxolitinib for the Treatment of T-Cell Large Granular Lymphocytic Leukemia

A Phase II Study Evaluating the Efficacy of Ruxolitinib in Patients With T-Cell Large Granular Lymphocytic Leukemia (T-LGLL)

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: Jonathan Brammer · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This phase II trial tests whether ruxolitinib works to shrink tumors in patients with T-cell large granular lymphocyte leukemia. Ruxolitinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

Detailed description

PRIMARY OBJECTIVE: I. Determine the overall response rate (ORR) of ruxolitinib in patients with T-cell large granular lymphocytic leukemia (T-LGLL) as compared to historical controls. SECONDARY OBJECTIVES: I. Rate of conversion from PR at 4 months to CR at 8 and 12 months (at full ruxolitinib dosage). II. Rate of molecular remission (T-cell receptor \[TCR\] clearance, STAT3 mutation clearance) at 4, 8, 12 months. III. Incidence of grade III/IV toxicities (at full ruxolitinib dosage). IV. Quality of life using the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30), Health Assessment Questionnaire Disability Index (HAQDi), and Short Form (SF)-36 questionnaire at baseline, 5 months, and every 6 months during response follow up for up to 12 months. EXPLORATORY OBJECTIVE: I. Objective benefit (OB) rate at 4 months defined as a patient that had improvement in their cytopenias, transfusion dependence but not attaining a partial response (PR). II. Leukemia-free survival III. Progression-free survival OUTLINE: Patients receive ruxolitinib orally (PO) twice daily (BID) on days 1-28. Cycles repeat every 28 days for 12 months in the absence of disease progression or unacceptable toxicity. Patients who achieve a response (CR or PR) may receive an additional 12 months of ruxolitinib, for a maximum of 24 months. Additionally, patients undergo blood sample collection throughout study.

Conditions

• T-Cell Large Granular Lymphocyte Leukemia

Interventions

Type	Name	Description
DRUG	Ruxolitinib	Given PO

Timeline

• Start date: 2023-05-03
• Primary completion: 2026-07-31
• Completion: 2026-07-31
• First posted: 2022-10-24
• Last updated: 2026-02-10

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study