An open-label, multicenter, phase IIIb study assessing the long-term efficacy and safety of AOP2014 and standard first line treatment (BAT) in patients with Polycythemia Vera who previously participated in the PROUD-PV Study

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 200

Inclusion Criteria

1. Patients who completed the PROUD-PV Study:
a. normalization of at least two out of three main blood parameters (Hct, PLTs and WBCs) if these parameters were moderately increased (Hct<50%, WBC<20 x 109/L, PLTs<600 x 109/L) at baseline of the PROUD-PV Study, OR
b. >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 of the PROUD-PV Study, OR
c. normalization of spleen size, if spleen was enlarged at baseline of the PROUD-PV Study, OR
d. otherwise a clear, medically verified benefit from treatment (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 140
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 60

Exclusion Criteria

1. Non-recovery from the treatment related toxicities to the grade (usually, Grade I) which allows continuation of the treatment.
2. HADS score of 11 or higher on either or both of the subscales, and /or development or worsening of the 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.
the study.
5. AOP2014 arm only: Loss of efficacy of study treatment or any comparable situation where no further benefits of treatment continuation are expected by the investigator.

Sub-study 2 (ongoing): Evaluation of PK of AOP2014 the CONTINUATION-PV Study
a) AOP2014 PK characterisation in steady state (PK group 1 and 2)
For the estimation of the systemic exposure of AOP2014, blood samples will be collected in the dosing interval between two AOP2014 administrations (i.e. 2-weekly and 4-weekly). Patients within the CONTINUATION-PV Study are eligible for blood sampling who
• are treated with AOP2014 with the pre-filled pen (applicable for all dose levels), and
• are receiving AOP2014 in a 2-weekly (PK group 1) or 4-weekly interval (PK group 2), and
• having received at least 4 stable and consecutive (no dose interruption within 2 months prior entry in sub-study) AOP2014 doses with the same type of pen (250 µg or 500 µg) and
• are giving consent.

b) Estimation of the AOP2014 terminal elimination half-life (PK group 3)
Patients within the CONTINUATION-PV Study are eligible for blood sampling at the last AOP2014 administration until elimination to unquantifiable levels who
• have a temporally interruption of the AOP2014 treatment long enough to participate in the PK sub-study (up to 56 days [+/- 2 days])
• do not directly continue with AOP2014 (or another interferon) therapy after study end, or
• drop-out from the study due to reasons not related to safety and who decided to not continue with AOP2014/IFN therapy, or
• drop-out from the study due to safety reasons where the treating physician decided that blood sampling is feasible and where the patients decided to not continue with AOP2014/interferon therapy, and
• are giving consent.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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