Polycythemia Vera (PV) is a disease of bone marrow stem cells that manifests in a drastic increase of red blood cells and frequently also of white blood cells. The "thickening" of the blood in relation with a modified function of the cells has several consequences like increased blood pressure, pruritus of the skin, fatigue, disturbed blood circulation in the brain as well as fingers and toes and an increased risk of arterial and venous thrombosis (thrombosis is the formation of a blood clot in a vessel); like stroke, cardiac infarction, deep vein thrombosis in the legs. In case of a strong increase of platelets there is an additional risk of bleedings. As the disease progresses the size of spleen and liver increased in most cases and the bone marrow shows signs of fibrosis. In some cases of PV a progression at a later time point to a leukemia (increased formation of white blood cells) can occur. The aim of this study is to assess the ease of AOP2014 self-administration using dedicated questionnaires. * To assess safety and tolerability: adverse events (AEs), laboratory parameters, electrocardiogram (ECG) throughout study. * To assess maintenance of the blood efficacy parameters Hct (Hematocrit), WBC (white blood cells) and PLTs (platelets) and spleen size (comparing values at Visit P7 vs. values at Visit P1). * To assess the feasibility of AOP2014 self-administration: defined as the ability of the patients to use the pen as a self-administration tool (ease of handling, safety, tolerability and efficacy).
This is a Phase III, single-arm study performed in patients who completed the AOP2014 arm of the PROUD-PV study or are currently participating in the CONTINUATION-PV study. After signing the informed consent form (ICF), approximately 30 patients will be enrolled consecutively into the study at participating sites according to the inclusion and exclusion criteria.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
30
Subjects will continue to receive the dosage which delivers the optimal disease response (hematocrit \[Hct\]\<45%, platelets \[PLTs\]\<400 x 109/L and leukocytes \[WBCs\]\<10 x 109/L), as determined in the PROUD-PV study, preferably at the level of target blood values.
LKH Graz
Graz, Austria
University Hospital Innsbruck
Innsbruck, Austria
Elisabethinen Hospital Linz
Linz, Austria
Salzburg Regional Hospital
Salzburg, Austria
Hanusch Hospital
Vienna, Austria
Medical University Vienna
Vienna, Austria
To evaluate ease of self-administration of AOP2014
To evaluate ease of self-administration of AOP2014 as assessed by staff and patients using dedicated questionnaires, using rates of full success and failure rates (defined in the statistics section of the synopsis).
Time frame: 3 months
Adverse Event
biweekly, using dedicated questionnaires
Time frame: 3 month
number of phlebotomies
biweekly
Time frame: 3 months
Disease response
The main efficacy evaluation criterion will be disease response defined as: • Hct (Hematocrit)\< 45% without phlebotomy (at least 3 months since the last phlebotomy). The hematological parameters will be measured by the local laboratories at clinical sites.
Time frame: 3 months
Disease response
The main efficacy evaluation criterion will be disease response defined as: • PLTs (Platelets)\< 400 x 109/L. The hematological parameters will be measured by the local laboratories at clinical sites.
Time frame: 3 months
Disease response
The main efficacy evaluation criterion will be disease response defined as: • WBCs (White blood cells)\< 10 x 109/L. The hematological parameters will be measured by the local laboratories at clinical sites.
Time frame: 3 months
blood parameters
first biweekly than monthly The main efficacy evaluation criterion will be disease response defined as: • Hct\< 45% without phlebotomy (at least 3 months since the last phlebotomy). The hematological parameters will be measured by the local laboratories at clinical sites.
Time frame: 3 months
blood parameters
first biweekly than monthly The main efficacy evaluation criterion will be disease response defined as: • WBCs\< 10 x 109/L. The hematological parameters will be measured by the local laboratories at clinical sites.
Time frame: 3 months
blood parameters
first biweekly than monthly The main efficacy evaluation criterion will be disease response defined as: • PLTs\< 400 x 109/L. The hematological parameters will be measured by the local laboratories at clinical sites.
Time frame: 3 months
spleen size
locally, Sonography will be used for measuring the spleen size (length). at Visit 1 and at the End of the study (week 12)
Time frame: 3 months
disease related symptoms
biweekly, using dedicated questionnaires
Time frame: 3 months
protocol-specific adverse events of special interest
biweekly, using dedicated questionnaires
Time frame: 3 months
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