NCT02092324

Ruxolitinib Phosphate in Treating Patients With Chronic Neutrophilic Leukemia or Atypical Chronic Myeloid Leukemia

Prospective Evaluation of Ruxolitinib Efficacy for CNL/aCML Patients With Mutation of CSF3R

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 51 (actual)
• Sponsor: OHSU Knight Cancer Institute · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This phase II trial studies how well ruxolitinib phosphate works in treating patients with chronic neutrophilic leukemia (CNL) or atypical chronic myeloid leukemia (aCML). Ruxolitinib phosphate may stop the growth of cancer cells by blocking some of the enzymes needed for cells to reproduce. This trial also studies the genetic makeup of patients. Certain genes in cancer cells may determine how the cancer grows or spreads and how it may respond to different drugs. Studying how the genes associated with CNL and aCML respond to the study drug may help doctors learn more about CNL and aCML and improve the treatment for these diseases.

Detailed description

PRIMARY OBJECTIVES: I. To determine the proportion of patients with chronic neutrophilic leukemia (CNL) and atypical chronic myeloid leukemia (aCML) who have a hematologic response to ruxolitinib (ruxolitinib phosphate) (partial response \[PR\], complete response \[CR\], complete response, partial \[CRp\]). SECONDARY OBJECTIVES: I. To determine the frequency of grade 3 or 4 hematologic and non-hematologic adverse events experienced by subjects during therapy with ruxolitinib. II. To determine whether hematologic responses correlate with certain types of mutations in colony stimulating factor 3 receptor (CSF3R) and reduction in mutant CSF3R allele burden in the peripheral blood. III. To determine the maximum clinical responses for each subject and the median duration of maximum clinical responses. IV. To determine the mean % reduction of spleen size, estimated by volume using the conventional prolate ellipsoid method as measured by ultrasound compare to baseline. V. To determine the mean % reduction of total symptom score as measured by a modified Myeloproliferative Neoplasm Symptom Assessment Form version 2.0 (MPN-SAF) compared to start of study (day 1, cycle 1). VI. To determine overall survival in subjects who complete a minimum of 1 dose of study drug. VII. To determine the proportion of subjects who discontinue after completion of \> 3 cycles but \< 6 cycles. VIII. To determine the proportion of subjects who discontinue prior to completion of cycle 3. OUTLINE: Patients receive ruxolitinib phosphate orally (PO) every other day, once daily (QD), or twice daily (BID) on days 1-28. Each patient will be followed for a maximum of 96 weeks (24 cycles, 1 cycle is 4 weeks long). If the study drug continues to be effective, the patient may be eligible to continue on study drug past 24 cycles. After completion of study treatment, patients are followed up within 2 weeks and at 4-6 weeks.

Conditions

• Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative
• Chronic Neutrophilic Leukemia

Interventions

Type	Name	Description
OTHER	Laboratory Biomarker Analysis	Correlative studies
OTHER	Quality-of-Life Assessment	Ancillary studies
OTHER	Questionnaire Administration	Ancillary studies
DRUG	Ruxolitinib Phosphate	Given PO

Timeline

• Start date: 2014-07-08
• Primary completion: 2019-11-22
• Completion: 2020-01-24
• First posted: 2014-03-20
• Last updated: 2020-11-16
• Results posted: 2020-11-16

Locations

7 sites across 1 country: United States