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A Phase 2 Study of CPI-0610 With and Without Ruxolitinib in Patients With Myelofibrosis

Trial Status: Active

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.

Inclusion Criteria

  • 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 10^9/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 10^9/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 10^9/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

Exclusion Criteria

  • 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.

California

Los Angeles
UCLA / Jonsson Comprehensive Cancer Center
Status: ACTIVE
Contact: Vlad Kustanovich
Phone: 310-206-5755

Illinois

Chicago
Northwestern University
Status: ACTIVE

Massachusetts

Boston
Dana-Farber Cancer Institute
Status: IN_REVIEW
Contact: Martha Wadleigh
Phone: 617-632-6685
Massachusetts General Hospital Cancer Center
Status: CLOSED_TO_ACCRUAL
Contact: Gabriela Soriano Hobbs
Phone: 617-724-1124

Michigan

Ann Arbor
University of Michigan Comprehensive Cancer Center
Status: ACTIVE

Missouri

Saint Louis
Siteman Cancer Center at Washington University
Status: ACTIVE

New York

New York
Icahn School of Medicine at Mount Sinai
Status: ACTIVE
Memorial Sloan Kettering Cancer Center
Status: COMPLETED
Contact: Eytan M Stein
Phone: 212-639-3314
NYP / Columbia University Medical Center / Herbert Irving Comprehensive Cancer Center
Status: ACTIVE

Pennsylvania

Philadelphia
University of Pennsylvania / Abramson Cancer Center
Status: COMPLETED

Texas

Houston
M D Anderson Cancer Center
Status: ACTIVE
San Antonio
Cancer Therapy and Research Center at The UT Health Science Center at San Antonio
Status: WITHDRAWN
Contact: Robyn Marie Scherber
Phone: 210-450-8605

Trial Phase Phase I/II

Trial Type Treatment

Lead Organization
Constellation Pharmaceuticals

  • Primary ID 0610-02
  • Secondary IDs NCI-2014-02045
  • Clinicaltrials.gov ID NCT02158858