AOP2014 vs. BAT in Patients With Polycythemia Vera Who Previously Participated in the PROUD-PV Study.
Keywords
Abstract
Description
This is a Phase III, parallel-arm, open-label continuation of the PROUD-PV study performed in adults diagnosed with Polycythemia Vera (PV). Patients who received AOP2014 in the primary study, PROUD-PV will continue on AOP2014, patients who received HU in the PROUD-PV study will receive best available therapy as selected by the investigator. Only patients who completed PROUD-PV including the end of study visit will be enrolled into this continuation study.
Dates
| Last Verified: | 12/31/2019 |
| First Submitted: | 08/13/2014 |
| Estimated Enrollment Submitted: | 08/13/2014 |
| First Posted: | 08/14/2014 |
| Last Update Submitted: | 01/12/2020 |
| Last Update Posted: | 01/13/2020 |
| Actual Study Start Date: | 10/31/2014 |
| Estimated Primary Completion Date: | 03/31/2021 |
| Estimated Study Completion Date: | 03/31/2021 |
Condition or disease
Intervention/treatment
Drug: Pegylated-Proline-interferon alpha-2b
Drug: Best available therapy (BAT)
Phase
Arm Groups
| Arm | Intervention/treatment |
|---|---|
| Active Comparator: Best available therapy (BAT) Best available therapy, as chosen by the investigator for patients who had been on HU in the 1 Year PROUD-PV study | Drug: Best available therapy (BAT) Best available therapy as selected by the investigator |
| Experimental: Pegylated-Proline-interferon alpha-2b AOP2014 for those patients who had been on AOP2014 in the PROUD-PV study | Drug: Pegylated-Proline-interferon alpha-2b 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. |
Eligibility Criteria
| Ages Eligible for Study | 18 Years To 18 Years |
| Sexes Eligible for Study | All |
| Accepts Healthy Volunteers | Yes |
| Criteria | 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: - 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 - >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 - normalization of spleen size, if spleen was enlarged at baseline 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 allelic burden). 2. Signed written ICF. Exclusion criteria: Withdrawal criteria, as specified in the PROUD-PV study, 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 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. 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. The main efficacy endpoint will be the maintenance rate of disease response at assessment visits (every three months). |
Outcome
Primary Outcome Measures
1. Disease response at quarterly assessment visits [3 years]
