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Trial record 3 of 18 for:    "Bone Marrow Cancer" | "interferons"

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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

Tracking Information
First Submitted Date  ICMJE June 12, 2015
First Posted Date  ICMJE August 14, 2015
Last Update Posted Date February 17, 2016
Study Start Date  ICMJE July 2015
Actual Primary Completion Date December 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 12, 2015)
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 Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02523638 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: August 12, 2015)
  • 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 Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study to Assess the Self-administration of AOP2014 Using a Pen, Developed for the Treatment of Polycythemia Vera Patients
Official Title  ICMJE 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 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  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Polycythemia Vera
Intervention  ICMJE 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
Study Arms  ICMJE Pegylated- Proline-Interferon alpha-2b
Pegylated-Proline-Interferon alpha-2b in a Pre-filled Pen single arm
Intervention: Drug: Pegylated-Proline-Interferon alpha-2b in a Pre-filled Pen
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Estimated Enrollment  ICMJE
 (submitted: August 12, 2015)
30
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE December 2015
Actual Primary Completion Date December 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 99 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Austria,   Bulgaria,   Czech Republic,   France,   Hungary,   Poland,   Slovakia,   Ukraine
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02523638
Other Study ID Numbers  ICMJE PEN-PV
2014-001356-31 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party AOP Orphan Pharmaceuticals AG
Study Sponsor  ICMJE AOP Orphan Pharmaceuticals AG
Collaborators  ICMJE PharmaEssentia Corporation (Co-Sponsor for USA)
Investigators  ICMJE
Principal Investigator: Heinz Gisslinger, MD Med Uni Wien
PRS Account AOP Orphan Pharmaceuticals AG
Verification Date February 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP