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Ruxolitinib in Thrombocythemia and Polycythemia Vera

ConditionsEssential ThrombocythemiaPolycythemia Vera
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorMassachusetts General Hospital

About this trial

This research is being done to see if the drug ruxolitinib is effective in reducing the symptoms caused by low-risk essential thrombocythemia (ET) and polycythemia vera (PV).

\- This research study involves the study drug Ruxolitinib.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Patients who have been diagnosed with essential thrombocythemia or polycythemia vera by World Health Organization 2016 diagnostic criteria.

Patients with essential thrombocythemia must be very low (no history of thrombosis, age <60, and no JAK2 mutation), low (no history of thrombosis, age <60, presence of JAK2 mutation), or intermediate risk (no history of thrombosis, age >60, no JAK2 mutation) by IPSET criteria. Patients with polycythemia vera must be low risk (no history of thrombosis and age <60) by NCCN guidelines.

Patients with an MPN-SAF TSS (MPN-10) score >10 AND at least one individual feature >5 documented on a separate visit within 3 months prior to study registration, as documented in the clinical record or obtained by clinician. If not previously documented in the electronic medical record, participants must be blinded to purpose of MPN SAF TSS scoring for eligibility determination. Average daily MPN-SAF TSS (MPN-10) score must remain >10 with any individual feature >5 for the week-long baseline assessment prior to ruxolitinib initiation.

Patients who have previously received or are receiving cytoreductive therapy (i.e. hydroxyurea, anagrelide, interferon) are eligible for the study if therapy was used for the indication of symptom control, or if therapy was used for pre-operative control of blood counts. If a subject is still receiving cytoreductive therapy at the time of screening and enrollment, there will be a wash-out period from prior cytoreductive therapy at least 7 days prior to ruxolitinib initiation.

Disqualifiers

Essential thrombocythemia patients who are high risk by IPSET-R criteria (age > 60 with JAK2 V617F mutation and/or history of thrombosis).1 Polycythemia vera patients who are high risk by NCCN guidelines (age > 60 and/or history of thrombosis).

Patients with >5% blasts on baseline marrow exam or at any other time in peripheral blood

Participants who are receiving any other investigational agents.

Participants with a history of splenectomy. Participants may still be eligible after discussion with and approval by the Overall PI, however.

Trial design

Design model

Sequential

Treatments tested in this trial

  • Ruxolitinib

    Drug

    Pill taken by mouth.

Treatment groups

60 Participants
are divided into 2 treatment groups
Group A: Ruxolitinib Stage 1Experimental treatment 1 intervention
Group B: Ruxolitinib Stage 2Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Percentage of patients who achieve >50% reduction from baseline to Myeloproliferative Neoplasm Symptom Assessment Total Symptom Score

Percentage of patients with \>50% change in Myeloproliferative Neoplasm Symptom Assessment Total Symptom Score (MPN-SAF TSS), which has use in major clinical trials and as symptom assessment in all patients in clinical practice. Modeling has established baseline MPN-SAF TSS cut-off scores (total MPN-SAF TSS \>20 or an individual component score \>5) at which symptomatic treatment would be significantly beneficial

Time frame
baseline to 12 weeks

Secondary outcomes

1

Proportion of patients achieving complete hematologic rate at week 12

Evaluate the efficacy of ruxolitinib in improving hematologic remission rates, as measured by International Working Group (IWG) working criteria ET: Platelet count ≤ 400 x 109/L, WBC count \<10 x 109/L, and absence of leukoerythroblastosis PV: hematocrit \<45% and WBC count \<10 x 109/L

Time frame
Week 12
2

Proportion of patients achieving complete hematologic rate at 24 Weeks

Evaluate the efficacy of ruxolitinib in improving hematologic remission rates, as measured by International Working Group (IWG) working criteria ET: Platelet count ≤ 400 x 109/L, WBC count \<10 x 109/L, and absence of leukoerythroblastosis PV: hematocrit \<45% and WBC count \<10 x 109/L

Time frame
Week 24
3

Best MPN-SAF TSS score

Myeloproliferative Neoplasm Symptom Assessment Total Symptom Score (MPN-SAF TSS) for use in major clinical trials and as symptom assessment in all patients in clinical practice. Modeling has established baseline MPN-SAF TSS cut-off scores (total MPN-SAF TSS \>20 or an individual component score \>5) at which symptomatic treatment would be significantly beneficial. Scale title includes absence of symptom (0) and worst imaginable symptoms (10) per question, with minimum score 0 and maximum score 100. Higher scores indicate worse outcomes.

Time frame
12 Weeks
4

Best MPN-SAF TSS score

Myeloproliferative Neoplasm Symptom Assessment Total Symptom Score (MPN-SAF TSS) for use in major clinical trials and as symptom assessment in all patients in clinical practice. Modeling has established baseline MPN-SAF TSS cut-off scores (total MPN-SAF TSS \>20 or an individual component score \>5) at which symptomatic treatment would be significantly beneficial. . Scale title includes absence of symptom (0) and worst imaginable symptoms (10) per question, with minimum score 0 and maximum score 100. Higher scores indicate worse outcomes.

Time frame
24 Weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Massachusetts General Hospital

Lead sponsor

Incyte Corporation

Collaborator

Source ClinicalTrials.gov
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