NCT02523638

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

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: AOP Orphan Pharmaceuticals AG · Industry
• Sex: All
• Age: 18 Years – 99 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Polycythemia Vera

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2015-07-01
• Primary completion: 2015-12-01
• Completion: 2015-12-01
• First posted: 2015-08-14
• Last updated: 2016-02-17

Locations

35 sites across 8 countries: Austria, Bulgaria, Czechia, France, Hungary, Poland, Slovakia, Ukraine