An open-label, multicenter, phase IIIb study assessing the long-term efficacy and safety of AOP2014 in patients with Polycythemia Vera who previously participated in the PROUD-PV study

Phase 1

• Conditions: Polycythemia Vera; MedDRA version: 17.1Level: LLTClassification code 10036061Term: Polycythemia veraSystem Organ Class: 100000004864; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2014-001357-17-ES
• Lead Sponsor: AOP Orphan Pharmaceuticals AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2014-12-16
• Last Update Posted: 17 January 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 130

Inclusion Criteria

1. Patients who completed the 12 months AOP2014 treatment arm of the PROUD-PV study and at the end-of-treatment visit (EoT) of the PROUD-PV study who 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 (Hct<50%, WBC<20 x 10(9)/L, PLTs<600 x 10(9)/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 10(9)/L, PLTs>600 x 10(9)/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 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 90
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 40

Exclusion Criteria

1. Non-recovery from the AOP2014 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.
5. Loss of efficacy of AOP2014 or any comparable situation where no further benefits of treatment continuation are expected by the investigator.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the long-term efficacy of AOP2014 in terms of disease response rate in patients diagnosed with PV, who were previously treated with AOP2014 in the PROUD-PV study and who completed this study.;Secondary Objective: To further assess the long-term efficacy, safety, quality of life (QoL) and change of JAK2 allelic burden in patients diagnosed with PV and previously treated with AOP2014 in the PROUD-PV study.;Primary end point(s): The main efficacy evaluation criterion will be disease response defined as Hct<45% without phlebotomy (at least 3 months since the last phlebotomy), PLTs<400 x 109/L, WBCs<10 x 109/L, and normal spleen size.<br>The main efficacy endpoint will be the maintenance rate of disease response at assessment visits (every three months).;Timepoint(s) of evaluation of this end point: Every three months.