Chronic Myeloid LeukemiaPhase 1/2NCT02885766
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
A Phase 1/2 study testing PF-114 in people with Chronic Myeloid Leukemia and Leukemia, Myelogenous, Chronic, BCR-ABL Positive, run by Fusion Pharma LLC at 3 sites in Russia.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Myeloid Leukemia, Leukemia, Myelogenous, Chronic, BCR-ABL Positive
- What is being tested?
- PF-114
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 65 participants
- Where
- 3 sitesRussia
- Study dates
- Jul 2016to May 2020
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients must meet all of the following criteria in order to be eligible for participation in the study:
- Able to give written informed consent;
- Male or female patient ≥ 18 years old;
- Confirmed diagnosis of CML in chronic or accelerated phase according to European LeukemiaNet guideline as of 2013;
- Available information regarding resistance to the therapy with least one 2-nd generation Bcr-Abl inhibitor (dasatinib or nilotinib or bosutinib), or intolerance of approved Bcr-Abl inhibitors, or presence of T315I mutation irrespective of treatment history;
- In case of previous history of blast crisis phase of CML at least 6 months are required to pass after the end of blast crisis phase before the first dose of PF-114;
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- ECOG performance status ≤ 2 (see Appendix 2);
- Adequate renal function defined as serum creatinine ≤ 1.5 times upper limit of normal (ULN);
- Adequate hepatic function defied as:
- serum bilirubin ≤ 1.5 X ULN unless a patient is diagnosed with Gilbert's syndrome;
- serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 X ULN;
- alkaline phosphatase ≤ 2.5 X ULN;
- INR ≤ 1.5 X ULN;
- Adequate cardiac function defined as LVEF > 40 % by echocardiogram;
- QTcF < 470 ms;
- Patient has recovered (to Grade 1 or less according to NCI CTCAE V 4.0) from toxicities (excluding alopecia) associated with any prior treatments;
- Female patients of childbearing potential and male patients who have female partners of childbearing potential must agree with abstinence from sexual relations or use effective methods of contraception throughout participation in the study;
- Ability to comply with study procedures in the Investigator's opinion.
Who cannot join
- Patients must not meet any of the following criteria in order to be eligible for participation in the study:
- Use of the following previous therapy:
- chemotherapy ≤ 21 days (except hydroxyurea for which washout is not required) prior to the first dose of PF-114 mesylate; оr nitrosoureas оr mitomycin С ≤ 42 days prior to the first dose of PF-114 mesylate;
- approved tyrosine kinase inhibitors or investigational agents ≤ 4 days prior to the first dose of PF-114;
- radiotherapy ≤ 28 days prior to the first dose of PF-114 ;
- autologous оr allogeneic stem сеll transplant < 90 days prior to enrollment;
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- Significant uncontrolled cardiac disease;
- Sustained uncontrolled hypertension ≥ Grade 2 (according to NCI CTC AE v4);
- Patient is taking medicinal products known to prolong the QT interval on the electrocardiogram, unless they are absolutely necessary in the opinion of the investigator;
- Evidence of on-going graft versus host disease (GVHD), or GVHD requiring immunosuppressive therapy. Patients should be off immunosuppressive therapy for prophylaxis and/or treatment for at least 14 days prior to the first dose of PF-114;
- Major surgery within 35 days prior to enrollment;
- Uncontrolled intercurrent illness including, but not limited to the following: active systemic infection, uncontrolled seizure disorder, psychiatric or social circumstances that would limit compliance with study requirements or misrepresent results of the study;
- Patient is unable to swallow study drug or has gastro-intestinal disorders that could negatively affect oral absorption of PF-114 ;
- Any malignancy other than CML within the past 3 years (except for non-melanoma skin cancer or cervical cancer in situ).
- Pregnancy or breast feeding.
Trial locations and contacts
3 sites in 1 country.
Russia3 sites
Federal Haematological Scientific Center
Moscow, 125167
Moscow City Centre of Hematology based on City Hospital named by S.Botkin
Moscow, 125284
Federal Almazov North-West Medical Research Centre
Saint Petersburg
About this study
Official 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
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.
Read the detailed description
PF-114 is a low molecular inhibitor of a Bcr-Abl kinase activity, which is active with respect to native and mutated forms of this enzyme with mutations in Abl kinase domain. Preclinical in vitro and in vivo studies have demonstrated the ability of PF-114 to inhibit wild Bcr-Abl type and with T315I mutation, as well as other kinds of Bcr-Abl with mutations in kinase domain, including combined mutations.
In contrast to ponatinib, PF-114 is being developed to increase the action selectivity with respect to Bcr-Abl, which potentially should increase safety of drug application in people. The results of performed preclinical studies confirmed improved selectivity of PF-114 action with respect to Bcr-Abl kinases as compared to ponatinib.
Indication:
Adult patients with Ph+ CML in chronic phase (CP) or accelerated phase (AP) resistant to previous treatment with at least one 2-nd generation inhibitor of Bcr-Abl (dasatinib, nilotinib, bosutinib) or intolerant of approved Bcr-Abl inhibitors or with T315I mutation in the BCR-ABL gene
- Being tested
- PF-114
- Keywords
- CML, Myeloid leukaemia chronic, CML progression, Chronic phase chronic myeloid leukemia, PF-114, Cytogenetic response CCyR, Major molecular response MMR, Complete molecular response CMR, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, 2-nd generation Bcr-Abl inhibitors resistant
- Medical subject headings
- Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Leukemia, Myeloid, Chronic-Phase
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PF-114Drug
| Study group | Role | What they receive |
|---|---|---|
| PF-114 | Experimental | PF-114 From 50 mg up to the MTD. Dose escalation for each next cohort is conducted by increasing the dose by 20 % (or the closest lower level, which is a multiple of 25 mg) if there are Grade 3 ADRs according to NCI CTC AE v.4 without reaching а MTD. An increase of the dose by 40 % is applied if there were Grade 2 ADRs. In the absence of Grade 2 or 3 ADRs an increase of 100 % is applied.
When the dose reaches 400 mg/day, the following increase in dose can be made after discussing results of safety findings of PF-114 between the Investigators and the Sponsor.
Orally, once daily Receives: Drug: PF-114 |
What is being measured
The primary outcome is the main question the study is designed to answer.
DLTs during the first cycle of therapy
To study the dose-limiting toxicities (DLTs) of PF-114 mesylate in the target patient population during the 1-st cycle of treatment
Measured: 1-st Cycle of Therapy - 28 days
MTD
Primary Objectives:
To determine the maximum tolerated dose (MTD) of PF-114 in the target patient population.
Measured: 1-st Cycle of Therapy - 28 days
Secondary outcomes (12)
The incidence of AEs
To assess the safety and tolerability of PF-114 in the target patient population
Measured: through study completion, an average of 1 year
Cmax for oral PF-114 in the target patient population
Measured: 31 days
Tmax for oral PF-114 in the target patient population
Measured: 31 days
AUC0-t for oral PF-114 in the target patient population
Measured: 31 days
AUC0-∞ for oral PF-114 in the target patient population
Measured: 31 days
T1/2 for oral PF-114 in the target patient population
Measured: 31 days
CL/F for oral PF-114 in the target patient population
Measured: 31 days
Vd/F for single and multiple dosing for oral PF-114 in the target patient population
Measured: 31 days
Ctrough for multiple dosing for oral PF-114 in the target patient population
Measured: 31 days
Hematological response to the treatment based on European LeukemiaNet criteria, 2013.
Hematological response is evaluated on Day 1 of each therapy cycle
Full hematologic response:
Leukocytes < 10 х 109 /L Basophils < 5 % Thrombocytes < 450 х 109 /L No myelocytes, promyelocyts, myeloblasts in the differential Absence of splenomegaly - spleen non palpable
Measured: through study completion, an average of 1 year
Molecular response - the level of BCR-ABL transcripts in the peripheral blood, determined by the method of quantitative polymerase chain reaction (qPCR) using the international scale.
Molecular response is evaluated on Day 1 of Cycles 2, 4, 7, 10. For cycles > 12, the molecular response will be evaluated once in 3 months, where the procedure is carried out for the first time during Cycle 13.
Measured: through study completion, an average of 1 year
Cytogenetic response evaluated using the chromosome banding method (in situ (FISH) fluorescence hybridization is allowed only if the chromosome banding method cannot provide enough information).
Cytogenetic response is evaluated on Day 1, Cycles 4, 7, 13. Then if the level of BCR-ABL transcripts exceeds the level of 0.1% using the qPCR method using the international scale, cytogenetic response is evaluated no earlier than in 3 months after the previous cytogenetic analysis. After the complete cytogenetic response has been reached (CCyR), cytogenetic analysis will be carried out every 12 months.
Measured: through study completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Aug 2016 (estimated)
- Study startJul 2016
- Primary completionJun 2018
- Study completionMay 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Chronic Myeloid LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 65 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fusion Pharma LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- OCT LLC, Data Matrix Solutions, Skolkovo Innovation Center
- Responsible party
- Sponsor
- Investigators
- Anna Turkina, Professor, Principal investigator, Federal Haematological Scientific Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02885766
- Sponsor's protocol ID
- PF-114-01
- Status last verified
- Feb 2020
- First submitted
- 3 Aug 2016
- Last updated
- 17 Feb 2020
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 25394714Turkina 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.
PubMed 40298994
Study at a glance
From the registry record, last updated by the sponsor on 17 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT02885766
- Acronym / sponsor ID
- PF-114-01
- Keywords
- CML, Myeloid leukaemia chronic, CML progression, Chronic phase chronic myeloid leukemia, PF-114, Cytogenetic response CCyR, Major molecular response MMR, Complete molecular response CMR
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 65 participants (actual)
- Lead sponsor
- Fusion Pharma LLCIndustry (pharma, biotech, devices)
- Collaborators
- OCT LLC, Data Matrix Solutions, Skolkovo Innovation Center
- Study start
- Jul 2016 (actual)
- Primary completion
- Jun 2018 (actual)
- Study completion
- May 2020 (estimated)
- First posted
- 31 Aug 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
PF-114 is a low molecular inhibitor of a Bcr-Abl kinase activity, which is active with respect to native and mutated forms of this enzyme with mutations in Abl kinase domain. Preclinical in vitro and in vivo studies have demonstrated the ability of PF-114 to inhibit wild Bcr-Abl type and with T315I mutation, as well as other kinds of Bcr-Abl with mutations in kinase domain, including combined mutations.
In contrast to ponatinib, PF-114 is being developed to increase the action selectivity with respect to Bcr-Abl, which potentially should increase safety of drug application in people. The results of performed preclinical studies confirmed improved selectivity of PF-114 action with respect to Bcr-Abl kinases as compared to ponatinib.
Indication:
Adult patients with Ph+ CML in chronic phase (CP) or accelerated phase (AP) resistant to previous treatment with at least one 2-nd generation inhibitor of Bcr-Abl (dasatinib, nilotinib, bosutinib) or intolerant of approved Bcr-Abl inhibitors or with T315I mutation in the BCR-ABL gene
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PF-114 | Experimental | Drug: PF-114PF-114 From 50 mg up to the MTD. Dose escalation for each next cohort is conducted by increasing the dose by 20 % (or the closest lower level, which is a multiple of 25 mg) if there are Grade 3 ADRs according to NCI CTC AE v.4 without reaching а MTD. An increase of the dose by 40 % is applied if there were Grade 2 ADRs. In the absence of Grade 2 or 3 ADRs an increase of 100 % is applied. When the dose reaches 400 mg/day, the following increase in dose can be made after discussing results of safety findings of PF-114 between the Investigators and the Sponsor. Orally, once daily |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- Patients must meet all of the following criteria in order to be eligible for participation in the study:
- Able to give written informed consent;
- Male or female patient ≥ 18 years old;
- Confirmed diagnosis of CML in chronic or accelerated phase according to European LeukemiaNet guideline as of 2013;
- Available information regarding resistance to the therapy with least one 2-nd generation Bcr-Abl inhibitor (dasatinib or nilotinib or bosutinib), or intolerance of approved Bcr-Abl inhibitors, or presence of T315I mutation irrespective of treatment history;
Key exclusion criteria (15 listed)
- Patients must not meet any of the following criteria in order to be eligible for participation in the study:
- Use of the following previous therapy:
- Significant uncontrolled cardiac disease;
- Sustained uncontrolled hypertension ≥ Grade 2 (according to NCI CTC AE v4);
- Patient is taking medicinal products known to prolong the QT interval on the electrocardiogram, unless they are absolutely necessary in the opinion of the investigator;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PF-114 | Drug | PF-114 |
Primary outcome
DLTs during the first cycle of therapy
Time frame: 1-st Cycle of Therapy - 28 days
To study the dose-limiting toxicities (DLTs) of PF-114 mesylate in the target patient population during the 1-st cycle of treatment
MTD
Time frame: 1-st Cycle of Therapy - 28 days
Primary Objectives: To determine the maximum tolerated dose (MTD) of PF-114 in the target patient population.
12 secondary outcomes
The incidence of AEs
Time frame: through study completion, an average of 1 year
Cmax for oral PF-114 in the target patient population
Time frame: 31 days
Tmax for oral PF-114 in the target patient population
Time frame: 31 days
AUC0-t for oral PF-114 in the target patient population
Time frame: 31 days
AUC0-∞ for oral PF-114 in the target patient population
Time frame: 31 days
T1/2 for oral PF-114 in the target patient population
Time frame: 31 days
CL/F for oral PF-114 in the target patient population
Time frame: 31 days
Vd/F for single and multiple dosing for oral PF-114 in the target patient population
Time frame: 31 days
Ctrough for multiple dosing for oral PF-114 in the target patient population
Time frame: 31 days
Hematological response to the treatment based on European LeukemiaNet criteria, 2013.
Time frame: through study completion, an average of 1 year
Molecular response - the level of BCR-ABL transcripts in the peripheral blood, determined by the method of quantitative polymerase chain reaction (qPCR) using the international scale.
Time frame: through study completion, an average of 1 year
Cytogenetic response evaluated using the chromosome banding method (in situ (FISH) fluorescence hybridization is allowed only if the chromosome banding method cannot provide enough information).
Time frame: through study completion, an average of 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Feb 2020.
- Results publication: PubMed 25394714Mian 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.
- Derived from this study: PubMed 40298994Turkina 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.
- doi:10.1038/leu.2014.326Link provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Anna Turkina, Professor principal investigator
Federal Haematological Scientific Center
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing PF-114 in people with Chronic Myeloid Leukemia and Leukemia, Myelogenous, Chronic, BCR-ABL Positive, run by Fusion Pharma LLC at 3 sites in Russia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 18 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 17 Feb 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Russia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
DLTs during the first cycle of therapy (time frame: 1-st Cycle of Therapy - 28 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02885766, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Feb 2020.