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MyelofibrosisPhase 1/2NCT00668421

CEP-701 (Lestaurtinib) in Myelofibrosis

A Phase 1/2 study testing CEP-701 (Lestaurtinib) in people with Myelofibrosis and Essential Thrombocythemia, run by Ronald Hoffman at 7 sites in United States.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
CEP-701 (Lestaurtinib)
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
60 participants
Where
7 sitesUnited States
Study dates
Apr 2008to Jan 2015
Phase
Phase 1/2
Are there results?
None posted in the registry

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

  • 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.
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  • 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.
  • Serum bilirubin levels less than or equal to 2 times the upper limit of the normal range for the laboratory (ULN). Higher levels are acceptable if these can be attributed by treating physician to active hemolysis or ineffective erythropoiesis due to myelofibrosis.
  • Serum glutamic-pyruvic transaminase (SGPT) (alanine aminotransferase [ALT]) levels less than or equal to 2 x ULN.
  • Serum creatinine levels less than or equal to 1.5 x ULN.
  • Women of childbearing potential must have a negative serum or urine pregnancy test prior to Lestaurtinib treatment and should be advised to avoid becoming pregnant. Men must be advised to not father a child while receiving treatment with Lestaurtinib. Both women of childbearing potential and men must practice effective methods of contraception (those generally accepted as standard of care measures). Women of child bearing potential are women who are not menopausal for 12 months or who have not undergone previous surgical sterilization. If the subject is a woman of childbearing potential, she must use a medically acceptable form of contraception during the study period and for 30 days thereafter. If the subject is a man he must be surgically sterile or must use a medically approved method of contraception for the duration of the study and for 60 days following the last dose of CEP-701.
  • Age > 18 years.

Who cannot join

  • 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.
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  • 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.
  • The subject requires treatment with a CYP3A4 inhibitor, including azole antifungals (topicals are permitted); protease inhibitors; nefazodone; cyclosporine; erythromycin; clarithromycin; and troleandomycin.

Trial locations and contacts

7 sites in 1 country.

7 sites in 1 country

United States7 sites

  • University of Maryland Marlene and Stewart Greenebaum Cancer Center

    Baltimore, Maryland, 21201

  • Weill Cornell

    Ithaca, New York, 14851

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029

  • Palo Alto Medical Facilities

    Palo Alto, California, 94301

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19107

  • University of Utah

    Salt Lake City, Utah, 84102

  • Georgetown University Medicine Center

    Washington D.C., District of Columbia, 20007

Other trials in United States

About this study

Official title: A Multicenter, Open Label Phase I/II Study of CEP-701 (Lestaurtinib) in Adults With Myelofibrosis

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.

Read more

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.

Being tested
CEP-701 (Lestaurtinib)
Keywords
Myelofibrosis, Essential Thrombocythemia, Polycythemia Vera, JAK2 V617F Mutation, Lestaurtinib
Medical subject headings
Primary Myelofibrosis, Thrombocythemia, Essential, Polycythemia Vera

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CEP-701 (Lestaurtinib)Drug

    Lestaurtinib (CEP-701), oral formulation. Phase 1: 80 BID - 160 BID; phase 2: 140 mg

Study groups in NCT00668421
Study groupRoleWhat they receive
CEP-701 (Lestaurtinib)ExperimentalSubject 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
Receives: Drug: CEP-701 (Lestaurtinib)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. To determine the safety and maximum tolerated dose of a novel kinase inhibitor in subjects with myelofibrosis.

    Measured: 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.

    Measured: 2 years

Secondary outcomes (2)

  1. To estimate the incidence, severity, and attribution of treatment-emergent adverse events.

    Measured: 2 years

  2. 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.

    Measured: 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted29 Apr 2008 (estimated)
  2. Study startApr 2008
  3. Primary completionSep 2013
  4. Study completionJan 2015 (estimated)

Resources

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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
60 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00668421
Sponsor's protocol ID
GCO 07-0548-00104
Other IDs
P01CA108671-01A2, MPD-RC 104
Status last verified
Nov 2014
First submitted
25 Apr 2008
Last updated
26 Nov 2014
Results posted
No
Data monitoring committee
Yes

Publications

  • Hexner EO, Mascarenhas J, Prchal J, Roboz GJ, Baer MR, Ritchie EK, Leibowitz D, Demakos EP, Miller C, Siuty J, Kleczko J, Price L, Jeschke G, Weinberg R, Basu T, Pahl HL, Orazi A, Najfeld V, Marchioli R, Goldberg JD, Silverman LR, Hoffman R. Phase I dose escalation study of lestaurtinib in patients with myelofibrosis. Leuk Lymphoma. 2015;56(9):2543-51. doi: 10.3109/10428194.2014.1001986. Epub 2015 Feb 20.

    PubMed 25563429

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing CEP-701 (Lestaurtinib) in people with Myelofibrosis and Essential Thrombocythemia, run by Ronald Hoffman at 7 sites 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 13 inclusion and 9 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: status not confirmed, as of the sponsor's last update on 26 Nov 2014. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 sites 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

Results are not available in the source record.

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?

To determine the safety and maximum tolerated dose of a novel kinase inhibitor in subjects with myelofibrosis. (time frame: 2 years). The study lists 2 primary outcomes in all. 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 NCT00668421, 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 26 Nov 2014.

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