Trial of Erlotinib in Patients With JAK-2 V617F Positive Polycythemia Vera

Phase 2

Terminated

• Conditions: Polycythemia Vera
• Interventions: Drug: Erlotinib

• Registration Number: NCT01038856
• Lead Sponsor: University of Oklahoma

Brief Summary

The primary objective of this study is to determine the overall response rate to erlotinib in patients with polycythemia vera (PV). Response rate will be assessed by improvement in the complete blood count, ultrasound of the spleen, and JAK2 molecular status. It is purposed in this study to explore a possible molecular targeting of the driving mechanism of PV.

Detailed Description

This is a phase II open-label study. Patients will be screened for MPN diagnoses and patients with Polycythemia vera proven to have JAK2V617F mutation will be given the option to enroll. Consenting patients will take erlotinib daily for 16 weeks. Blood work and pharmacokinetics will be drawn for serum level monitoring. Doses will be administered according to side effects or held. First assessment will be at day 15 wth subsequent assessments at 28 day intervals. Non-responders will be taken off the study and managed according to standard of care. Patients who do respond will continue taking the therapy for a total of 12 months. Observation will be for a total of 12 months after finishing treatment. In addition to the clinical aspect of this study, there will be correlative studies where molecular response will be checked and its correlation with clinical response.

Study Timeline

• Study Start: 2009-12
• Study First Submitted: 2009-12-22
• Study First Submitted QC: 2009-12-22
• Study First Posted: 2009-12-24
• Primary Completion: 2012-12
• Study Completion: 2014-02
• Results First Submitted: 2015-05-28
• Results First Submitted QC: 2018-01-10
• Results First Posted: 2018-02-07
• Status Verified: 2020-08
• Last Update Submitted: 2020-08-07
• Last Update Posted: 2020-08-24

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 5

Inclusion Criteria

• WHO 2008 diagnosis of Polycythemia Vera Hemoglobin > 18.5 g/dl for men (16.5 g/dl for women) and presence of JAK2V617F mutation and either bone marrow trilineage myeloproliferation or subnormal serum erythropoietin level Patients may be on active treatment (phlebotomy, aspirin) ECOG performance status 0,1,2,or 3 Adequate hepatic function, adequate renal function

Exclusion Criteria

• Patient with active malignancy Patients with clinically significant cardiac disease within 1 year Opthalmologic or gastrointestinal abnormalities Concurrent cytoreductive therapy is not allowed

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
+JAK2V61F mutation	Erlotinib	Patients with MPN diagnoses and polycythemia vera who also have a confirmed JAK2V617F mutation

Primary Outcome Measures

Name	Time	Method
Overall Response Rate to Include Complete Hematological Response, Complete Molecular Response, Partial Hematological Response, and Minimal Hematological Response	Day 15

Secondary Outcome Measures

Name	Time	Method
Improvement in Splenomegaly Size	4 months, end of treatment and 12 months end of treatment
Incidence of Toxicities	First assessment at day 15, subsequent assessments at 28 day intervals for an average of 1 year	Grade 3 or grade 4 toxicities as measured by CTCAE v3.0
Decrease of Mutant JAK2V617F Allele Burden	every 2 months until end of treatment and 12 months after end of treatment

Trial Locations

Locations (1)

University of Oklahoma Health Sciences Center; 🇺🇸 Oklahoma City, Oklahoma, United States