Open-Label Study of Oral CEP-701 (Lestaurtinib) in Patients With Polycythemia Vera or Essential Thrombocytosis

Phase 2

Completed

• Conditions: Polycythemia Vera; Essential Thrombocytosis
• Interventions: Drug: lestaurtinib

• Registration Number: NCT00586651
• Lead Sponsor: Cephalon

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.

Study Timeline

• Study Start: 2007-12
• Study First Submitted: 2007-12-21
• Study First Submitted QC: 2007-12-21
• Study First Posted: 2008-01-04
• Primary Completion: 2009-10
• Study Completion: 2010-09
• Disposition First Submitted: 2013-08-22
• Disposition First Submitted QC: 2013-08-22
• Disposition First Posted: 2013-08-30
• Status Verified: 2015-09
• Last Update Submitted: 2015-09-22
• Last Update Posted: 2015-10-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 39

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
lestaurtinib	lestaurtinib	-

Primary Outcome Measures

Name	Time	Method
Determine whether a specific reduction in the JAK2 V617F allele has been indicated in this study.	18 weeks +

Secondary Outcome Measures

Name	Time	Method
- improvements in hemoglobin values, neutrophil count, and platelet count. - reduction in dose of hydroxyurea - reduction in splenic enlargement - rate of phlebotomy	18 weeks +

Trial Locations

Locations (4)

Mount Sinai School of Medicine; 🇺🇸 New York, New York, United States

University of Pennsylvania; 🇺🇸 Philadelphia, Pennsylvania, United States

Johns Hopkins University; 🇺🇸 Baltimore, Maryland, United States

NY Presbyterian-Cornell; 🇺🇸 New York City, New York, United States