LCL161 in Treating Patients With Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or … (NCT02098161) | Clinical Trial Compass
CompletedPhase 2
LCL161 in Treating Patients With Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocytosis Myelofibrosis
United States50 participantsStarted 2014-12-18
Plain-language summary
This phase II trial studies how well second mitochondrial-derived activator of caspases (SMAC) mimetic LCL161 (LCL161) works in treating patients with primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocytosis myelofibrosis. SMAC mimetic LCL161 may help control the growth of abnormal cells by promoting apoptosis (programmed cell death).
Who can participate
Age range18 Years
SexALL
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Inclusion Criteria:
* Patients must provide written informed consent
* Willing and able to comply with scheduled visits, treatment plan and laboratory tests
* Patient is able to swallow and retain oral medication
* Must be diagnosed with treatment requiring PMF or post ET/PV MF with intermediate-1, intermediate -2 or high risk disease according to the International Working Group (IWG) prognostic scoring system, or if with low risk disease then with symptomatic splenomegaly that is \>= 5 cm below left costal margin by physical exam
* Patients who are not candidates for, intolerant, or relapsed/refractory to ruxolitinib
* Eastern Cooperative Oncology Group (ECOG) performance status 0-2
* Absolute neutrophil count (ANC) \>= 0.5 x 10\^9/L (1500/mm\^3)
* Serum direct bilirubin =\< 2.0 x ULN (upper limit of normal)
* Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase \[SGOT\]) and alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) =\< 2.5 x ULN, except for patients with MF involvement of the liver who must have AST and ALT =\< 5 x ULN
* Serum creatinine =\< 1.5 x ULN
* Treatment-related toxicities from prior therapies must have resolved to grade =\< 1
* At least 2 weeks from prior MF-directed treatment (till the start of study drug)
Exclusion Criteria:
* Any concurrent severe and/or uncontrolled medical conditions that could increase the patient's risk for toxicity while in the study or that could confound discrimination betwee…