MPN Clinical Trial Highlights for Summer 2015

If you have MF, PV or ET but don’t need medication now, you may think that clinical trials don’t matter to you. But that could change, at which point you may want to review your options. Thankfully, over the past years the interest in developing treatments for people living with these rare, chronic blood cancers has exploded. Here are some clinical trials we’re keeping our eye on:

Baxalta / CTI BioPharma had good press at ASCO with details on their Pacritinib trial and are recruiting more patients at this time for their trial “Persist-2: A randomized, controlled Phase 3 study of oral Pacritinib vs. best available therapy in patients with thrombocytopenia and primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis” For trial locations and more information at

Gilead is conducting a Phase 3 trial of momolotinib, and is recruiting patients with Primary Myelofibrosis (MF), Post-polycythemia Vera MF, or Post-essential Thrombocythemia MF. Find trial locations and more information here.

Janssen acquired Imetelstat from Geron and has an open study to Evaluate Activity of 2 Dose Levels of Imetelstat in Participants With Intermediate-2 or High-Risk Myelofibrosis (MF) Previously Treated With Janus Kinase (JAK) Inhibitor. For more information Contact Laurie Sherman at

KaloBios Pharmaceuticals, Inc. is now recruiting the Phase 2 portion of their trial of KB004 in MF and MDS patients. The study consists of IV infusion once weekly for a 21 day dosing cycle with KB004 250 mg (the recommended Phase 2 dose).   This portion of the study will be to further study the activity of KB004 and explore the safety of KB004  For more information contact Kate Doherty at / 1-650-243-3126. They’ve also made further information on this study available here.

The Myeloproliferative Disorders Research Consortium (MPD-RC) is an international, multi-institutional non profit consortium funded by the National Cancer Institute set up to coordinate, facilitate, and perform basic and clinical research investigating with the ultimate goal of developing new treatment options for people with PV, ET and MF. They are doing several investigator-initiated clinical trials now, including trials with Pegasys, RG7388, decitabine + ruxolitinib and more.  Patients interested in learning more can reach Dr. John Mascerenhas directly at / 212 241 3417 or reach Ruben Mesa at / 480-301-8335. Information on these trials is also available here.

Novartis is testing LCL-161 an antagonist of inhibitor of apoptosis protein (IAP), which protect cancer cells from cell death by inhibiting caspases—the enzymes that mediate apoptosis. Their trial” Phase 2 LCL-161 in Patients With Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (Post-PV MF) or Post-Essential Thrombocytosis Myelofibrosis (Post-ET MF)” is recruiting right now at University of Texas, MD Anderson Cancer Center. For more information Contact: Naveen Pemmaraju, MD at 713-792-4956.

NS-Pharma is actively recruiting for their trial “Safety and Tolerability Study of Oral NS-018 in Patients with Primary Myelofibrosis (MF), Post-PV MF or Post-ET MF” with various locations across the United States. For more information contact Tomonori Uno of NS Pharma at:

Pfizer is testing their Hedgehog Inhibitor PF-04449913 in people who have been previously treated with a JAK Inhibitor. Please check this link as sites are continuously added or contact the Pfizer Clinical Trial Call center: 1-800-718-1021  

This is meant to be informative rather than to suggest a course of action. It is our policy to share any relevant trial in the MPN space that is Phase II, III or IV. Remember to consult your doctor if you're considering participation in any clinical trial!

Author: Woehrle, Michelle

Michelle Woehrle joined the MPN Research Foundation in 2007 after being inspired by their mission to fund research that will improve the lives of people living with the rare blood cancers PV, ET and MF. In 2015 she was promoted to Executive Director. She has assisted with the expansion of the grants program, grassroots initiatives to connect with more patients internationally and optimize operations for the Foundation.

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