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

NCT02523638 | Completed Phase 3 DMC

• 2 other identifiers: PEN-PV2014-001356-31
• Study Type: interventional
• Target: 30
• Locations: 8 countries
• Sites: 35

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).

Conditions

Polycythemia Vera

Keywords

Pegylated-Proline-interferon alpha-2b (AOP2014); Polycythemia Vera; PEN-PV; PROUD-PV; CONTINUATION-PV; AOP Orphan; Polycythemia; Hematologic Diseases; Myeloproliferative Disorders; Bone Marrow Diseases; Interferon-alpha; Peginterferon alfa-2b

Outcome Measures

Primary Outcomes (1)

• 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).

  3 months

Secondary Outcomes (11)

• Adverse Event

  3 month

• number of phlebotomies

  3 months

• Disease response

  3 months

• Disease response

  3 months

• Disease response

  3 months

Study Arms (1)

Pegylated- Proline-Interferon alpha-2b

OTHER

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

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

Interventions

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

Also known as: AOP2014

Pegylated- Proline-Interferon alpha-2b

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• AOP Orphan Pharmaceuticals AG: lead
• PharmaEssentia Corporation (Co-Sponsor for USA): collaborator

Study Sites (35)

LKH Graz

Graz, Austria

Location

University Hospital Innsbruck

Innsbruck, Austria

Location

Elisabethinen Hospital Linz

Linz, Austria

Location

Salzburg Regional Hospital

Salzburg, Austria

Location

Hanusch Hospital

Vienna, Austria

Location

Medical University Vienna

Vienna, Austria

Location

Hospital Wels-Grieskirchen

Wels, Austria

Location

Specialized Hospital for Active Treatment of Hematological Diseases

Sofia, Bulgaria

Location

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

Vratsa, Bulgaria

Location

University Hospital Brno

Brno, Czechia

Location

University Hospital Hradec Kralove

Hradec Králové, Czechia

Location

Institute of Hematology and Blood Transfusion

Prague, Czechia

Location

University Hospital Kralovske Vinohrady

Prague, Czechia

Location

University Hospital Motol

Prague, Czechia

Location

Institute Paoli-Calmettes

Marseille, France

Location

Hospital Saint-Louis

Paris, France

Location

Clinical Research Center CIC

Poitiers, France

Location

St Istvan and St Laszlo Hospital of Budapest

Budapest, Hungary

Location

University of Debrecen

Debrecen, Hungary

Location

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

Gyula, Hungary

Location

Kaposi Mor County Teaching Hospital

Kaposvár, Hungary

Location

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

Szeged, Hungary

Location

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

Katowice, Poland

Location

University Hospital in Cracow

Krakow, Poland

Location

Independent Public Teaching Hospital No.1 in Lublin

Lublin, Poland

Location

Fryderyk Chopin Provincial Specialized Hospital

Rzeszów, Poland

Location

Nicolaus Copernicus Municipal Specialist Hospital

Torun, Poland

Location

Institute of Hematology and Transfusion Medicine

Warsaw, Poland

Location

University Hospital with Outpatient Clinic F.D. Roosevelt

Banská Bystrica, Slovakia

Location

Saint Cyril and Metod University Hospital Bratislava

Bratislava, Slovakia

Location

Cherkasy Regional Oncology Center, Regional Treatment and Diagnostics Hematology Center

Cherkasy, Ukraine

Location

Dnipropetrovsk City Multispecialty Clinical Hospital #4

Dnipropetrovsk, Ukraine

Location

National Research Center for Radiation Medicine, Institute of Clinical Radiology

Kiev, Ukraine

Location

Institute of Blood Pathology and Transfusion Medicine

Lviv, Ukraine

Location

O.F. Herbachevskyi Regional Clinical Hospital

Zhytomyr, Ukraine

Location

MeSH Terms

Conditions

Polycythemia Vera; Polycythemia; Hematologic Diseases; Myeloproliferative Disorders; Bone Marrow Diseases

Condition Hierarchy (Ancestors)

Bone Marrow Neoplasms; Hematologic Neoplasms; Neoplasms by Site; Neoplasms; Hemic and Lymphatic Diseases

Study Officials

• Heinz Gisslinger, MD

  Med Uni Wien

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 12, 2015
• First Posted: August 14, 2015
• Study Start: July 1, 2015
• Primary Completion: December 1, 2015
• Study Completion: December 1, 2015
• Last Updated: February 17, 2016

Record last verified: 2016-02

Locations

PL (6); CZ (5); SL (2); BU (2); HU (5); AU (7); UA (5); FR (3)