CEP-701 (Lestaurtinib) in Myelofibrosis (NCT00668421) | Clinical Trial Compass
UnknownPhase 1/2
CEP-701 (Lestaurtinib) in Myelofibrosis
United States60 participantsStarted 2008-04
Plain-language 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.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Diagnosis of primary myelofibrosis, essential thrombocythemia related myelofibrosis, or polycythemia vera related myelofibrosis requiring therapy, including:
. those previously treated and relapsed or refractory
. or newly diagnosed, with intermediate or high risk according to Lille Scoring system (adverse prognostic factors are: Hb \< 10 g/dl, WBC \< 4 or \> 30 x 109/L; risk group: 0 = low, 1 = intermediate, 2 = high)
. or with symptomatic splenomegaly (must be \>=10 cm below the left costal margin in the mid-clavicular line).
. The subject must not be considered as a candidate to receive allogeneic hematopoietic stem cell transplant at the time of being enrolled into the study.
. The subject has a detectable JAK2 V617F mutation.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
To determine the safety and maximum tolerated dose of a novel kinase inhibitor in subjects with myelofibrosis.
Timeframe: 2 years
2
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.
. Signed informed consent: Patients must have signed consents for both the Lestaurtinib protocol and for the mandatory biomarker MDP-RC 107 protocol to be eligible to participate.
. Patients must have been off any PMF-directed therapy for 4 weeks prior to entering this study and have recovered from the toxic effects (grade 0-1) of that therapy. Treatment with erythropoietin is permitted.
Exclusion criteria
. Nursing and pregnant females. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
. New York Heart Association (NYHA) Grade II or greater congestive heart failure.
. Unstable angina.
. Major surgical procedure, open biopsy, or significant traumatic injury within 28 days, or anticipation of the need for major surgical procedure during the course of the study.
. Biopsy or other minor surgical procedure, excluding placement of a vascular access device or bone marrow biopsy, within 7 days prior to study enrollment.
. Ongoing serious, non-healing wound, ulcer, or bone fracture.
. Known hypersensitivity to any component of Lestaurtinib.
. The subject has received a donor stem cell transplant in the past and has detectable full or partial donor chimerism.