In the randomized open-label Phase 3 TRANSFORM-2 study, the investigational medication (navitoclax) in combination with ruxolitinib is compared to best available therapy to evaluate its effect on splenomegaly, bone marrow fibrosis, anemia responses and quality of life in relapsed/refractory myelofibrosis patients.
- Are 18 years of age or older
- Have been diagnosed with primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis, characterized by bone marrow fibrosis grades 2 or 3.
- Currently have Intermediate-2 or High-Risk myelofibrosis
- Have received prior treatment with a JAK-2 inhibitor therapy, ruxolitinib
- Treatment with ruxolitinib for ≥ 24 weeks that was stopped due to lack of spleen response (refractory), or loss of spleen response or symptom control after a previous response (relapsed), or was continued despite relapsed/refractory status.
- Treatment with ruxolitinib for < 24 weeks with documented disease progression
- Prior or current treatment with ruxolitinib of at least 10 mg BID for ≥ 28 days with intolerance defined as new RBC transfusion requirement (at least 2 units/month for 2 months) while receiving a total daily ruxolitinib dose of ≥ 30 mg but unable to reduce dose further due to lack of efficacy.
- Have splenomegaly of at least 5 cm or spleen volume ≥ 450 cm3
- Have at least 2 symptoms each with an average score ≥ 3 or an average total score of ≥ 12, as measured by the MFSAF v4.0 prior to randomization. Subject must complete the MFSAF v4.0 on at least 4 out of the 7 days immediately prior to the date of randomization and must agree to collect MFSAF data daily by ePRO device during the study collection window.
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: NCT04468984
EudraCT Number: 2020-000557-27