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

Primary Purpose

Polycythemia Vera

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Pegylated-Proline-Interferon alpha-2b in a Pre-filled Pen
Sponsored by
AOP Orphan Pharmaceuticals AG
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Polycythemia Vera focused on measuring 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

Eligibility Criteria

18 Years - 99 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • LKH Graz
  • University Hospital Innsbruck
  • Elisabethinen Hospital Linz
  • Salzburg Regional Hospital
  • Hanusch Hospital
  • Medical University Vienna
  • Hospital Wels-Grieskirchen
  • Specialized Hospital for Active Treatment of Hematological Diseases
  • Multiprofile Hospital for Active Treatment - Hristo Botev, Vratsa, First Department of Internal Medicine
  • University Hospital Brno
  • University Hospital Hradec Kralove
  • Institute of Hematology and Blood Transfusion
  • University Hospital Kralovske Vinohrady
  • University Hospital Motol
  • Institute Paoli-Calmettes
  • Hospital Saint-Louis
  • Clinical Research Center CIC
  • St Istvan and St Laszlo Hospital of Budapest
  • University of Debrecen
  • Bekes County Pandy Kalman Hospital, 1st Department of Medicine, Hematology
  • Kaposi Mor County Teaching Hospital
  • University of Szeged, Albert Szent-Gyorgyi Clinical Center, Koranyi fasor 6
  • Andrzej Mielecki Independent Public Clinical Hospital of Medical University of Silesia in Katowice
  • University Hospital in Cracow
  • Independent Public Teaching Hospital No.1 in Lublin
  • Fryderyk Chopin Provincial Specialized Hospital
  • Nicolaus Copernicus Municipal Specialist Hospital
  • Institute of Hematology and Transfusion Medicine
  • University Hospital with Outpatient Clinic F.D. Roosevelt
  • Saint Cyril and Metod University Hospital Bratislava
  • Cherkasy Regional Oncology Center, Regional Treatment and Diagnostics Hematology Center
  • Dnipropetrovsk City Multispecialty Clinical Hospital #4
  • National Research Center for Radiation Medicine, Institute of Clinical Radiology
  • Institute of Blood Pathology and Transfusion Medicine
  • O.F. Herbachevskyi Regional Clinical Hospital

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

Pegylated- Proline-Interferon alpha-2b

Arm Description

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

Outcomes

Primary Outcome Measures

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).

Secondary Outcome Measures

Adverse Event
biweekly, using dedicated questionnaires
number of phlebotomies
biweekly
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.
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.
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.
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.
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.
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.
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)
disease related symptoms
biweekly, using dedicated questionnaires
protocol-specific adverse events of special interest
biweekly, using dedicated questionnaires

Full Information

First Posted
June 12, 2015
Last Updated
February 16, 2016
Sponsor
AOP Orphan Pharmaceuticals AG
Collaborators
PharmaEssentia Corporation (Co-Sponsor for USA)
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1. Study Identification

Unique Protocol Identification Number
NCT02523638
Brief Title
Study to Assess the Self-administration of AOP2014 Using a Pen, Developed for the Treatment of Polycythemia Vera Patients
Acronym
PEN-PV
Official Title
An Open-label, Single Arm, Phase III Study to Assess the Self-administration of AOP2014 Using a Pre-filled Pen, Developed for the Treatment of Polycythemia Vera Patients
Study Type
Interventional

2. Study Status

Record Verification Date
February 2016
Overall Recruitment Status
Completed
Study Start Date
July 2015 (undefined)
Primary Completion Date
December 2015 (Actual)
Study Completion Date
December 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
AOP Orphan Pharmaceuticals AG
Collaborators
PharmaEssentia Corporation (Co-Sponsor for USA)

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Pegylated- Proline-Interferon alpha-2b
Arm Type
Other
Arm Description
Pegylated-Proline-Interferon alpha-2b in a Pre-filled Pen single arm
Intervention Type
Drug
Intervention Name(s)
Pegylated-Proline-Interferon alpha-2b in a Pre-filled Pen
Other Intervention Name(s)
AOP2014
Intervention Description
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.
Primary Outcome Measure Information:
Title
To evaluate ease of self-administration of AOP2014
Description
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
Secondary Outcome Measure Information:
Title
Adverse Event
Description
biweekly, using dedicated questionnaires
Time Frame
3 month
Title
number of phlebotomies
Description
biweekly
Time Frame
3 months
Title
Disease response
Description
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
Title
Disease response
Description
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
Title
Disease response
Description
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
Title
blood parameters
Description
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
Title
blood parameters
Description
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
Title
blood parameters
Description
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
Title
spleen size
Description
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
Title
disease related symptoms
Description
biweekly, using dedicated questionnaires
Time Frame
3 months
Title
protocol-specific adverse events of special interest
Description
biweekly, using dedicated questionnaires
Time Frame
3 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
99 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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. Exclusion Criteria: 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Heinz Gisslinger, MD
Organizational Affiliation
Med Uni Wien
Official's Role
Principal Investigator
Facility Information:
Facility Name
LKH Graz
City
Graz
Country
Austria
Facility Name
University Hospital Innsbruck
City
Innsbruck
Country
Austria
Facility Name
Elisabethinen Hospital Linz
City
Linz
Country
Austria
Facility Name
Salzburg Regional Hospital
City
Salzburg
Country
Austria
Facility Name
Hanusch Hospital
City
Vienna
Country
Austria
Facility Name
Medical University Vienna
City
Vienna
Country
Austria
Facility Name
Hospital Wels-Grieskirchen
City
Wels
Country
Austria
Facility Name
Specialized Hospital for Active Treatment of Hematological Diseases
City
Sofia
Country
Bulgaria
Facility Name
Multiprofile Hospital for Active Treatment - Hristo Botev, Vratsa, First Department of Internal Medicine
City
Vratsa
Country
Bulgaria
Facility Name
University Hospital Brno
City
Brno
Country
Czech Republic
Facility Name
University Hospital Hradec Kralove
City
Hradec Kralove
Country
Czech Republic
Facility Name
Institute of Hematology and Blood Transfusion
City
Prague
Country
Czech Republic
Facility Name
University Hospital Kralovske Vinohrady
City
Prague
Country
Czech Republic
Facility Name
University Hospital Motol
City
Prague
Country
Czech Republic
Facility Name
Institute Paoli-Calmettes
City
Marseilles
Country
France
Facility Name
Hospital Saint-Louis
City
Paris
Country
France
Facility Name
Clinical Research Center CIC
City
Poitiers
Country
France
Facility Name
St Istvan and St Laszlo Hospital of Budapest
City
Budapest
Country
Hungary
Facility Name
University of Debrecen
City
Debrecen
Country
Hungary
Facility Name
Bekes County Pandy Kalman Hospital, 1st Department of Medicine, Hematology
City
Gyula
Country
Hungary
Facility Name
Kaposi Mor County Teaching Hospital
City
Kaposvar
Country
Hungary
Facility Name
University of Szeged, Albert Szent-Gyorgyi Clinical Center, Koranyi fasor 6
City
Szeged
Country
Hungary
Facility Name
Andrzej Mielecki Independent Public Clinical Hospital of Medical University of Silesia in Katowice
City
Katowice
Country
Poland
Facility Name
University Hospital in Cracow
City
Krakow
Country
Poland
Facility Name
Independent Public Teaching Hospital No.1 in Lublin
City
Lublin
Country
Poland
Facility Name
Fryderyk Chopin Provincial Specialized Hospital
City
Rzeszow
Country
Poland
Facility Name
Nicolaus Copernicus Municipal Specialist Hospital
City
Torun
Country
Poland
Facility Name
Institute of Hematology and Transfusion Medicine
City
Warsaw
Country
Poland
Facility Name
University Hospital with Outpatient Clinic F.D. Roosevelt
City
Banska Bystrica
Country
Slovakia
Facility Name
Saint Cyril and Metod University Hospital Bratislava
City
Bratislava
Country
Slovakia
Facility Name
Cherkasy Regional Oncology Center, Regional Treatment and Diagnostics Hematology Center
City
Cherkasy
Country
Ukraine
Facility Name
Dnipropetrovsk City Multispecialty Clinical Hospital #4
City
Dnipropetrovsk
Country
Ukraine
Facility Name
National Research Center for Radiation Medicine, Institute of Clinical Radiology
City
Kiev
Country
Ukraine
Facility Name
Institute of Blood Pathology and Transfusion Medicine
City
Lviv
Country
Ukraine
Facility Name
O.F. Herbachevskyi Regional Clinical Hospital
City
Zhytomyr
Country
Ukraine

12. IPD Sharing Statement

Learn more about this trial

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

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