Study to Evaluate Tolerability, Safety, Pharmacokinetics and Preliminary Efficacy of PF-114 for Oral Administration in Adults With Ph+ Chronic Myeloid Leukemia, Which is Resistant to the 2-nd Generation Bcr-Abl Inhibitors or Has T315I Mutation in the BCR-ABL Gene

Summary

A multicenter, open label cohort Phase 1 dose finding study to evaluate tolerability, safety, pharmacokinetics and preliminary efficacy of PF-114 for oral administration in adult patients with Philadelphia chromosome positive (Ph+) chronic myeloid leukemia (CML), which is resistant to the 2-nd generation Bcr-Abl inhibitors or has T315I mutation in the BCR-ABL gene.

Timeline

Start

2016-07

Primary completion

2018-06

Completion

2020-05

Publications

• Results Mian AA, Rafiei A, Haberbosch I, Zeifman A, Titov I, Stroylov V, Metodieva A, Stroganov O, Novikov F, Brill B, Chilov G, Hoelzer D, Ottmann OG, Ruthardt M. PF-114, a potent and selective inhibitor of native and mutated BCR/ABL is active against Philadelphia chromosome-positive (Ph+) leukemias harboring the T315I mutation. Leukemia. 2015 May;29(5):1104-14. doi: 10.1038/leu.2014.326. Epub 2014 Nov 14.
• Turkina A, Vinogradova O, Lomaia E, Shatokhina E, Shukhov O, Chelysheva E, Shikhbabaeva D, Nemchenko I, Petrova A, Bykova A, Siordiya N, Shuvaev V, Mikhailov I, Novikov F, Shulgina V, Hochhaus A, Ottmann O, Cortes J, Gale RP, Chilov G. Phase-1 study of vamotinib (PF-114), a 3rd generation BCR::ABL1 tyrosine kinase-inhibitor, in chronic myeloid leukaemia. Ann Hematol. 2025 May;104(5):2707-2715. doi: 10.1007/s00277-025-06239-8. Epub 2025 Apr 29.

Drugs

Indications