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Investigational Therapy for Previously Treated Patients with Myelofibrosis

Trial ID:
Marin Xavier, MD
This is a randomized, placebo controlled study to compare the safety and effectiveness of adding a PI3K inhibitor (INCB050465) to standard treatment for patients with Myelofibrosis previously treated with ruxolitinib.

Inclusion Criteria

Patients must:
  • Be diagnosed with Primary Myelofibrosis(PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia myelofibrosis (PET-MF).
  • Have been treated with ruxolitinib for =3 months with a stable dose for at least the last 8 weeks.
  • Have a suboptimal response to Ruxolitinib.
  • Be 18 years or older.
  • Be willing to avoid pregnancy, fathering a child, or donating sperm.

Exclusion Criteria

Patients must not:
  • Have been previously treated with any drug that inhibits PI3K (For example: Idelalisib, Duvelisib, Buparlisib, Copanlisib, and Umbralisib).
  • Have used any experimental drug therapy for Myelofibrosis or any other standard drug used for Myelofibrosis with the exception of Ruxolitinib, within 3 months of starting the study.
  • Have any splenic radiation in the past 6 months.
  • Have an active HBV, HCV, HIV, or other viral infection
  • Be currently breastfeeding or pregnant.

Additional Info

  • Eligible patients will be randomized to receive standard treatment with Ruxolitinib or the investigational combination of Ruxolitinib plus the study drug INCB050465.
  • This study is double-blind which means neither you or the study physician will know which group you are randomized to receive.
  • Bone Marrow Biopsy is required at screening and every 6 months while on trial.
  • This is a crossover study so you may have the option of receiving the investigational combination (Ruxolitinib + INCB050465) after 6 months. Study participation will last up to 2.5years.
  • Search NCT04551066 at https://clinicaltrials.gov website for more information.

Contact Info:

  • Clinical Research Services
  • CRSLeadership@scrippshealth.org