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smac 2.0 registration id
Layout table for study information Study Type : Interventional (Clinical Trial) ActualEnrollment : 53 participants Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment Official Title: Open Label Phase 2 Single Agent Study of LCL-161 in Patients With Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (Post-PV MF), or Post-Essential Thrombocytosis Myelofibrosis (Post-ET MF) Actual Study Start Date : December 18, 2014 Actual Primary Completion Date : May 19, 2022 Actual Study Completion Date : May 19, 2022 Resource links provided by the National Library of Medicine MedlinePlus Genetics related topics: Essential thrombocythemia Primary myelofibrosis Polycythemia vera Genetic and Rare Diseases Information Center resources: Primary Myelofibrosis Polycythemia Vera Essential Thrombocythemia Chronic Myeloproliferative Disorders U.S. FDA Resources Arms and Interventions Go to Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information Arm Intervention/treatment Experimental: Treatment (SMAC mimetic LCL161)Patients receive SMAC mimetic LCL161 PO on days 1, 8, 15, and 22. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Other: Laboratory Biomarker AnalysisCorrelative studies Other: Questionnaire AdministrationAncillary studies Drug: Smac Mimetic LCL161Given POOther Name: LCL161 Outcome Measures Go to Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information Primary Outcome Measures : Objective response rate (ORR) [ Time Frame: After 84 days (3 courses) of treatment ]Will be defined as complete remission (CR), partial remission (PR), or clinical improvement (CI) after 3 courses of treatment according to International Working Group (IWG) consensus criteria for myelofibrosis. The Optimum two-stage design proposed by Simon will be implemented. ORR will be estimated along with the Bayesian 95% credible interval. Secondary Outcome Measures : Incidence of grade 3-4 clinically relevant non-hematologic toxicity or a serious adverse event that is at least possibly drug related [ Time Frame: Up to 30 days post-treatment ]Will be graded by the National Cancer Institute Common Terminology Criteria for Adverse Events version 4.03. Safety data will be summarized by category, severity and frequency. The proportion of patients with adverse events will be estimated, along with the Bayesian 95% credible interval. Duration of response [ Time Frame: Date at which the subject's objective status is first noted to the date of progression (no longer meeting criteria for any type of response), assessed up to 30 days post-treatment ]The distribution for duration of response will be estimated by Kaplan-Meier curves. Time to response [ Time Frame: Time from study registration to the first date at which the subject's objective status was classified as a response, assessed up to 30 days post-treatment ]The distribution for time to response will be estimated by Kaplan-Meier curves. Eligibility CriteriaGo to Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information Information from the National Library of Medicine Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies. Layout table for eligibility information Ages Eligible for Study: 18 Years and older (Adult, Older Adult) Sexes Eligible for Study: All Accepts Healthy Volunteers: No Criteria Inclusion Criteria: 2ff7e9595c
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