Trial of Erlotinib in Patients With JAK-2 V617F Positive Polycythemia Vera (OSI-TAR-766)

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

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.

Study Overview

• Status: Terminated
• Conditions: Polycythemia Vera
• Intervention / Treatment: Drug: Erlotinib

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 Type: Interventional
• Enrollment (Actual): 5
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73104
      • University of Oklahoma Health Sciences Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 99 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: +JAK2V61F mutation; Patients with MPN diagnoses and polycythemia vera who also have a confirmed JAK2V617F mutation	Drug: Erlotinib; Erlotinib supplied as tablets; oral dose of erlotinib of 150 mg daily to be continued for 16 weeks. Responders will continue for up to 12 months, non-responders will cease taking erlotinib; Other Names: Tarceva; OSI-744

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Overall Response Rate to Include Complete Hematological Response, Complete Molecular Response, Partial Hematological Response, and Minimal Hematological Response	Day 15

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: University of Oklahoma
• Collaborators: OSI Pharmaceuticals
• Investigators: Principal Investigator: Mohamad Cherry, MD, University of Oklahoma

Study record dates

Study Major Dates

• Study Start: December 1, 2009
• Primary Completion (Actual): December 1, 2012
• Study Completion (Actual): February 1, 2014

Study Registration Dates

• First Submitted: December 22, 2009
• First Submitted That Met QC Criteria: December 22, 2009
• First Posted (Estimate): December 24, 2009

Study Record Updates

• Last Update Posted (Actual): August 24, 2020
• Last Update Submitted That Met QC Criteria: August 7, 2020
• Last Verified: August 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Site; Bone Marrow Diseases; Hematologic Diseases; Myeloproliferative Disorders; Bone Marrow Neoplasms; Hematologic Neoplasms; Polycythemia Vera; Polycythemia; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antineoplastic Agents; Protein Kinase Inhibitors; Erlotinib Hydrochloride
• Other Study ID Numbers: 2249