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

NCT ID: NCT02523638

Last Updated: 2016-02-17

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-07-31
• Study Completion Date: 2015-12-31

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.

Conditions

Polycythemia Vera

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Pegylated- Proline-Interferon alpha-2b

Pegylated-Proline-Interferon alpha-2b in a Pre-filled Pen single arm

Group Type: OTHER

Pegylated-Proline-Interferon alpha-2b in a Pre-filled Pen

Intervention Type: DRUG

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.

Interventions

Pegylated-Proline-Interferon alpha-2b in a Pre-filled Pen

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.

Intervention Type: DRUG

Other Intervention Names

AOP2014

Eligibility Criteria

Inclusion Criteria

1. 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).

2. Signed written ICF.

Exclusion Criteria

Withdrawal criteria, as specified in the PROUD-PV and CONTINUATION-PV studies, which mandate treatment discontinuation.

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

2. 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.

3. 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.

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

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 99 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

PharmaEssentia Corporation (Co-Sponsor for USA)

UNKNOWN

Sponsor Role: collaborator

AOP Orphan Pharmaceuticals AG

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Heinz Gisslinger, MD

Role: PRINCIPAL_INVESTIGATOR

Med Uni Wien

Locations

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

Hospital Wels-Grieskirchen

Wels, , Austria

Specialized Hospital for Active Treatment of Hematological Diseases

Sofia, , Bulgaria

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

Vratsa, , Bulgaria

University Hospital Brno

Brno, , Czechia

University Hospital Hradec Kralove

Hradec Králové, , Czechia

Institute of Hematology and Blood Transfusion

Prague, , Czechia

University Hospital Kralovske Vinohrady

Prague, , Czechia

University Hospital Motol

Prague, , Czechia

Institute Paoli-Calmettes

Marseille, , France

Hospital Saint-Louis

Paris, , France

Clinical Research Center CIC

Poitiers, , France

St Istvan and St Laszlo Hospital of Budapest

Budapest, , Hungary

University of Debrecen

Debrecen, , Hungary

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

Gyula, , Hungary

Kaposi Mor County Teaching Hospital

Kaposvár, , Hungary

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

Szeged, , Hungary

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

Katowice, , Poland

University Hospital in Cracow

Krakow, , Poland

Independent Public Teaching Hospital No.1 in Lublin

Lublin, , Poland

Fryderyk Chopin Provincial Specialized Hospital

Rzeszów, , Poland

Nicolaus Copernicus Municipal Specialist Hospital

Torun, , Poland

Institute of Hematology and Transfusion Medicine

Warsaw, , Poland

University Hospital with Outpatient Clinic F.D. Roosevelt

Banská Bystrica, , Slovakia

Saint Cyril and Metod University Hospital Bratislava

Bratislava, , Slovakia

Cherkasy Regional Oncology Center, Regional Treatment and Diagnostics Hematology Center

Cherkasy, , Ukraine

Dnipropetrovsk City Multispecialty Clinical Hospital #4

Dnipropetrovsk, , Ukraine

National Research Center for Radiation Medicine, Institute of Clinical Radiology

Kiev, , Ukraine

Institute of Blood Pathology and Transfusion Medicine

Lviv, , Ukraine

O.F. Herbachevskyi Regional Clinical Hospital

Zhytomyr, , Ukraine

Countries

Austria; Bulgaria; Czechia; France; Hungary; Poland; Slovakia; Ukraine

Other Identifiers

2014-001356-31

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

PEN-PV

Identifier Type: -

Identifier Source: org_study_id