Third-line therapy for chronic myeloid leukemia: current status and future directions

Table 4 Ongoing clinical trials for BCR-ABL1–targeted therapies for CML in 3L + setting

Drug/trial	Trial number/phase	Goals	Primary endpoint(s)
HQP1351 (BCR-ABL1 Inhibitor) [132, 133]	NCT04126681/phase II	To evaluate the efficacy of HQP1351 in patients with CML-CP who are resistant and/or intolerant to 1G and 2GTKIs	EFS
HQP1351 (BCR-ABL1 Inhibitor) [132, 133]	NCT03883087/phase II	To evaluate the efficacy of HQP1351 in patients with CML-CP and a T315I mutation	MCyR
PF-114 (BCR-ABL1 Inhibitor) [72]	NCT02885766/phase I/II	To evaluate tolerability, safety, pharmacokinetics, and preliminary efficacy of PF-114 in patients with Ph + CML who are resistant to 2GTKIs or have the T315I mutation	DLTs MTD
K0706 (BCR-ABL1 Inhibitor) [75]	NCT02629692/phase I/II	To determine safety, tolerability, pharmacokinetics, and activity of K0706 in patients with CML or Ph + ALL	MTD TEAEs MCyR or partial cytogenetic response (CML-CP) CHR (CML-AP/BC)
Asciminib (STAMP inhibitor) [82, 86]	NCT03106779/phase III	To compare the efficacy of asciminib with that of bosutinib in patients with CML-CP in the 3L + setting	MMR at 24 weeks
Asciminib (STAMP inhibitor) [82, 86]	NCT02081378/phase I	A dose-finding study of asciminib alone or in combination with nilotinib, imatinib, or dasatinib in patients with CML and Ph + ALL who are relapsed/refractory to or are intolerant of TKIs	MTD and/or RDE DLTs

1G first generation, 2GTKI second-generation tyrosine kinase inhibitor, 3L third line, ALL acute lymphoid leukemia, CHR complete hematologic response, CML-AP chronic myeloid leukemia in acute phase, CML-BC chronic myeloid leukemia in blast crisis, CML-CP chronic myeloid leukemia in chronic phase, DLT dose-limiting toxicity, EFS event-free survival, MCyR major cytogenetic response, MMR major molecular response, MTD maximum tolerated dose, Ph+ Philadelphia chromosome positive, RDE recommended dose for expansion, STAMP Specifically Targeting the ABL Myristoyl Pocket, TEAE treatment-emergent adverse event, TKI tyrosine kinase inhibitor

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