Phase 2 Study of Oral Pacritinib for Safety and Effectiveness in Adults with Relapsed or Refractory Waldenstrf6m Macroglobulinemia

What this Trial Is About

Researchers are studying pacritinib as a possible treatment for people with Waldenstrm macroglobulinemia WM, a type of blood cancer. This Phase II trial aims to evaluate how safe and effective pacritinib is for participants whose disease has returned or not responded to previous treatments. Pacritinib works by blocking proteins, JAK2 and IRAK1, that help WM cells survive and grow. The U.S. Food and Drug Administration has approved pacritinib for another condition called Myelofibrosis, but not yet for WM. In this open-label study, about 30 participants will receive pacritinib capsules taken orally twice daily in 28-day cycles. The treatment period can last up to 4 years, including cycles 1 through 48 with specific visits for assessments at cycles 3, 6, 9, 12, and every 3 cycles thereafter. Bone marrow biopsies and aspirations will be performed at cycles 6 and 12 and then yearly. After treatment, participants will be followed every 12 weeks for 2 years or until they start a new therapy. Participants will undergo various tests and procedures including blood and urine tests, CT scans, X-rays, heart monitoring ECGs, and bone marrow biopsies. Questionnaires will also be completed. Researchers will assess response rates to treatment, time to response and survival outcomes, and monitor any treatment-related side effects. The total participation may last several years, combining treatment and follow-up visits to track safety and effectiveness.

A Phase 2 Study to Evaluate the Safety and Efficacy of Pacritinib in Relapsed or Refractory Waldenström Macroglobulinemia