Phase 2
N=51
Ruxolitinib Phosphate in Treating Patients With Chronic Neutrophilic Leukemia or Atypical Chronic Myeloid Leukemia
Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative · Chronic Neutrophilic Leukemia
Bottom Line
View on ClinicalTrials.gov: NCT02092324 ↗Enrolled (actual)
51
Serious AEs
61.2%
Results posted
Nov 2020
Primary outcome: Primary: Percentage of First 25 Enrolled Patients With a Hematologic Response to Ruxolitinib (Complete Response (CR), Partial Response (PR)) — 32; 4 percentage of participants — p=0.002
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Laboratory Biomarker Analysis (Other); Quality-of-Life Assessment (Other); Questionnaire Administration (Other); Ruxolitinib Phosphate (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- OHSU Knight Cancer Institute
- Primary completion
- Nov 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of First 25 Enrolled Patients With a Hematologic Response to Ruxolitinib (Complete Response (CR), Partial Response (PR)) |
32; 4 | 0.002 sig |
| SECONDARY Percentage of Participant With Any Hematologic Grade III or IV Adverse Events. |
46.9 | — |
| SECONDARY Percentage of Participants With Any Non-hematologic Grade III or IV Adverse Events. |
69.4 | — |
| SECONDARY Percentage of Participants Who Achieved Clinical Response of Partial Response or Better |
33; 8 | <0.0001 sig |
| SECONDARY Median Time on Study (Months) for Early Drop Offs |
2.4 | — |
| SECONDARY Median Time on Study (Months) for All Enrolled Subjects |
8.5 | — |
| SECONDARY Percentage of Participants With Early Drop Off (Prior to Completion of Cycle 3) |
16 | — |
| SECONDARY Percentage of Participants Who Reach Cycle 7 |
65 | — |
| SECONDARY Percentage of Participants With Early Drop Off (After Completion of Cycle 3 and Prior to Completion of Cycle 6) |
18 | — |
| SECONDARY Maximum Clinical Responses |
8; 24; 29; 37 | — |
| SECONDARY Change in Spleen Size, Evaluated by Ultrasound |
-219.7 | — |
| SECONDARY Change in Symptom Score as Measured by a Modified Myeloproliferative Neoplasm Symptom Assessment Form [MPN-SAF] |
-3.0 | — |
| SECONDARY Overall Survival in All Enrolled Patients |
17.9 | — |
| SECONDARY Percentage of Patients Who Achieve Clinical Response of Partial Response or Better by CSF3R Mutation Status |
9; 54; 0; 15 | 0.003 sig |
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.
Eligibility Criteria
Inclusion Criteria
- Subjects must be newly diagnosed or previously diagnosed with CNL or aCML; all patients must have a bone marrow biopsy completed during the screening or baseline period if one has not been done within 90 days of day 1, cycle one
- Subjects must have platelet count greater than 25,000 per microliter at baseline and at the start of study (day 1, cycle 1) visit
- Subjects must be able to discontinue any drug treatment aimed at lowering disease burden in CNL or aCML; subjects should discontinue hydroxyurea to treat underlying CNL or aCML disease no later than day -7 (one week before starting ruxolitinib); for drugs that have more long-lasting effects on the marrow, such as thalidomide and its analogs, and interferon, subjects should discontinue these no later than day -28
- Subjects must be willing to accept/continue transfusions to treat low hemoglobin levels
- Subjects must have a life expectancy of > 6 months
Exclusion Criteria
- Subjects unable to review and sign informed consent form
- Females who are pregnant or breastfeeding, and males and females who cannot comply with requirements to avoid fathering a child or becoming pregnant
- Subjects with known diagnosis of human immunodeficiency virus (HIV) or chronic active Hepatitis B or C; viral testing is not required; subjects with a history of Hepatitis B and/or C are allowed on trial if the virus is undetected at the time of enrollment
- Subjects with inadequate liver (alanine aminotransferase [ALT]/serum glutamate pyruvate transaminase [SGPT] above 4 X upper limit of normal [ULN] or direct bilirubin 4 X ULN AND the lab abnormalities are felt to be due to underlying liver dysfunction)
- Subjects with end stage renal function (creatinine clearance [CrCl] < 15 mL/min or glomerular filtration rate [GFR] <15 mL/min) regardless of whether hemodialysis is required
- Subjects with clinically serious infections requiring ongoing antibiotic therapy
- Subjects with severe (immediately life threatening) and recent (occurring within the last 3 months) cardiac dysfunction, pulmonary dysfunction, esophageal variceal bleeding, hemorrhagic strokes, or intracranial hemorrhage are not eligible for study participation
- Subjects requiring therapeutic doses of anticoagulation or anti-platelet therapies (aspirin above 81 mg daily, Plavix or similar agents) AND platelet counts are below 50,000 on two different laboratory evaluations, separated by minimum of two weeks
- Taking investigational or commercial agents or therapies with the intent to treat the subject's malignancy other than those therapies permitted
- Subjects with invasive malignancy over the previous 2 years except treated early stage carcinomas of the skin, completely resected intraepithelial carcinoma of the cervix, and completely resected papillary thyroid and follicular thyroid cancers
- Previous allergic reactions to janus kinase (JAK) inhibitors or excipients
- Prior therapy with ruxolitinib or other JAK inhibitors
- Subjects who have had major surgery within 4 weeks prior to entering the study
- Subjects who are anticipated to receive a transplant within the first 6 months of treatment on trial
Data sourced from ClinicalTrials.gov (NCT02092324). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.