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Table 1 Clinical studies and results from non ABL-directed Inhibitors

From: Non ABL-directed inhibitors as alternative treatment strategies for chronic myeloid leukemia

Non ABL-directed Inhibitors Clinical Study Drugs Combination Patient Characteristics (pts) Results
Tipifarnib (42) CP, AP, BP
(n = 22)
HR: 27% CP, 4% AP
CyR: 18% CP
PhaseI NCT00040105(43) IM CP having ABL KD mutation
(n = 25)
HR: 76%
CyR: 36%
Lonafarnib Pilot Study(44) CP, AP
IM resistant
(n = 13)
HR: 8% CP, 8% AP
PhaseI NCT00047502(45) IM CP, AP, BP
(n = 23)
HR: 9% CP, 17% AP/BP
CyR: 4% CP, 4% AP/BP
Rapamycin PhaseI/II NCT00776373 Cytarabine Etoposide AP, BP NDP
Everolimus PhaseI/II NCT00081874 BP NDP
PhaseI/II NCT00093639 IM CP NDP
BEZ235 PhaseI NCT01756118 AP, BP NDP
Temsirolimus PhaseI NCT00101088 IM AP, BP NDP
LDE225 PhaseI NCT01456676 NIL AP, BP NDP
BMS833923 PhaseI NCT1218477(66) DAS CP, AP, BP
(n = 27)
CyR: 4% CP
PA/BP: no responded
PhaseI NCT01357655 DAS CP No participants were enrolled
PF-04449913 Phase I NCT00953758 CP Safety, Pharmacokinetics and Pharmacodynamics study
Ruxolitinib PhaseI NCT01702064 NIL CP ongoing
PhaseI/II NCT02253277 NIL CP, Ph + ALL ongoing
PhaseI/II NCT01751425 CP with MRD ongoing
PhaseI/II NCT01914484 NIL AP, BP, Ph + ALL ongoing
PhaseI/II NCT02973711 NIL CP ongoing
17-AAG PhaseI NCT00093821 BP NDP
PhaseI NCT00098423 Cytarabine BP NDP
STA-9090 PhaseI NCT00964873 BP NDP
PhaseI NCT00858572 refractory or relapsed CML NDP
Vorinostat PhaseI NCT00275080 Decitabine BP NDP
PhaseI NCT00816283 DAS AP, BP NDP
PhaseI NCT00278330 Flavopiridol BP NDP
LBH589 Phase II/III NCT00451035(91) CML TKIs resistant
(n = 29)
HR: 3%
PhaseI NCT00686218 IM CP with MRD NDP
Obatoclax PhaseI NCT00438178 BP NDP
MK-0457 Phase I/II(108) AP, BP, Ph + ALL
All with T315I
(n = 18)
HR: 39% AP/BP
CyR: 5% Ph + ALL
PhaseII NCT00405054(110) AP, BP, Ph + ALL
All with T315I
(n = 52)
CyR: 8% CP, 6% AP/BP
HR: 4% CP
PhaseI NCT00500006 DAS CP No Data Results Posted
PHA-739358 PhaseI(113) AP, BP
(n = 29)
HR: 7% AP/BP,
7% Ph + ALL
CyR: 3% AP/BP,
3% Ph + ALL
MR: 3% Ph + ALL
AT9283 PhaseI NCT00522990 CP, AP, BP NDP
XL288 PhaseI NCT00464113 CP, AP, BP, Ph + ALL NDP
Omacetaxine PhaseII(120) CP, TKIs resistant
(n = 46)
HR: 67%, CyR: 22
PhaseI/II(121) CP, previously
exposed to TKIs
(n = 81)
HR: 81%, CyR: 20%
PhaseII NCT00375219(122) CP, BCR-ABL1
T315I mutant
(n = 62)
HR: 77%, CyR: 22%
  1. FT-Is Farnesyl Transferase Inhibitors, mTOR-Is mammalian Target of Rapamycin, Smo-As Smo Antagonists, JAK2-Is JAK2 Inhibitors, Hsp-90-Is Hsp-90 Inhibitors, HDAC-Is HDAC Inhibitors, BCL2-Is BCL2 Inhibitors, AURK-Is Aurora Kinase Inhibitors, PT-Is Protein Translation Inhibitors, HR Hematological Remission, CyR Cytogenetic Remission, CP Chronic Phase, AP Accelerated Phase, BP Blast Phase, NDP No Data Posted, MRD Minimal Residual Disease