Ruxolitinib for Adult T-Cell Leukemia

Background: * The human T-cell leukemia virus 1 (HTLV-1) causes adult T-cell leukemia (ATL). Infection does not immediately cause ATL, but it can develop over time. ATL is a rare and aggressive type ...

Lead sponsor: National Cancer Institute (NCI)•1 U.S. locations•View source on ClinicalTrials.gov

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Sponsors

Lead Sponsor

National Cancer Institute (NCI)

Background: * Adult T-cell leukemia is a lymphoproliferative disorder characterized by the presence of cluster of differentiation 4 (CD4)/cluster of differentiation 25 (CD25) expressing T cells (interleukin-2 receptor (IL-2R) alpha expressing) in the peripheral blood, in lymphoid and other tissues. * In smoldering and chronic adult T-cell leukemia (ATL) the human T-cell leukemia virus 1 (HTLV-1) encoded protein, Tax constitutively activates interleukin-2 (IL-2), IL-9 and IL-15 autocrine/paracrine systems that in turn activate the Janus kinase (JAK)-1/3/signal transducer and activator of transcription 5 (STAT5) pathways. * Ruxolitinib a therapeutic agent inhibits cytokine mediated Janus kinase (JAK)-1/2 activation and ex vivo proliferation of malignant T cells from subjects with ATL. * Ruxolitinib is a potent orally bioavailable JAK1/2 inhibitor not licensed for the treatment of ATL. Primary Objective: Objective Initial Phase II: * To determine clinical or objective response rate for ruxolitinib given at 20 mg twice daily * Primary Objective Dose Escalation Phase I: To determine the maximum tolerated dose and clinical response rate for ruxolitinib given at doses of 30, 40 or 50 mg orally twice daily in subjects with smoldering, chronic and biologically indolent acute or lymphomatous subtype of ATL Eligibility * Subjects greater than or equal to 18 years old with pathologically confirmed adult T-cell leukemia: smoldering or chronic or previously treated lymphomatous or acute subtypes with clinically indolent behavior indicated by lack of significant symptoms and treatment free interval of greater than 6 months. * Subjects must have measurable or evaluable disease. Subjects with \> 10% of their PBMCs having the characteristic abnormal (i.e., CD3dim, CD4 plus CD25 plus expressing) fluorescence activated cell sorting (FACS) profile for circulating ATL cells will be considered to have measurable disease. * Subjects with symptomatic leukemic meningitis, bony or gastrointestinal (GI) tract involvement, serum calcium or Lactate dehydrogenase (LDH) \> 1.5 times the upper limit of normal will be excluded. However, subjects that have both ATL and another HTLV-1 associated disease such as tropical spastic paraparesis (human T-cell leukemia virus 1 (HTLV-1) Associated Myelopathy (HAM)/tropical spastic paraparesis (TSP)) will be included. * No prior treatment with another JAK inhibitor; subjects previously treated in this protocol at the lower dose are eligible to restart treatment at the higher dose levels. Design \- This is a pilot open-label, trial with off label-use of oral ruxolitinib that will treat 27 to 33 Subjects with smoldering or chronic or clinically indolent ATL. Groups of 3 to 6 newly enrolled or reenrolled Subjects will begin treatment at an elevated dose of 30 mg orally given twice daily. If this dose is tolerated without exceeding the criteria for dose limiting toxicity (DLT) during the first cycle of treatment, the tolerability of treatment at 40 mg and then 50 mg twice daily will be evaluated.

Data from ClinicalTrials.govLast updated on ClinicalTrials.gov: January 10, 2023Terms

Trial snapshot

StatusTERMINATED

PhasePHASE1/PHASE2

Enrollment19

Start dateOct 2012

Last updatedJan 10, 2023

Condition

T Cell Leukemia, Adult Leukemia, Adult T-Cell T Cell Leukemia, HTLV I Associated

Locations shown

National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: January 10, 2023Data synced to Clareo: May 3, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

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