LeukemiaPhase 2NCT00494585
CEP-701 for PH-negative Myelofibrosis
A Phase 2 study testing CEP-701 in people with Leukemia and Myelofibrosis, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
The goal of this clinical research study is to find out if CEP-701 can help control myelofibrosis (MF). The safety of CEP-701 will also be studied. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leukemia, Myelofibrosis
- What is being tested?
- CEP-701
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2007to May 2010
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with Chronic Idiopathic Myelofibrosis (CIMF) requiring therapy, including those 1) previously treated by CIMF-directed therapy and relapsed, intolerant, or refractory to therapy; or 2) if newly diagnosed then with intermediate or high risk according to Lille scoring system (adverse prognostic factors are: Hb < 10 g/dl, WBC < 4 or > 30 x 10^9/L; risk group: 0 = low, 1 = intermediate, 2 = high), or with symptomatic spleen that is >/= 10cm below costal margin. However, patients with asymptomatic intermediate risk disease are not eligible.
- JAK2 mutation positive test
- Age of at least 18 years
- Eastern Cooperative Oncology Group (ECOG) performance status 0-2
- Adequate liver and renal function: total bilirubin </=2.0 mg/dL, alanine aminotransferase (ALT or SGPT) </=2.0 x institutional upper limit of normal (ULN), and creatinine </=2.0 mg/dl
- Patients must be at least 2 weeks from prior chemotherapy, biological therapy, radiation therapy, major surgery, or other investigational anticancer therapy that is considered MF-directed, and have recovered from prior toxicities to Grade 0-1. Concurrent therapy with supportive care medications (hydroxyurea, anagrelide) is allowed during the study.
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- All men of reproductive potential and women of child-bearing potential (WOCBP) must agree to practice effective contraception (iud, birth control pill, latex condoms, diaphragm) during the entire study period and for one month after the study ends, unless documentation of infertility exists. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately. WOCBP are women who are not menopausal for 12 months or no previous surgical sterilization."
- Ability to understand and willingness to sign the informed consent form
- Not willing to undergo, not a candidate for, or not having a donor for, a bone marrow transplant
Who cannot join
- Pregnant or nursing women, due to the unknown effects of therapy on the developing fetus or newborn infant.
- Patients diagnosed with another malignancy - unless following curative intent therapy the patient has been disease free for at least 3 years. Patients with early stage squamous cell carcinoma of the skin, basal cell carcinoma of the skin, or cervical intraepithelial neoplasia (CIN) are eligible for this study
- Any condition, including serious medical condition, laboratory abnormality, or psychiatric illness, which places the subject at unacceptable risk as judged by the Principal Investigator, if he/she was to participate in the study
- Known positive for Human immunodeficiency virus (HIV) or infectious hepatitis, type A, B or C
- Presence of any gastrointestinal condition or concomitant medication use (e.g. coumadin) that would render a patient at high risk for gastrointestinal bleeding as judged by treating physician
- History of any upper or lower gastrointestinal bleeding in the 6 months prior to enrollment
- Elevated international normalized ratio (INR) or Partial thromboplastin time (PTT)
Trial locations and contacts
1 site in 1 country.
United States1 site
The University of Texas M.D. Anderson Cancer Center
Houston, Texas, 77030
About this study
Official title: Evaluation of CEP-701, an Orally Available JAK2 Tyrosine Kinase Inhibitor, as a Therapy for Patients With Myelofibrosis
The goal of this clinical research study is to find out if CEP-701 can help control myelofibrosis (MF). The safety of CEP-701 will also be studied.
Read the detailed description
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.
Study Treatment:
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.
Study Visits:
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.
- Conditions
- Leukemia, Myelofibrosis
- Being tested
- CEP-701
- Keywords
- CEP-701, Leukemia, Myelofibrosis, MF, JAK2, Tyrosine Kinase Inhibitor, Lestaurtinib, Chronic Idiopathic Myelofibrosis, CIMF
- Medical subject headings
- Leukemia, Primary Myelofibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CEP-701Drug
Also called lestaurtinib
80 mg orally twice a day for 30 days
| Study group | Role | What they receive |
|---|---|---|
| CEP-701 | Experimental | 80 mg orally twice a day for 30 days Receives: Drug: CEP-701 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Objective Response
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]
Measured: Response assessed after each 3 cycles (cycle = 30 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jun 2007 (estimated)
- Study startJun 2007
- Primary completionMay 2010
- Study completionMay 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for LeukemiaStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Cephalon
- Responsible party
- Sponsor
- Investigators
- Srdan Verstovsek, M.D., Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00494585
- Sponsor's protocol ID
- 2007-0070
- Other IDs
- CS-2007-20040
- Status last verified
- Jun 2012
- First submitted
- 28 Jun 2007
- Last updated
- 25 Jun 2012
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Santos FP, Kantarjian HM, Jain N, Manshouri T, Thomas DA, Garcia-Manero G, Kennedy D, Estrov Z, Cortes J, Verstovsek S. Phase 2 study of CEP-701, an orally available JAK2 inhibitor, in patients with primary or post-polycythemia vera/essential thrombocythemia myelofibrosis. Blood. 2010 Feb 11;115(6):1131-6. doi: 10.1182/blood-2009-10-246363. Epub 2009 Dec 11.
PubMed 20008298
Study at a glance
From the registry record, last updated by the sponsor on 25 Jun 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of CEP-701, an Orally Available JAK2 Tyrosine Kinase Inhibitor, as a Therapy for Patients With Myelofibrosis
- ClinicalTrials.gov ID
- NCT00494585
- Acronym / sponsor ID
- 2007-0070
- Conditions
- Leukemia, Myelofibrosis
- Keywords
- CEP-701, Leukemia, Myelofibrosis, MF, JAK2, Tyrosine Kinase Inhibitor, Lestaurtinib, Chronic Idiopathic Myelofibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- Cephalon
- Study start
- Jun 2007
- Primary completion
- May 2010 (actual)
- Study completion
- May 2010 (actual)
- First posted
- 29 Jun 2007 (estimated)
- Results
- Posted 4 May 2011
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical research study is to find out if CEP-701 can help control myelofibrosis (MF). The safety of CEP-701 will also be studied.
Detailed description
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.
Study Treatment:
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.
Study Visits:
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CEP-701 | Experimental | Drug: CEP-70180 mg orally twice a day for 30 days |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Patients with Chronic Idiopathic Myelofibrosis (CIMF) requiring therapy, including those 1) previously treated by CIMF-directed therapy and relapsed, intolerant, or refractory to therapy; or 2) if newly diagnosed then with intermediate or high risk according to Lille scoring system (adverse prognostic factors are: Hb < 10 g/dl, WBC < 4 or > 30 x 10^9/L; risk group: 0 = low, 1 = intermediate, 2 = high), or with symptomatic spleen that is >/= 10cm below costal margin. However, patients with asymptomatic intermediate risk disease are not eligible.
- JAK2 mutation positive test
- Age of at least 18 years
- Eastern Cooperative Oncology Group (ECOG) performance status 0-2
- Adequate liver and renal function: total bilirubin </=2.0 mg/dL, alanine aminotransferase (ALT or SGPT) </=2.0 x institutional upper limit of normal (ULN), and creatinine </=2.0 mg/dl
Key exclusion criteria (7 listed)
- Pregnant or nursing women, due to the unknown effects of therapy on the developing fetus or newborn infant.
- Patients diagnosed with another malignancy - unless following curative intent therapy the patient has been disease free for at least 3 years. Patients with early stage squamous cell carcinoma of the skin, basal cell carcinoma of the skin, or cervical intraepithelial neoplasia (CIN) are eligible for this study
- Any condition, including serious medical condition, laboratory abnormality, or psychiatric illness, which places the subject at unacceptable risk as judged by the Principal Investigator, if he/she was to participate in the study
- Known positive for Human immunodeficiency virus (HIV) or infectious hepatitis, type A, B or C
- Presence of any gastrointestinal condition or concomitant medication use (e.g. coumadin) that would render a patient at high risk for gastrointestinal bleeding as judged by treating physician
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CEP-701Also: lestaurtinib80 mg orally twice a day for 30 days | Drug | CEP-701 |
Primary outcome
Number of Participants With Objective Response
Time frame: Response assessed after each 3 cycles (cycle = 30 days)
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]
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jun 2012.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 20008298Santos FP, Kantarjian HM, Jain N, Manshouri T, Thomas DA, Garcia-Manero G, Kennedy D, Estrov Z, Cortes J, Verstovsek S. Phase 2 study of CEP-701, an orally available JAK2 inhibitor, in patients with primary or post-polycythemia vera/essential thrombocythemia myelofibrosis. Blood. 2010 Feb 11;115(6):1131-6. doi: 10.1182/blood-2009-10-246363. Epub 2009 Dec 11.
- M.D. Anderson's websiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Srdan Verstovsek, M.D. principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing CEP-701 in people with Leukemia and Myelofibrosis, run by M.D. Anderson Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Jun 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 4 May 2011). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Objective Response (time frame: Response assessed after each 3 cycles (cycle = 30 days)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00494585, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Jun 2012.