CEP-701 (Lestaurtinib) in Myelofibrosis
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|ClinicalTrials.gov Identifier: NCT00668421|
Recruitment Status : Unknown
Verified November 2014 by Ronald Hoffman, Icahn School of Medicine at Mount Sinai.
Recruitment status was: Active, not recruiting
First Posted : April 29, 2008
Last Update Posted : November 26, 2014
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.
|Condition or disease||Intervention/treatment||Phase|
|Myelofibrosis Essential Thrombocythemia Polycythemia Vera||Drug: CEP-701 (Lestaurtinib)||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||60 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Multicenter, Open Label Phase I/II Study of CEP-701 (Lestaurtinib) in Adults With Myelofibrosis|
|Study Start Date :||April 2008|
|Actual Primary Completion Date :||September 2013|
|Estimated Study Completion Date :||January 2015|
Experimental: CEP-701 (Lestaurtinib)
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
Drug: CEP-701 (Lestaurtinib)
Lestaurtinib (CEP-701), oral formulation. Phase 1: 80 BID - 160 BID; phase 2: 140 mg
- To determine the safety and maximum tolerated dose of a novel kinase inhibitor in subjects with myelofibrosis. [ Time Frame: 2 years ]
- 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. [ Time Frame: 2 years ]
- To estimate the incidence, severity, and attribution of treatment-emergent adverse events. [ Time Frame: 2 years ]
- 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. [ Time Frame: 2 years ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00668421
|United States, California|
|Palo Alto Medical Facilities|
|Palo Alto, California, United States, 94301|
|United States, District of Columbia|
|Georgetown University Medicine Center|
|Washington, District of Columbia, United States, 20007|
|United States, Maryland|
|University of Maryland Marlene and Stewart Greenebaum Cancer Center|
|Baltimore, Maryland, United States, 21201|
|United States, New York|
|Ithaca, New York, United States, 14851|
|Icahn School of Medicine at Mount Sinai|
|New York, New York, United States, 10029|
|United States, Pennsylvania|
|University of Pennsylvania|
|Philadelphia, Pennsylvania, United States, 19107|
|United States, Utah|
|University of Utah|
|Salt Lake City, Utah, United States, 84102|
|Principal Investigator:||Ronald Hoffman, MD||Icahn School of Medicine at Mount Sinai|