Accelerated Phase MPN Clinical Trial
Official title:
A Phase 2 Open-label Study of Zelavespib (PU-H71) in Subjects With Accelerated Phase Myeloproliferative Neoplasm (AP MPN) or Blast Phase Myeloproliferative Neoplasm
This is a multicenter, Phase 2 Simon 2-Stage study designed to assess the safety, tolerability, PK, and efficacy of oral zelavespib (PU-H71) administered daily in adults with accelerated phase (10% to 19% blasts in peripheral or bone marrow) myeloproliferative neoplasm, with or without ongoing concomitant treatment with ruxolitinib.
Oral zelavespib 1501050 mg will be administered once daily (QD), in the morning ≥ 1 hour before eating breakfast, for each day of a 21-day cycle, for 6 cycles. Subjects who derive clinical benefit may continue treatment until disease progression, unacceptable toxicity, death, or study termination. In Stage 1, up to 2317 subjects will be enrolled and will complete 6 cycles of treatment. If fewer than 2 subjects achieve <CR?/PR?/CBR?/PBR?> in Stage 1, the trial will be stopped for futility. If 2 or more subjects achieve <CR?/PR?/CBR?/PBR?>a response , an additional 33 subjects will be enrolled, enrollment will begin in Stage 2 for a total of 56 subjects. . If fewer than 2 subjects achieve a response in Stage 1, the trial will be stopped for futility. In Cycle 1, subjects will attend 3 clinic visits (Day 1, Day 8, and Day 15) and will be contacted by phone by the site at approximately Day 4 . In subsequent cycles, subjects will attend a clinic visit on Day 1 onlyand will be contacted by phone on Days 8 and 15 so the site staff can inquire about changes in concomitant medications and potential AEs. On days of clinic visits, subjects will arrive at the clinic without eating anything in the morning or taking the study drug. Predose biological samples will be collected for safety laboratory tests and other clinical assessments, and subjects will undergo physical examinations and ECGs. Subjects will receive the study drug at the clinic and at least 1 hour after dosing, breakfast will be provided. On some clinic days, subjects may be required to remain in the clinic for up to 8 hours for additional ECGs and collection of samples for PK testing. Each subject will participate in the study for approximately 6.5 months , which includes a 28-day screening period, 6 cycles of treatment, and a final follow-up visit 30 days after the final dose of study treatment. ;