Polycythemia Vera MedDRA version: 17.1 Level: LLT Classification code 10036061 Term: Polycythemia vera System Organ Class: 100000004864
Conditions
Interventions
Product Name: Pegylated proline-interferon alpha-2b
Product Code: AOP2014
Pharmaceutical Form: Solution for injection in pre-filled pen
INN or Proposed INN: PEGylated Proline-interferone alpha-2b reco
P1101
Other descriptive name: PEGINTERFERON ALFA-2B
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 0.5-
Sponsors
AOP Orphan Pharmaceuticals AG
Eligibility
Sex/Gender
All
Inclusion 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 "end-of-treatment visit" (EoT) of the PROUD-PV study or two weeks after the last visit of the CONTINUATION-PV study, fulfill at least one of the following criteria: a. Normalization of at least two out of three main blood parameters (Hct, PLTs and WBCs) if these parameters were moderately increased (Hct35% 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 10 9/L, PLTs>600 x 10 9/L) at baseline visit of the PROUD-PV study, OR c. Normalization of spleen size, if spleen was enlarged at baseline visit of the PROUD-PV study, OR d. Otherwise a clear, medically verified benefit from treatment with AOP2014 (e.g. normalization of disease-related micro-vasculatory symptoms, substantial decrease of JAK2 allelic burden). 2. Signed written ICF. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 22 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 8
Exclusion criteria
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. Hospital Anxiety Depression Scale (HADS) 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the ease of AOP2014 self-administration using dedicated questionnaires;Secondary Objective: - To assess safety and tolerability: adverse events (AEs), laboratory parameters, electrocardiogram (ECG) throughout study. - To assess maintenance of the blood efficacy parameters Hct, WBC and PLTs 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).;Primary end point(s): 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.;Timepoint(s) of evaluation of this end point: every two weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Efficacy Evaluation 1. Disease response rate. 2. Number of phlebotomies performed during the study. 3. Hct, erythrocyte (RBC), WBC and PLT changes comparing values at the assessment visit (P7) vs. values at baseline visit (P1) and changes at every visit. 4. Spleen size at the end of the study vs. baseline visit (P1). Safety evaluation 1. Disease-related symptoms (microvascular disturbances, pruritus, headache), assessed at every visit. 2. AEs, incl. adverse events of special interest (AESI). 3. Laboratory data. 4. Standard 12-lead ECG. 5. Physical examination. 6. Vital signs: heart rate, systolic and diastolic blood pressure. 7. Immunogenicity. 8. Urine.;Timepoint(s) of evaluation of this end point: every two weeks | — |
Countries
Austria, Bulgaria, Czech Republic, France, Hungary, Poland, Slovakia, Ukraine
Contacts
Public ContactClinical Project Manager
AOP Orphan Pharmaceuticals AG
Outcome results
None listed