Oral Pacritinib Versus Best Available Therapy to Treat Myelofibrosis With Thrombocytopenia

• Status: Terminated
• Trial end date: 2016-11-01
• Target enrollment: 0
• Participant gender: All

Summary

The primary hypothesis of the study is that treatment with either once-daily or twice-daily pacritinib results in a greater proportion of patients with thrombocytopenia and myelofibrosis achieving ≥ 35% reduction in spleen volume from baseline to Week 24 than treatment with Best Available Therapy, and a greater proportion of patients achieving a ≥ 50% reduction in total symptom score from baseline to Week 24 as measured by the Myeloproliferative Neoplasm Symptom Assessment Form 2.0.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: CTI BioPharma

Criteria

Inclusion Criteria:

• Intermediate -1 or -2 or high-risk Myelofibrosis (per Passamonti et al 2010)

• Thrombocytopenia (platelet count ≤ 100,000/µL) at any time after signing informed consent

• Palpable splenomegaly ≥ 5 cm on physical examination

• Total Symptom Score ≥ 13 on the MPN-SAF TSS 2.0, not including the inactivity question

• Patients who are platelet or red blood cell transfusion-dependent are eligible

• Adequate white blood cell counts (with low blast counts), liver function, and renal function

• At least 6 months from prior splenic irradiation

• At least 1-4 weeks since prior myelofibrosis therapy, including any erythropoietic or thrombopoietic agent

• Not pregnant, not lactating, and agree to use effective birth control

• Able and willing to undergo frequent MRI or CT assessments and complete symptom assessments using a patient-reported outcome instrument

Exclusion Criteria:

• Prior treatment with more than 2 JAK2 inhibitors or with pacritinib

• There is no maximum cumulative prior JAK2 inhibitor treatment

• History of (or plans to undergo) spleen removal surgery or allogeneic stem cell transplant

• Ongoing gastrointestinal medical condition such as Crohn's disease, Inflammatory bowel disease, chronic diarrhea, or constipation

• Active bleeding that requires hospitalization during the screening period

• Cardiovascular disease, including recent history or currently clinically symptomatic and uncontrolled: congestive heart failure, arrhythmia, angina, QTc prolongation or other QTc risk factors, myocardial infarction

• Other malignancy within last 3 years other than certain limited skin, cervical, prostate, breast, or bladder cancers

• Other ongoing, uncontrolled illnesses (including HIV infection and active hepatitis A, B, or C), psychiatric disorder, or social situation that would prevent good care on this study

• Life expectancy < 6 months