CEP-701 for PH-negative Myelofibrosis
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Evaluation of CEP-701, an Orally Available JAK2 Tyrosine Kinase Inhibitor, as a Therapy for Patients With Myelofibrosis|
- Number of Participants With Objective Response [ Time Frame: Response assessed after each 3 cycles (cycle = 30 days) ] [ Designated as safety issue: No ]Objective response = Complete Response, absence sign/symptoms of disease (without use of growth factors, hydroxyurea, anagrelide, or transfusions for > 1 month); Partial Response, absence of progressive disease (PD), and improvement in 2+ parameters (if abnormal): Absolute neutrophil count (ANC), hemoglobin, platelets, transfusions, splenomegaly, or bone marrow blasts; Clinical Improvement, absence of PD, and improvement in 1 parameter: ANC, hemoglobin, platelets, transfusions, splenomegaly, or bone marrow blasts). [International Working Group on Myelofibrosis Research and Treatment]
|Study Start Date:||June 2007|
|Study Completion Date:||May 2010|
|Primary Completion Date:||May 2010 (Final data collection date for primary outcome measure)|
80 mg orally twice a day for 30 days
80 mg orally twice a day for 30 days
Other Name: lestaurtinib
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The Study Drug:
CEP-701 is designed to help prevent a certain type of molecule (called a mutated JAK2 receptor) that is found on myelofibrosis cells from sending continuous chemical signals that lead to the growth of cancer cells.
If you are found to be eligible to take part in this study, you will take CEP-701 by mouth (in liquid form) 2 times a day (once in the morning and once in the evening) every day in 30-day repeating cycles. You should take each dose about 12 hours apart.
The study doctor or nurse will teach you and/or a caregiver or family member how to prepare each dose of the study drug, as well as how much should be taken each time. At each study visit, you will be supplied with enough syringes, dosing cups, and study drug to last until your next study visit. For each dose, you will use the syringe to draw the proper amount of CEP-701. You will add the entire contents of the syringe to an approved juice in 1 of the provided dosing cups. You should also drink an additional dosing cup of juice after taking the drug dose. The following juices (100% juice only) are approved for use with CEP-701: grape, pineapple, apple, V8 vegetable juice, and orange juice.
The study drug mixture may be stored (in an areas that are protected from light, such as in a cabinet) for up to 1 hour at room temperature and up to 8 hours refrigerated (at about 35°F to 45 °F). If you miss a dose, you should not take another dose until your next scheduled dose.
You will initially have study visits at M. D. Anderson once a month. You will need to return monthly for 6 months, then every 3 months if there are no side effects during the previous 3 cycles. After 2 years of therapy, your visits can be extended to every 6 months. After 6 cycles you may have either a study visit or a phone call from a member of study staff. If you have a phone call, you will be asked how you are feeling, if you have experienced any side effects since your last visit, and your blood tests will be reviewed with you. During most study visits, you will have the following tests:
- You will have a physical exam.
- You will be asked how you are feeling and about any side effects you may have experienced since your last visit.
- You will have blood (about 2 tablespoons) drawn to check your kidney and liver function and blood cell count. This will be done every 2 weeks up to 3 months, then every 1-2 months.
- You will have a bone marrow/aspirate once every 3-6 months to see if you are responding to treatment. After 2 years of therapy, this can be extended to every 12 months.
Length of Study:
You will continue on this study for at least 6 months to allow time for response. If you do respond to study treatment, you may continue to receive cycles for up to 5 years.
If you do not respond to study treatment within 6 months, if the disease gets worse, if intolerable side effects occur, if you have an illness that keeps you from taking the study drug, or your doctor thinks it is in your best interest to stop taking part in this study, you will be taken off this study.
This is an investigational study. CEP-701 is not Food and Drug Administration (FDA) approved or commercially available. At this time, it is being used for research purposes only in this study. Up to 41 patients will take part in this study. All will be enrolled at M. D. Anderson.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00494585
|United States, Texas|
|The University of Texas M.D. Anderson Cancer Center|
|Houston, Texas, United States, 77030|
|Principal Investigator:||Srdan Verstovsek, M.D.||M.D. Anderson Cancer Center|