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Study of Navtemadlin add-on to Ruxolitinib in JAK Inhibitor-Naïve Patients With Myelofibrosis Who Have a Suboptimal Response to Ruxolitinib
Trial Status: active
This clinical trial is evaluating whether addition of navtemadlin to ruxolitinib
treatment will provide more clinical benefit than ruxolitinib alone for patients with
Myelofibrosis who have a suboptimal response to ruxolitinib treatment alone.
Subjects will start by receiving ruxolitinib alone in the run-in period. Those who
demostrate a suboptimal response from ruxolitinib alone will then be randomized 2:1 to
receive navtemadlin or navtemadlin placebo as add-on treatment to their ongoing
ruxolitinib. Randomized means that subjects will be assigned to a group by chance, like a
flip of a coin. The study is blinded, meaning the subjects, doctors, central endpoint
assessors and sponsor will not know which add on treatment (navtemadlin or navtemadlin
placebo) the subject is receiving.
Inclusion Criteria
Inclusion Criteria for Ruxolitinib Alone Period:
- Confirmed diagnosis of PMF, post-PV MF, or post-ET MF, as assessed by the treating
physician according to the World Health Organization (WHO) criteria
- High, Intermediate-1, Intermediate-2 risk category International Prognosis System
Score (IPSS)
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2
- JAK-inhibitor treatment naive
Exclusion Criteria for Ruxolitinib Alone Period:
- Prior Splenectomy
- Splenic irradiation within 3 months prior to the first dose
- Prior BCL-XL, BET, MDM2, PI3K, PIM, or XPO1 inhibitors therapy or p53-directed
therapy
- Eligible for Bone Marrow Transplant
- Peripheral blood or bone marrow blast count ≥ 10 percent
Inclusion Criteria for Randomized Period:
- PMF, post-PV MF, or post-ET MF that is TP53WT as assessed by central testing
- ECOG performance status of 0 to 2
- Treatment with a stable dose of ruxolitinib
- Suboptimal response to run-in ruxolitinib treatment
Exclusion Criteria for Randomized Period:
- Elevated white blood cell count that doubles (or more) during ruxolitinib treatment
and exceeds 50 × 10^9/L
- Peripheral blood or bone marrow blast count ≥ 10 percent
Additional locations may be listed on ClinicalTrials.gov for NCT06479135.
