for people ages 18 years and up (full criteria)
study started
estimated completion
Principal Investigator
by Gary Schiller, MD (ucla)



Phase 1 Part (Complete): Open-label, sequential dose escalation study of CPI-0610 in patients with previously treated Acute Leukemia, Myelodysplastic Syndrome, Myelodysplastic/Myeloproliferative Neoplasms, and Myelofibrosis. Phase 2 Part: Open-label study of CPI-0610 with and without Ruxolitinib in patients with Myelofibrosis. CPI-0610 is a small molecule inhibitor of bromodomain and extra-terminal (BET) proteins.

Official Title

A Phase 1/2 Study of CPI-0610, a Small Molecule Inhibitor of BET Proteins: Phase 1 (in Patients With Hematological Malignancies) and Phase 2 (Dose Expansion of CPI-0610 With and Without Ruxolitinib in Patients With Myelofibrosis)


Myelofibrosis Leukemia, Myelocytic, Acute Myelodysplastic/Myeloproliferative Neoplasm Myelodysplastic Syndrome (MDS) Phase 1 Phase 2 Oncology BET Inhibitor Ruxolitinib Pelabresib Preleukemia Leukemia, Myeloid Leukemia, Myeloid, Acute Myelodysplastic Syndromes Primary Myelofibrosis Myeloproliferative Disorders Myelodysplastic-Myeloproliferative Diseases CPI-0610


You can join if…

Open to people ages 18 years and up

  • Adult (aged ≥ 18 years)
  • Phase 2 part: Patients with confirmed diagnosis of MF who meet all of the following criteria:
  • Dynamic International Prognostic Scoring System (DIPSS) risk category of intermediate-1 or higher.
  • ANC ≥ 1 x 109/L without the assistance of granulocyte growth factors

  • Peripheral blood blast count <10%
  • ECOG performance status ≤ 2.
  • Adequate hematological, renal, hepatic, and coagulation laboratory assessments
  • Patients must give written informed consent to participate in this study before the performance of any study-related procedure.

For Arm 1 and 2 the following criteria should be considered:

  • Palpable spleen ≥ 5 cm that is below the costal margin on physical examination OR RBC transfusion dependent (defined as an average of ≥2 units of RBC transfusions per month over the 12 weeks prior to enrollment)
  • At least 2 symptoms measurable (score ≥ 1) using the Myelofibrosis Symptom Assessment Form Version 4.0 (MFSAF v4.0)
  • Platelet count ≥ 75 x 109/L without the assistance of thrombopoietic factors or transfusions for at least 14 days

  • Monotherapy Arm (Arm 1): Previously treated with a JAK inhibitor and be intolerant, resistant, refractory or lost response to the JAK inhibitor
  • Combination Arm (Arm 2): Must have received single agent ruxolitinib and be on a stable dose for a minimum 8 weeks

For Arm 3 (JAK inhibitors naïve) the following criteria should be considered:

  • Platelet count ≥ 100 x 109/L without the assistance of thrombopoietic factors or transfusions

  • Palpable spleen ≥ 5 cm that is below the costal margin on physical examination
  • At least 2 symptoms measurable (score ≥ 3) or a total score of ≥ 10 using the MFSAF v4.0
  • No prior treatment with JAKi allowed

You CAN'T join if...

  • Current known active or chronic infection with human immunodeficiency virus (HIV), Hepatitis B or Hepatitis C.
  • Impaired cardiac function or clinically significant cardiac diseases
  • Patients with Child-Pugh Class B or C
  • Impairment of gastrointestinal (GI) function or GI disease that could significantly alter the absorption of CPI-0610 and/or ruxolitinib, including any unresolved nausea, vomiting, or diarrhea that is CTCAE grade >1
  • Prior treatment with a BET inhibitor.
  • Pregnant or lactating women
  • Any other concurrent severe and/or uncontrolled concomitant medical condition that could compromise participation in the study
  • Patients unwilling or unable to comply with this study protocol.


  • UCLA Medical Center accepting new patients
    Los Angeles California 90095 United States
  • Mayo Clinic - Research Center accepting new patients
    Phoenix Arizona 85054 United States

Lead Scientist at University of California Health

  • Gary Schiller, MD (ucla)
    Professor-in-Residence, Medicine. Authored (or co-authored) 75 research publications.


accepting new patients
Start Date
Completion Date
Constellation Pharmaceuticals
Phase 1/2
Study Type
Last Updated