Polycythemia Vera MedDRA version: 20.0 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
INN or Proposed INN: ropeginterferon alfa-2b
CAS Number: 1335098-50-4
Current Spon
P1101
Concentration unit: µg microgram(s)
Concentration type: equal
Concentration number: 100-
Sponsors
FONDAZIONE PER LA RICERCA OSPEDALE MAGGIORE DI BERGAMO - FROM
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Age 18-60 years 2. Diagnosis of Polycythemia Vera according to WHO 2008 criteria 3. Diagnosis of Polycythemia Vera performed within 3 years prior inclusion in the study and never treated with cytoreductive drugs 4. HCT=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Any previous well documented cardiovascular PV-related events (see Appendix 1 for description) 2. Previous cytoreductive drugs 3. Known hypersensitivity or contraindications to the IMP (Pegylated Proline-Interferon alpha-2b) 4. Previous exposure to a non-pegylated or pegylated interferon a 5. Clinically relevant pulmonary infiltrates, pneumonia, and pneumonitis 6. Systemic infections, e.g. hepatitis B, hepatitis C, or HIV at screening 7. Significant liver (AST or ALT > 2.5 times ULN) or renal disease (creatinine > 2 mg/ml) 8. Presence of any life-threatening condition or of any disease (e.g. cancer) that is likely to significantly shorten life expectancy 9. History of active substance or alcohol abuse within the last year 10. Any condition that in the opinion of the investigator would jeopardize the evaluation of efficacy or safety or be associated with poor adherence to the protocol 11. Pregnant or lactating women and women*/men of childbearing potential who are not using or are not willing to use any effective means of contraception
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess whether the addition of Pegylated Proline-interferon alpha-2b to the best therapeutic current strategy available based on phlebotomies and low dose acetylsalicilic acid (ASA) could improve the efficacy of treatment of patients with PV at low risk in term of control of recommended level of hematocrit < 45%, over a period of 12 months.;Secondary Objective: Comparative evaluation of: •Reduction of the need of phlebotomies •Hematological response •Thrombotic and hemorrhagic events •Proportion of patients with not palpable spleen •Bone marrow histological remission •Molecular response •Quality of life of patients •Rate of assigned treatment withdrawal due to any protocol drug-related toxicity •Adverse events rate ;Primary end point(s): Proportion (%) of responders to assigned treatment strategy defined as patients who maintain the median HCT values <45% during 12 months after treatment in each arm, without progression of disease and no need of any extra-protocol cytoreductive drugs.;Timepoint(s) of evaluation of this end point: 12 montns | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Quality of Life (QoL): • Functional Assessment of Cancer Therapy-Anemia (FACT-An) • Myeloproliferative Neoplasm Symptoms Assessment Form total symptom score (MPN-SAF TSS/MPN10);Timepoint(s) of evaluation of this end point: At baseline (before starting the protocol therapy), every 3 months until the end of the study and at the completion of the study. | — |
Countries
Italy
Contacts
Public ContactUfficio Sperimentazioni FROM
Fondazione per la ricerca Ospedale Maggiore (FROM)
Outcome results
None listed