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Table 3 A selection of active studies evaluating BCL-2 and JAK/STAT inhibitors in AML

From: Molecular targeting in acute myeloid leukemia

Protocol title NCT identifier
Agent: BCL-2 inhibitors
 Phase I dose-escalation study of the orally administered selective Bcl-2 Inhibitor S 055746 as monotherapy for the treatment of patients with acute myeloid leukaemia (AML) or high or very high risk myelodysplastic syndrome (MDS) (Sponsor: Institut de Recherches Internationales Servier) NCT02920541
 A phase 2a, open-label, dose-escalation study evaluating the safety, pharmacokinetics, pharmacodynamics, and clinical effects of intravenously administered nerofe in subjects with acute myelogenous leukemia or myelodysplastic syndrome (Sponsor: Immune System Key Ltd) NCT03059615
 A phase 1b study evaluating the safety, pharmacokinetics and efficacy of venetoclax as a single-agent and in combination with azacitidine in subjects with higher-risk myelodysplastic syndromes after hypomethylating agent-failure (Sponsor: AbbVie) NCT02966782
 A phase I and expansion cohort study of venetoclax in combination with chemotherapy in pediatric patients with refractory or relapsed acute myeloid leukemia (Sponsor: St. Jude Children’s Research Hospital) NCT03194932
 A randomized, double-blind, placebo controlled study of venetoclax co-administered with low dose cytarabine versus low dose cytarabine in treatment naïve patients with acute myeloid leukemia who are ineligible for intensive chemotherapy (Sponsor: AbbVie) NCT03069352
 A randomized, double-blind, placebo controlled phase 3 study of venetoclax in combination with azacitidine versus azacitidine in treatment naïve subjects with acute myeloid leukemia who are ineligible for standard induction therapy (Sponsor: AbbVie) NCT02993523
 A phase 1b study of ABT-199 (GDC-0199) in combination with azacitidine or decitabine in treatment-naive subjects with acute myelogenous leukemia who are ≥60 years of age and who are not eligible for standard induction therapy (Sponsor: AbbVie) NCT02203773
Agent: JAK/STAT inhibitors
 Induction therapy with pacritinib combined with decitabine or cytarabine in older patients with acute myeloid leukemia (AML) (Sponsor: Weill Medical College of Cornell University) NCT02532010
 Phase I study of pacritinib and chemotherapy in patients with acute myeloid leukemia and FLT3 mutations (Sponsor: Ohio State University Comprehensive Cancer Center) NCT02323607