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Polycythemia VeraPhase 3PEN-PVNCT02523638

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

A Phase 3 study testing Pegylated-Proline-Interferon alpha-2b in a Pre-filled Pen in people with Polycythemia Vera, run by AOP Orphan Pharmaceuticals AG at 35 sites in 8 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 18 to 99 years
Conditions the study is for
Polycythemia Vera
What is being tested?
Pegylated-Proline-Interferon alpha-2b in a Pre-filled Pen
Could I receive a placebo?
No, everyone receives an active treatment
How many people
30 participants
Where
35 sitesin 8 countries
Study dates
Jul 2015to Dec 2015
Phase
Phase 3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 99 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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.

Trial locations and contacts

35 sites in 8 countries.

35 sites in 8 countries

Austria7 sites

  • LKH Graz

    Graz

  • University Hospital Innsbruck

    Innsbruck

  • Elisabethinen Hospital Linz

    Linz

  • Salzburg Regional Hospital

    Salzburg

  • Hanusch Hospital

    Vienna

  • Medical University Vienna

    Vienna

  • Hospital Wels-Grieskirchen

    Wels

Other trials in Austria

Poland6 sites

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

    Katowice

  • University Hospital in Cracow

    Krakow

  • Independent Public Teaching Hospital No.1 in Lublin

    Lublin

  • Fryderyk Chopin Provincial Specialized Hospital

    Rzeszów

  • Nicolaus Copernicus Municipal Specialist Hospital

    Torun

  • Institute of Hematology and Transfusion Medicine

    Warsaw

Other trials in Poland

Czechia5 sites

  • University Hospital Brno

    Brno

  • University Hospital Hradec Kralove

    Hradec Králové

  • Institute of Hematology and Blood Transfusion

    Prague

  • University Hospital Kralovske Vinohrady

    Prague

  • University Hospital Motol

    Prague

Other trials in Czechia

Hungary5 sites

  • St Istvan and St Laszlo Hospital of Budapest

    Budapest

  • University of Debrecen

    Debrecen

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

    Gyula

  • Kaposi Mor County Teaching Hospital

    Kaposvár

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

    Szeged

Other trials in Hungary

Ukraine5 sites

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

    Cherkasy

  • Dnipropetrovsk City Multispecialty Clinical Hospital #4

    Dnipropetrovsk

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

    Kiev

  • Institute of Blood Pathology and Transfusion Medicine

    Lviv

  • O.F. Herbachevskyi Regional Clinical Hospital

    Zhytomyr

Other trials in Ukraine

France3 sites

  • Institute Paoli-Calmettes

    Marseille

  • Hospital Saint-Louis

    Paris

  • Clinical Research Center CIC

    Poitiers

Other trials in France

Bulgaria2 sites

  • Specialized Hospital for Active Treatment of Hematological Diseases

    Sofia

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

    Vratsa

Other trials in Bulgaria

Slovakia2 sites

  • University Hospital with Outpatient Clinic F.D. Roosevelt

    Banská Bystrica

  • Saint Cyril and Metod University Hospital Bratislava

    Bratislava

Other trials in Slovakia

About this study

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

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).
Read the 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.

Being tested
Pegylated-Proline-Interferon alpha-2b in a Pre-filled Pen
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
Medical subject headings
Polycythemia Vera, Polycythemia, Hematologic Diseases, Myeloproliferative Disorders, Bone Marrow Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Pegylated-Proline-Interferon alpha-2b in a Pre-filled PenDrug

    Also called AOP2014

    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.

Study groups in NCT02523638
Study groupRoleWhat they receive
Pegylated- Proline-Interferon alpha-2bOtherPegylated-Proline-Interferon alpha-2b in a Pre-filled Pen single arm
Receives: Drug: Pegylated-Proline-Interferon alpha-2b in a Pre-filled Pen

What is being measured

The primary outcome is the main question the study is designed to answer.

  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).

    Measured: 3 months

Secondary outcomes (11)

  1. Adverse Event

    biweekly, using dedicated questionnaires

    Measured: 3 month

  2. number of phlebotomies

    biweekly

    Measured: 3 months

  3. 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.

    Measured: 3 months

  4. 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.

    Measured: 3 months

  5. 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.

    Measured: 3 months

  6. 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.

    Measured: 3 months

  7. 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.

    Measured: 3 months

  8. 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.

    Measured: 3 months

  9. 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)

    Measured: 3 months

  10. disease related symptoms

    biweekly, using dedicated questionnaires

    Measured: 3 months

  11. protocol-specific adverse events of special interest

    biweekly, using dedicated questionnaires

    Measured: 3 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted14 Aug 2015 (estimated)
  2. Study startJul 2015
  3. Primary completionDec 2015
  4. Study completionDec 2015

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
30 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02523638
Sponsor's protocol ID
PEN-PV
Other IDs
2014-001356-31, 2014
Acronym
PEN-PV
Status last verified
Feb 2016
First submitted
12 Jun 2015
Last updated
17 Feb 2016
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Pegylated-Proline-Interferon alpha-2b in a Pre-filled Pen in people with Polycythemia Vera, run by AOP Orphan Pharmaceuticals AG at 35 sites in 8 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 17 Feb 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 35 sites in Austria, Poland, Czechia, Hungary, Ukraine and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

To evaluate ease of self-administration of AOP2014 (time frame: 3 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02523638.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Feb 2016.

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