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Tyrosine Kinase Inhibitor

CEP-701 (Lestaurtinib) for Polycythemia Vera

Phase 1 & 2
Waitlist Available
Led By Ronald Hoffman, MD
Research Sponsored by Ronald Hoffman
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 2 years
Awards & highlights

Study Summary

Myelofibrosis is the gradual replacement of bone marrow (place where most new blood cells are produced) by fibrous tissue which reduces the body's ability to produce new blood cells and results in the development of chronic anemia (low red blood cell count). One of the main distinctions of myelofibrosis is "extramedullary hematopoesis", the migration or traveling of the blood-forming cells out of the bones to other parts of the body, such as the liver or spleen, resulting in an enlarged spleen and liver. Treatment for myelofibrosis is unsatisfactory and there is no medication that is specifically used in the treatment of myelofibrosis. There is a protein that is found to be present in the majority of myelofibrosis patients (JAK2) and the drug Lestaurtinib is being studied to see if it will stop this protein from functioning and thereby help control the disease. This study is divided into two Phases (1 & 2). In phase 1 we will be looking for the dose of study medication (Lestaurtinib) that will be the highest dose a patient can take without experiencing serious side effects, maximum tolerated dose (MTD). In phase 2, after the MTD dose has been established in phase 1, we will be investigating how well CEP-701 (Lestaurtinib) works at suppressing the protein (JAK2). The investigators also wish to find out important biologic characteristics or features of myelofibrosis through an additional correlative biomarker study (MPD-RC #107). The correlative biomarker study is a study that is related to the main study, but is looking to answer different questions than the main study. The purpose of the biomarker study is to understand the causes of MPD and to develop improved methods for the diagnosis and treatment of these diseases, while the main study is trying to find out how well CEP-701 (Lestaurtinib) will work in treating the myeloproliferative disease.

Eligible Conditions
  • Polycythemia Vera
  • Essential Thrombocythemia
  • Myelofibrosis

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
To determine the safety and maximum tolerated dose of a novel kinase inhibitor in subjects with myelofibrosis.
To estimate the efficacy of a novel kinase inhibitor in subjects with myelofibrosis, as determined by a reduction in JAK2 V617F allele frequency in peripheral blood neutrophils.
Secondary outcome measures
To estimate the incidence, severity, and attribution of treatment-emergent adverse events.
To estimate the rate of complete or major clinical-hematological response from treatment with Lestaurtinib (CEP-701) in this subject population as measured by the EUMNET response criteria.

Trial Design

1Treatment groups
Experimental Treatment
Group I: CEP-701 (Lestaurtinib)Experimental Treatment1 Intervention
Subject is to receives Lestaurtinib, in Phase 1: standard cohort dose escalation; Phase 2: single stage design to estimate the percentage of subjects with a 15% or greater reduction in JAK2 V617F allele frequency in peripheral blood granulocytes in 6 months of treatment

Find a Location

Who is running the clinical trial?

Ronald HoffmanLead Sponsor
5 Previous Clinical Trials
338 Total Patients Enrolled
4 Trials studying Polycythemia Vera
325 Patients Enrolled for Polycythemia Vera
Myeloproliferative Disorders-Research ConsortiumNETWORK
11 Previous Clinical Trials
2,750 Total Patients Enrolled
8 Trials studying Polycythemia Vera
2,622 Patients Enrolled for Polycythemia Vera
National Cancer Institute (NCI)NIH
13,663 Previous Clinical Trials
40,925,845 Total Patients Enrolled
39 Trials studying Polycythemia Vera
4,113 Patients Enrolled for Polycythemia Vera

Frequently Asked Questions

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~4 spots leftby Apr 2025