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Trial record 4 of 234 for:    "Polycythemia vera"

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

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ClinicalTrials.gov Identifier: NCT02523638
Recruitment Status : Completed
First Posted : August 14, 2015
Last Update Posted : February 17, 2016
Sponsor:
Collaborator:
PharmaEssentia Corporation (Co-Sponsor for USA)
Information provided by (Responsible Party):
AOP Orphan Pharmaceuticals AG

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).

Condition or disease Intervention/treatment Phase
Polycythemia Vera Drug: Pegylated-Proline-Interferon alpha-2b in a Pre-filled Pen Phase 3

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.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Start Date : July 2015
Actual Primary Completion Date : December 2015
Actual Study Completion Date : December 2015


Arm Intervention/treatment
Pegylated- Proline-Interferon alpha-2b
Pegylated-Proline-Interferon alpha-2b in a Pre-filled Pen single arm
Drug: 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.
Other Name: AOP2014




Primary Outcome Measures :
  1. 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).


Secondary Outcome Measures :
  1. Adverse Event [ Time Frame: 3 month ]
    biweekly, using dedicated questionnaires

  2. number of phlebotomies [ Time Frame: 3 months ]
    biweekly

  3. 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.


  4. 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.


  5. 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.


  6. 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.


  7. 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.


  8. 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.


  9. 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)

  10. disease related symptoms [ Time Frame: 3 months ]
    biweekly, using dedicated questionnaires

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



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 99 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02523638


  Show 35 Study Locations
Sponsors and Collaborators
AOP Orphan Pharmaceuticals AG
PharmaEssentia Corporation (Co-Sponsor for USA)
Investigators
Principal Investigator: Heinz Gisslinger, MD Med Uni Wien

Responsible Party: AOP Orphan Pharmaceuticals AG
ClinicalTrials.gov Identifier: NCT02523638     History of Changes
Other Study ID Numbers: PEN-PV
2014-001356-31 ( EudraCT Number )
First Posted: August 14, 2015    Key Record Dates
Last Update Posted: February 17, 2016
Last Verified: February 2016

Keywords provided by AOP Orphan Pharmaceuticals AG:
Polycythemia Vera
Pegylated-Proline-interferon alpha-2b (AOP2014)
PEN-PV
PROUD-PV
CONTINUATION-PV
AOP Orphan
Polycythemia
Hematologic Diseases
Myeloproliferative Disorders
Bone Marrow Diseases
Interferon-alpha
Peginterferon alfa-2b

Additional relevant MeSH terms:
Polycythemia Vera
Polycythemia
Hematologic Diseases
Bone Marrow Neoplasms
Hematologic Neoplasms
Neoplasms by Site
Neoplasms
Bone Marrow Diseases
Myeloproliferative Disorders
Interferons
Interferon-alpha
Antineoplastic Agents
Antiviral Agents
Anti-Infective Agents
Immunologic Factors
Physiological Effects of Drugs