Study to Assess the Self-administration of AOP2014 Using a Pen, Developed for the Treatment of Polycythemia Vera Patients

Last updated: February 16, 2016
Sponsor: AOP Orphan Pharmaceuticals AG
Overall Status: Completed

Phase

3

Condition

Red Blood Cell Disorders

Leukemia (Pediatric)

Bone Marrow Disorder

Treatment

N/A

Clinical Study ID

NCT02523638
PEN-PV
2014-001356-31
  • Ages 18-99
  • All Genders

Study 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).

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Patients who either completed the 12 months AOP2014 treatment arm of the PROUD-PVstudy, 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 assessmentvisit 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 parameterswere moderately increased (Hct<50%, WBCs<20 x 109/L, PLTs<600 x 109/L) atbaseline visit of the PROUD-PV study, OR

  • >35% decrease of at least two out of three main blood parameters (Hct, PLTs andWBCs) 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 thePROUD-PV study, OR

  • Otherwise a clear, medically verified benefit from treatment with AOP2014 (e.g.normalization of disease-related micro-vasculatory symptoms, substantial decreaseof JAK2 (Januskinase 2) allelic burden).

  1. Signed written ICF.

Exclusion

Exclusion Criteria: Withdrawal criteria, as specified in the PROUD-PV and CONTINUATION-PV studies, whichmandate treatment discontinuation.

  1. Non-recovery from the AOP2014 related toxicities to the grade (usually, Grade I) whichallows continuation of the treatment.

  2. HADS (Hospital Anxiety and Depression Scale) score of 11 or higher on either or bothof the subscales, and /or development or worsening of clinically significantdepression or suicidal thoughts.

  3. Progressive and clinically significant increase of liver enzyme levels despite dosereduction, or if such increase is accompanied by increased bilirubin level, any signsor symptoms of a clinically significant autoimmune disease.

  4. Clinically significant development of a new ophthalmologic disorder, or worsening of apre-existing one, during the study.

  5. Loss of efficacy of AOP2014 or any comparable situation where no further benefits oftreatment continuation are expected by the investigator.

Study Design

Total Participants: 30
Study Start date:
July 01, 2015
Estimated Completion Date:
December 31, 2015

Study 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.

Connect with a study center

  • LKH Graz

    Graz,
    Austria

    Site Not Available

  • University Hospital Innsbruck

    Innsbruck,
    Austria

    Site Not Available

  • Elisabethinen Hospital Linz

    Linz,
    Austria

    Site Not Available

  • Salzburg Regional Hospital

    Salzburg,
    Austria

    Site Not Available

  • Hanusch Hospital

    Vienna,
    Austria

    Site Not Available

  • Medical University Vienna

    Vienna,
    Austria

    Site Not Available

  • Hospital Wels-Grieskirchen

    Wels,
    Austria

    Site Not Available

  • Specialized Hospital for Active Treatment of Hematological Diseases

    Sofia,
    Bulgaria

    Site Not Available

  • Multiprofile Hospital for Active Treatment - Hristo Botev, Vratsa, First Department of Internal Medicine

    Vratsa,
    Bulgaria

    Site Not Available

  • University Hospital Brno

    Brno,
    Czech Republic

    Site Not Available

  • University Hospital Hradec Kralove

    Hradec Kralove,
    Czech Republic

    Site Not Available

  • Institute of Hematology and Blood Transfusion

    Prague,
    Czech Republic

    Site Not Available

  • University Hospital Kralovske Vinohrady

    Prague,
    Czech Republic

    Site Not Available

  • University Hospital Motol

    Prague,
    Czech Republic

    Site Not Available

  • Institute Paoli-Calmettes

    Marseilles,
    France

    Site Not Available

  • Hospital Saint-Louis

    Paris,
    France

    Site Not Available

  • Clinical Research Center CIC

    Poitiers,
    France

    Site Not Available

  • St Istvan and St Laszlo Hospital of Budapest

    Budapest,
    Hungary

    Site Not Available

  • University of Debrecen

    Debrecen,
    Hungary

    Site Not Available

  • Bekes County Pandy Kalman Hospital, 1st Department of Medicine, Hematology

    Gyula,
    Hungary

    Site Not Available

  • Kaposi Mor County Teaching Hospital

    Kaposvar,
    Hungary

    Site Not Available

  • University of Szeged, Albert Szent-Gyorgyi Clinical Center, Koranyi fasor 6

    Szeged,
    Hungary

    Site Not Available

  • Andrzej Mielecki Independent Public Clinical Hospital of Medical University of Silesia in Katowice

    Katowice,
    Poland

    Site Not Available

  • University Hospital in Cracow

    Krakow,
    Poland

    Site Not Available

  • Independent Public Teaching Hospital No.1 in Lublin

    Lublin,
    Poland

    Site Not Available

  • Fryderyk Chopin Provincial Specialized Hospital

    Rzeszow,
    Poland

    Site Not Available

  • Nicolaus Copernicus Municipal Specialist Hospital

    Torun,
    Poland

    Site Not Available

  • Institute of Hematology and Transfusion Medicine

    Warsaw,
    Poland

    Site Not Available

  • University Hospital with Outpatient Clinic F.D. Roosevelt

    Banska Bystrica,
    Slovakia

    Site Not Available

  • Saint Cyril and Metod University Hospital Bratislava

    Bratislava,
    Slovakia

    Site Not Available

  • Cherkasy Regional Oncology Center, Regional Treatment and Diagnostics Hematology Center

    Cherkasy,
    Ukraine

    Site Not Available

  • Dnipropetrovsk City Multispecialty Clinical Hospital #4

    Dnipropetrovsk,
    Ukraine

    Site Not Available

  • National Research Center for Radiation Medicine, Institute of Clinical Radiology

    Kiev,
    Ukraine

    Site Not Available

  • Institute of Blood Pathology and Transfusion Medicine

    Lviv,
    Ukraine

    Site Not Available

  • O.F. Herbachevskyi Regional Clinical Hospital

    Zhytomyr,
    Ukraine

    Site Not Available

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