Refine Study for Myelofibrosis

In the open label single arm Phase 2 REFINE study, the investigational agent (navitoclax) will be administered either alone or in combination with ruxolitinib to evaluate its effect on controlling the signs and symptoms of myelofibrosis including impact on spleen size, blood counts, bone marrow fibrosis and quality of life.

Eligibility Criteria

  • 18 years of age or older
  • Have been diagnosed with primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis
  • Currently have Intermediate-2 or High-Risk myelofibrosis
  • Are ineligible or unwilling to undergo stem cell transplantation
  • Have received prior treatment with ruxolitinib or another JAK-2 inhibitor for myelofibrosis, however, prior treatment is not required for all patients
  • Have splenomegaly of at least 5 cm

Navitoclax, an investigational agent, is under clinical development and is not approved by regulatory health authorities. Safety and efficacy have not been established.

ENVELOP is a multi-part clinical research study for adults with Myelofibrosis (MF), Essential thrombocythemia (ET), Polycythemia vera (PV) or Chronic myelomonocytic leukemia (CMML). This information will be focusing on two parts of the study, Parts 3 and 4. You can learn more about Parts 3 and 4 of the study by reviewing the information below.

The study is for individuals requiring new treatment when previous treatment did not work, could not be tolerated or the individual declined to receive standard treatments. In addition to evaluating clinical outcomes with navitoclax monotherapy in these patients, Parts 3 and 4 will evaluate the following objectives:

Part 3: The main purpose of Part 3 is to understand the potential effect of navitoclax on the QT interval and other electrocardiogram measurements.

Part 4: The main purpose of Part 4 of is to understand the effects of the navitoclax on the pharmacokinetics and safety of a single dose of celecoxib (a CYP2C9 substrate).

Eligibility Criteria

  • Part 3 and Part 4 (Participants in US and Europe):
  • Part 3 Only: At screening or baseline (pre-dose on Day 1), participant has QT interval corrected for heart rate (QTc) interval by Fridericia’s correction (QTcF) <=450 msec.
  • Participants with a documented diagnosis of primary or secondary MF, ET, PV or chronic myelomonocytic leukemia (CMML) as defined by the WHO classification.
  • Participants must be requiring treatment and have failed or are intolerant to at least one prior therapy or who refuse standard therapy.
  • ECOG performance status <= 2.
  • Must have adequate bone marrow, kidney, liver and hematology blood values as detailed in the study protocol

Navitoclax, an investigational agent, is under clinical development and is not approved by regulatory health authorities. Safety and efficacy are under evaluation.

ClinicalTrials.gov Identifier: NCT04041050