Phase III MANIFEST-2: pelabresib + ruxolitinib vs placebo + ruxolitinib in JAK inhibitor treatment-naive myelofibrosis.
Claire N HarrisonVikas K GuptaAaron T GerdsRaajit K RampalSrdan VerstovsekMoshe TalpazJean-Jacques KiladjianRuben A MesaAndrew T KuykendallAlessandro Maria Maria VannucchiFrancesca PalandriSebastian GrosickiTimothy DevosEric JourdanMarielle J WondergemHaifa Kathrin Al-AliVeronika Buxhofer-AuschAlberto Álvarez-LarránAndrea PatriarcaMarina KremyanskayaAdam J MeadSanjay AkhaniYuri SheikineGozde ColakJohn O MascarenhasPublished in: Future oncology (London, England) (2022)
Myelofibrosis (MF) is a clonal myeloproliferative neoplasm, typically associated with disease-related symptoms, splenomegaly, cytopenias and bone marrow fibrosis. Patients experience a significant symptom burden and a reduced life expectancy. Patients with MF receive ruxolitinib as the current standard of care, but the depth and durability of responses and the percentage of patients achieving clinical outcome measures are limited; thus, a significant unmet medical need exists. Pelabresib is an investigational small-molecule bromodomain and extraterminal domain inhibitor currently in clinical development for MF. The aim of this article is to describe the design of the ongoing, global, phase III, double-blind, placebo-controlled MANIFEST-2 study evaluating the efficacy and safety of pelabresib and ruxolitinib versus placebo and ruxolitinib in patients with JAKi treatment-naive MF. Clinical Trial Registration: NCT04603495 (ClinicalTrials.gov).
Keyphrases
- phase iii
- double blind
- placebo controlled
- clinical trial
- open label
- phase ii
- end stage renal disease
- newly diagnosed
- bone marrow
- healthcare
- small molecule
- chronic kidney disease
- ejection fraction
- study protocol
- prognostic factors
- palliative care
- patient reported
- phase ii study
- patient reported outcomes
- pain management