Clinical trial NCT00586651: Open-Label Study of Oral CEP-701 (Lestaurtinib) in Patients With Polycythemia Vera or Essential Thrombocytosis
Official title: An Open-Label Study of Oral CEP-701 in Patients With Polycythemia Vera or Essential Thrombocytosis With the JAK2 V617F Mutation
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Polycythemia Vera
Essential Thrombocytosis
Lead sponsor: Teva Branded Pharmaceutical Products R&D, Inc.
Sponsor class: INDUSTRY
First posted: 2008-01-04
Enrollment: 39
Sex: ALL
Minimum age (years): 18
Maximum age (years): 99
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is an 18-week open-label, multicenter study to evaluate the efficacy and tolerability of CEP-701 (lestaurtinib) treatment in patients with Polycythemia Vera (PV) and patients with Essential Thrombocytosis (ET).

Detailed description: This is an 18-week open-label, multicenter study to evaluate the efficacy and tolerability of CEP-701 (lestaurtinib) treatment at a dosage of 80 mg bid for 18 weeks (126 days) in patients with Polycythemia Vera (PV) who have abnormal baseline neutrophil counts or require hydroxyurea therapy and patients with Essential Thrombocytosis (ET) who require hydroxyurea therapy for disease control.

Eligibility criteria: Inclusion Criteria:

* The patient has polycythemia vera (PV) or essential thrombocytosis (ET).
* The patient has a detectable JAK2 V617F mutation.
* Patients with PV have at least 1 of the following risk factors:

  1. neutrophil count greater than 7000/mm3
  2. receiving hydroxyurea treatment
* Patients with ET are receiving concomitant hydroxyurea.
* The patient has an ECOG performance score of 0, 1, or 2.

Exclusion Criteria:

* The patient has bilirubin levels or aspartate transaminases (AST) levels within exclusionary ranges.
* patient has serum creatinine concentrations within exclusionary ranges.
* patient has an untreated or progressive infection.
* patient has any physical or psychiatric condition that may compromise participation in the study.
* has a history of venous or arterial thrombosis within 6 months.
* use of hydroxyurea has been initiated or escalated in the month prior to screening.
* has active gastrointestinal ulceration or bleeding.
* patient has used an investigational drug within the past 30 days.
* patient is being treated with anagrelide.
* patient has previously taken CEP-701 (lestaurtinib).
* patient has hypersensitivity to CEP-701 (lestaurtinib) or any component of CEP-701 (lestaurtinib).
* patient has received interferon within the past 30 days.

Primary outcomes:
measure: Determine whether a specific reduction in the JAK2 V617F allele has been indicated in this study.
timeFrame: 18 weeks +
Secondary outcomes:
measure: - improvements in hemoglobin values, neutrophil count, and platelet count. - reduction in dose of hydroxyurea - reduction in splenic enlargement - rate of phlebotomy
timeFrame: 18 weeks +
Other outcomes:
Locations:
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: New York City
country: United States
facility: NY Presbyterian-Cornell
state: New York
city: New York
country: United States
facility: Mount Sinai School of Medicine
state: New York
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
Study officials:
References:
citation: Hexner E, Roboz G, Hoffman R, Luger S, Mascarenhas J, Carroll M, Clementi R, Bensen-Kennedy D, Moliterno A. Open-label study of oral CEP-701 (lestaurtinib) in patients with polycythaemia vera or essential thrombocythaemia with JAK2-V617F mutation. Br J Haematol. 2014 Jan;164(1):83-93. doi: 10.1111/bjh.12607. Epub 2013 Oct 28.