Clinical Trials /

Study of CPI-0610 in Myelofibrosis (MF)

NCT04603495

Description:

A Phase 3, randomized, blinded study comparing CPI-0610 and ruxolitinib with placebo and ruxolitinib in myelofibrosis (MF) patients that have not been exposed previously to Janus kinase inhibitors (JAKi). CPI-0610 is a small molecule inhibitor of bromodomain and extra-terminal (BET) proteins. Evidence suggests that inhibition of both BET and JAK pathways can result in synergistic reduction of disease and overall improvement in the prognosis of MF.

Related Conditions:
  • Myelofibrosis Transformation in Essential Thrombocythemia
  • Polycythemia Vera, Post-Polycythemic Myelofibrosis Phase
  • Primary Myelofibrosis
Recruiting Status:

Recruiting

Phase:

Phase 3

Trial Eligibility

Document

Title

  • Brief Title: Study of CPI-0610 in Myelofibrosis (MF)
  • Official Title: A Phase 3, Randomized, Double-blind, Active-Control Study of CPI-0610 and Ruxolitinib vs. Placebo and Ruxolitinib in JAKi Treatment Naive MF Patients

Clinical Trial IDs

  • ORG STUDY ID: CPI 0610-04
  • SECONDARY ID: 2020-001989-10
  • NCT ID: NCT04603495

Conditions

  • Myelofibrosis
  • Primary Myelofibrosis
  • Post-polycythemia Vera Myelofibrosis
  • Post-essential Thrombocythemia Myelofibrosis

Interventions

DrugSynonymsArms
CPI-0610CPI-0610 + ruxolitinib
RuxolitinibCPI-0610 + ruxolitinib
PlaceboPlacebo + ruxolitinib

Purpose

A Phase 3, randomized, blinded study comparing CPI-0610 and ruxolitinib with placebo and ruxolitinib in myelofibrosis (MF) patients that have not been exposed previously to Janus kinase inhibitors (JAKi). CPI-0610 is a small molecule inhibitor of bromodomain and extra-terminal (BET) proteins. Evidence suggests that inhibition of both BET and JAK pathways can result in synergistic reduction of disease and overall improvement in the prognosis of MF.

Trial Arms

NameTypeDescriptionInterventions
CPI-0610 + ruxolitinibExperimentalCPI-0610 monohydrate tablets + ruxolitinib phosphate tablets
  • CPI-0610
  • Ruxolitinib
Placebo + ruxolitinibActive ComparatorMatching placebo tablets + ruxolitinib phosphate tablets
  • Ruxolitinib
  • Placebo

Eligibility Criteria

        Inclusion Criteria:

          -  Aged ≥ 18 years

          -  Confirmed diagnosis of myelofibrosis (primary, post-polycythemia vera, or post
             essential thrombocythemia)

          -  Adequate hematologic, renal, and hepatic function

          -  Have at least 2 symptoms with an average score ≥ 3 or an average total score of ≥ 10
             over the 7-day period prior to randomization using the MFSAF v4.0

          -  Prognostic risk-factor score of Intermediate-1 or higher per Dynamic International
             Prognostic Scoring System (DIPSS) scoring system

          -  Spleen volume of ≥ 450 cm^3

          -  Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2

        Exclusion Criteria:

          -  Splenectomy or splenic irradiation in the previous 6 months

          -  Chronic or active conditions and/or concomitant medication use that would prohibit
             treatment

          -  Had prior treatment with any JAKi or BET inhibitor for treatment of a
             myeloproliferative neoplasm
      
Maximum Eligible Age:N/A
Minimum Eligible Age:18 Years
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Splenic response at Week 24
Time Frame:24 weeks of treatment
Safety Issue:
Description:The primary endpoint of the study is splenic response, defined as a ≥35% reduction from baseline in spleen volume as measured by magnetic resonance imaging (MRI) or computerized tomography (CT), at Week 24.

Secondary Outcome Measures

Measure:Total Symptom Score response at Week 24
Time Frame:24 weeks of treatment
Safety Issue:
Description:The key secondary endpoint of the study is Total Symptom Score response, defined as a ≥50% decrease from baseline in Total Symptom Score as measured by the Myelofibrosis Symptom Assessment Form v4.0, at Week 24. A higher Total Symptom Score indicates a higher disease burden and thus a worse outcome.

Details

Phase:Phase 3
Primary Purpose:Interventional
Overall Status:Recruiting
Lead Sponsor:Constellation Pharmaceuticals

Last Updated

November 24, 2020