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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

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 Summary: 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.
Sponsor: AOP Orphan Pharmaceuticals AG

Current Primary Outcome: To evaluate ease of self-administration of AOP2014 [ Time Frame: 3 months ]

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).


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Adverse Event [ Time Frame: 3 month ]
    biweekly, using dedicated questionnaires
  • number of phlebotomies [ Time Frame: 3 months ]
    biweekly
  • Disease response [ Time Frame: 3 months ]

    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 [ Time Frame: 3 months ]

    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 [ Time Frame: 3 months ]

    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 [ Time Frame: 3 months ]

    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 [ Time Frame: 3 months ]

    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 [ Time Frame: 3 months ]

    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 [ Time Frame: 3 months ]
    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 [ Time Frame: 3 months ]
    biweekly, using dedicated questionnaires
  • protocol-specific adverse events of special interest [ Time Frame: 3 months ]
    biweekly, using dedicated questionnaires


Original Secondary Outcome: Same as current

Information By: AOP Orphan Pharmaceuticals AG

Dates:
Date Received: June 12, 2015
Date Started: July 2015
Date Completion:
Last Updated: February 16, 2016
Last Verified: February 2016