Study to Assess the Self-administration of AOP2014 Using a Pen, Developed for the Treatment of Polycythemia Vera Patients
- Conditions
- Polycythemia Vera
- Interventions
- Drug: Pegylated-Proline-Interferon alpha-2b in a Pre-filled Pen
- Registration Number
- NCT02523638
- Lead Sponsor
- AOP Orphan Pharmaceuticals AG
- Brief Summary
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).
- Detailed Description
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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 30
-
Patients who either completed the 12 months AOP2014 treatment arm of the PROUD-PV study, or are currently participating in the CONTINUATION-PV, and at the "EoT visit" (End of treatment visit) of the PROUD-PV study or two weeks after the last assessment visit of the CONTINUATION-PV study, fulfill at least one of the following criteria:
- Normalization of at least two out of three main blood parameters (Hct (Hematocrit), PLTs (Platelets) and WBCs (white blood cells) if these parameters were moderately increased (Hct<50%, WBCs<20 x 109/L, PLTs<600 x 109/L) at baseline visit of the PROUD-PV study, OR
- >35% decrease of at least two out of three main blood parameters (Hct, PLTs and WBCs) if these parameters were massively increased (Hct>50%, WBCs>20 x 109/L, PLTs >600 x 109/L), at baseline visit of the PROUD-PV study, OR
- Normalization of spleen size, if spleen was enlarged at baseline visit of the PROUD-PV study, OR
- Otherwise a clear, medically verified benefit from treatment with AOP2014 (e.g. normalization of disease-related micro-vasculatory symptoms, substantial decrease of JAK2 (Januskinase 2) allelic burden).
-
Signed written ICF.
Withdrawal criteria, as specified in the PROUD-PV and CONTINUATION-PV studies, which mandate treatment discontinuation.
- Non-recovery from the AOP2014 related toxicities to the grade (usually, Grade I) which allows continuation of the treatment.
- HADS (Hospital Anxiety and Depression Scale) score of 11 or higher on either or both of the subscales, and /or development or worsening of clinically significant depression or suicidal thoughts.
- Progressive and clinically significant increase of liver enzyme levels despite dose reduction, or if such increase is accompanied by increased bilirubin level, any signs or symptoms of a clinically significant autoimmune disease.
- Clinically significant development of a new ophthalmologic disorder, or worsening of a pre-existing one, during the study.
- Loss of efficacy of AOP2014 or any comparable situation where no further benefits of treatment continuation are expected by the investigator.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Pegylated- Proline-Interferon alpha-2b Pegylated-Proline-Interferon alpha-2b in a Pre-filled Pen Pegylated-Proline-Interferon alpha-2b in a Pre-filled Pen single arm
- Primary Outcome Measures
Name Time Method To evaluate ease of self-administration of AOP2014 3 months 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).
- Secondary Outcome Measures
Name Time Method number of phlebotomies 3 months biweekly
spleen size 3 months locally, Sonography will be used for measuring the spleen size (length). at Visit 1 and at the End of the study (week 12)
disease related symptoms 3 months biweekly, using dedicated questionnaires
protocol-specific adverse events of special interest 3 months biweekly, using dedicated questionnaires
Adverse Event 3 month biweekly, using dedicated questionnaires
Disease response 3 months 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.blood parameters 3 months 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.
Trial Locations
- Locations (35)
Dnipropetrovsk City Multispecialty Clinical Hospital #4
🇺🇦Dnipropetrovsk, Ukraine
University of Szeged, Albert Szent-Gyorgyi Clinical Center, Koranyi fasor 6
ðŸ‡ðŸ‡ºSzeged, Hungary
O.F. Herbachevskyi Regional Clinical Hospital
🇺🇦Zhytomyr, Ukraine
University Hospital Innsbruck
🇦🇹Innsbruck, Austria
Medical University Vienna
🇦🇹Vienna, Austria
Institute of Hematology and Transfusion Medicine
🇵🇱Warsaw, Poland
Saint Cyril and Metod University Hospital Bratislava
🇸🇰Bratislava, Slovakia
Cherkasy Regional Oncology Center, Regional Treatment and Diagnostics Hematology Center
🇺🇦Cherkasy, Ukraine
Institute of Blood Pathology and Transfusion Medicine
🇺🇦Lviv, Ukraine
Nicolaus Copernicus Municipal Specialist Hospital
🇵🇱Torun, Poland
Elisabethinen Hospital Linz
🇦🇹Linz, Austria
Salzburg Regional Hospital
🇦🇹Salzburg, Austria
Independent Public Teaching Hospital No.1 in Lublin
🇵🇱Lublin, Poland
University Hospital with Outpatient Clinic F.D. Roosevelt
🇸🇰Banska Bystrica, Slovakia
Kaposi Mor County Teaching Hospital
ðŸ‡ðŸ‡ºKaposvar, Hungary
Institute of Hematology and Blood Transfusion
🇨🇿Prague, Czech Republic
Andrzej Mielecki Independent Public Clinical Hospital of Medical University of Silesia in Katowice
🇵🇱Katowice, Poland
University of Debrecen
ðŸ‡ðŸ‡ºDebrecen, Hungary
Hanusch Hospital
🇦🇹Vienna, Austria
LKH Graz
🇦🇹Graz, Austria
Multiprofile Hospital for Active Treatment - Hristo Botev, Vratsa, First Department of Internal Medicine
🇧🇬Vratsa, Bulgaria
Specialized Hospital for Active Treatment of Hematological Diseases
🇧🇬Sofia, Bulgaria
Hospital Wels-Grieskirchen
🇦🇹Wels, Austria
University Hospital Hradec Kralove
🇨🇿Hradec Kralove, Czech Republic
University Hospital Kralovske Vinohrady
🇨🇿Prague, Czech Republic
University Hospital Motol
🇨🇿Prague, Czech Republic
Institute Paoli-Calmettes
🇫🇷Marseilles, France
Hospital Saint-Louis
🇫🇷Paris, France
St Istvan and St Laszlo Hospital of Budapest
ðŸ‡ðŸ‡ºBudapest, Hungary
Clinical Research Center CIC
🇫🇷Poitiers, France
Bekes County Pandy Kalman Hospital, 1st Department of Medicine, Hematology
ðŸ‡ðŸ‡ºGyula, Hungary
University Hospital Brno
🇨🇿Brno, Czech Republic
University Hospital in Cracow
🇵🇱Krakow, Poland
Fryderyk Chopin Provincial Specialized Hospital
🇵🇱Rzeszow, Poland
National Research Center for Radiation Medicine, Institute of Clinical Radiology
🇺🇦Kiev, Ukraine