Titre 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)
Protocole ID CPI-0610-02
ClinicalTrials.gov ID NCT02158858
Type(s) de cancer Syndrome myélodysplasique
Phase Phase II
Stade Myélofibrose
Type étude Traitement
Médicament CPI-0610 avec Ruxolitinib vs CPI-0610 seul
Institution CIUSSS DU CENTRE-OUEST-DE-L'ILE-DE-MONTREAL
   HOPITAL GENERAL JUIF SIR MORTIMER B.DAVIS
      3755 rue de la Côte Ste. Catherine, Montréal, QC, H3T 1E2
Ville Montréal
Investigateur(trice) principal(e) Dre Shireen Sirhan
Coordonnateur(trice) Chadi Zakaria
 514-340-8222 poste 28326
Statut Fermé
Critètes d'éligibilité
  • 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; see Appendix 3) 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
  • Anemic, defined as a hemoglobin < 10g/dL
  • At least 2 symptoms measurable (score ≥ 3) or a total score of ≥ 10 using the MFSAF v4.0
  • No prior treatment with JAKi allowed
Critètes d'exclusion
  • 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.