PF-114 Dose-finding Study in Ph+ CML Patients Resistant to 2nd Generation TKI or Presenting T315I Mutation [Russia]

Study title

Dose-finding Study to Evaluate the Tolerability, Safety, Pharmacokinetics and Preliminary Efficacy of PF-114 in Adult Ph+ CML Patients Resistant to 2nd Generation TKI or Presenting T315I Mutation  [Russia]

Scientific title

A Multicenter, Open Label Cohort Phase 1 Dose Finding Study to Evaluate Tolerability, Safety, Pharmacokinetics and Preliminary Efficacy of PF-114 Mesylate 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
( No. NCT02885766)

Type of study

Trial after therapy failure or intolerance

Current status

No longer recruiting. Interim results available.

What is the purpose of the study

In this dose-finding study, PF-114 will be given by mouth, starting with a dose of 50 mg once daily in the first patient group. In the next patient groups, the dose is gradually increased up to 400 mg/day. The dose can be increased further based on the assessment of the safety findings by the investigators and the sponsor.

Key inclusion criteria

Male or female patients 18 years of age or older with Ph+ CML in chronic phase (CP) or accelerated phase (AP) according to European LeukemiaNet guidelines (2013) can be considered for inclusion if they are intolerant to approved Bcr-Abl inhibitors or have not responded to previous treatment with at least one 2nd generation inhibitor of Bcr-Abl (dasatinib or nilotinib or bosutinib) or if they present T315I mutation in the BCR-ABL gene. In case of previous history of blast crisis phase of CML at least 6 months must have passed after the end of blast crisis phase and intake of the first dose of PF-114. To be included, patients need to have an ECOG performance status below or equal to 2 and adequate kidney, liver and heart function.

Key exclusion criteria

Where can I find additional information

Study description in the US register, a service of the U. S. National Institutes of Health

Study sponsor

Fusion Pharma LLC

Scientific lead / contact

Veronika Shulgina MD, PhD
+7 916 815 05 44

Principal investigator

1. Prof. DMSci Anna Turkina
2. Elza Lomaia MD, PhD

Study centers / principal investigators

National Research Center for Hematology
Russia, 125 167, Moscow, Novii Zikovskii pr., 4

Principal Investigator:
Prof. DMSci Anna Turkina — Head of Myeloproliferative diseases
Clinical Study Coordinator:
Oleg Shuhov MD, PhD
+7 985 287 12 69

Moscow City Centre of Hematology based on City Hospital named by S.Botkin
5, 2-nd Botkin’s lane, build. 17, entrance 3
125284 Moscow

Principal Investigator:
Prof. DMSci Olga Vinogradova — Head of Moscow City Centre of Hematology
tel. +7(495)945-97-61

St. Petersburg
Federal Almazov North-West Medical Research Centre
Russia, 197 341, St. Petersburg, Akkuratova st., 2

Principal Investigator:
Elza Lomaia MD, PhD, — lead scientist of Onco-hematology Department
+7 981 936 16 67