Clinical trial NCT01038856: Trial of Erlotinib in Patients With JAK-2 V617F Positive Polycythemia Vera
Official title: Phase II Trial of Erlotinib in Patients With JAK-2 V617F Positive Polycythemia Vera
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Polycythemia Vera
Lead sponsor: University of Oklahoma
Sponsor class: OTHER
Collaborators: name: OSI Pharmaceuticals
First posted: 2009-12-24
Enrollment: 5
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: 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.

Eligibility criteria: 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

Primary outcomes:
measure: Overall Response Rate to Include Complete Hematological Response, Complete Molecular Response, Partial Hematological Response, and Minimal Hematological Response
timeFrame: Day 15
Secondary outcomes:
measure: Incidence of Toxicities
timeFrame: First assessment at day 15, subsequent assessments at 28 day intervals for an average of 1 year
measure: Improvement in Splenomegaly Size
timeFrame: 4 months, end of treatment and 12 months end of treatment
measure: Decrease of Mutant JAK2V617F Allele Burden
timeFrame: every 2 months until end of treatment and 12 months after end of treatment
Other outcomes:
Locations:
city: Oklahoma City
country: United States
facility: University of Oklahoma Health Sciences Center
state: Oklahoma
Study officials:
affiliation: University of Oklahoma
name: Mohamad Cherry, MD
role: PRINCIPAL_INVESTIGATOR
References: