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

Chronic Myeloid Leukemia Clinical Trial

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

Status Active, not recruiting

Clinical 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 inhibitors or has T315I mutation in the BCR-ABL gene.

Clinical Trial 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

Related Conditions & MeSH terms

Chronic Myeloid Leukemia
Leukemia
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Leukemia, Myeloid

Clinical Trial Details

NCT number	NCT02885766
Study type	Interventional
Source	Fusion Pharma LLC
Contact
Status	Active, not recruiting
Phase	Phase 1/Phase 2
Start date	July 2016
Completion date	May 2020

View Details

See also
Status	Clinical Trial	Phase
Recruiting	`NCT05400122` - Natural Killer (NK) Cells in Combination With Interleukin-2 (IL-2) and Transforming Growth Factor Beta (TGFbeta) Receptor I Inhibitor Vactosertib in Cancer	Phase 1
Completed	`NCT02057185` - Occupational Status and Hematological Disease
Recruiting	`NCT03326310` - Selumetinib and Azacitidine in High Risk Chronic Blood Cancers	Phase 1
Recruiting	`NCT04621851` - Retro-prospective Observational Study on Risk of Progression in CP-CML Patients Eligible for TKI Discontinuation
Completed	`NCT01207440` - Ponatinib for Chronic Myeloid Leukemia (CML) Evaluation and Ph+ Acute Lymphoblastic Leukemia (ALL)	Phase 2
Not yet recruiting	`NCT06409936` - PEARL Study: PotEntial of Asciminib in the eaRly Treatment of CML	Phase 2
Active, not recruiting	`NCT02917720` - 2nd or 3rd TKI-stop After 2 Years Nilotinib Pre-treatment in CML-patients	Phase 2
Not yet recruiting	`NCT02883036` - Vitro Study of Tigecycline to Treat Chronic Myeloid Leukemia	N/A
Withdrawn	`NCT01188889` - RAD001 in Patients With Chronic Phase Chronic Myeloid Leukemia w/ Molecular Disease.	Phase 1/Phase 2
Completed	`NCT01795716` - Bioequivalence Study of Mesylate Imatinib Capsule in Chronic Myeloid Leukemia Body	Phase 1
Completed	`NCT00988013` - Intensity Modulated Total Marrow Irradiation (IM-TMI) for Advanced Hematologic Malignancies	N/A
Approved for marketing	`NCT00905593` - Nilotinib in Adult Patients With Imatinib-resistant or Intolerant Chronic Myeloid Leukemia in Blast Crisis, Accelerated Phase or Chronic Phase	Phase 3
Terminated	`NCT00573378` - Imatinib or Nilotinib With Pegylated Interferon-α2b in Chronic Myeloid Leukemia	Phase 2
Completed	`NCT00469014` - Busulfan, Fludarabine, Clofarabine With Allogeneic Stem Cell Transplantation for Acute Myeloid Leukemia	Phase 2
Terminated	`NCT00522990` - Study to Assess the Safety of Escalating Doses of AT9283, in Patients With Leukemias	Phase 1/Phase 2
Completed	`NCT00257647` - Use of SV40 Vectors to Treat Chronic Myeloid Leukemia (CML)	N/A
Unknown status	`NCT00598624` - Clinical Trial to Evaluate the Safety and Efficacy of Treosulfan Based Conditioning Prior to Allogeneic Haematopoietic Stem Cell Transplantation (HSCT)	Phase 2
Completed	`NCT00219739` - STI571 ProspectIve RandomIzed Trial: SPIRIT	Phase 3
Completed	`NCT06148493` - Real-World Usage of Asciminib Among Patients With Chronic Myeloid Leukemia in Chronic Phase in the United States Using a Large Claims Database
Completed	`NCT00375219` - Homoharringtonine (Omacetaxine Mepesuccinate) in Treating Patients With Chronic Myeloid Leukemia (CML) With the T315I BCR-ABL Gene Mutation	Phase 2

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.