A randomized study comparing two drugs: hydroxyurea and Pegylated Interferon Alfa-2a, in an open label clinical trial in two independent diseases: (1) high risk polycythaemia vera and (2) high risk essential thrombocythaemia.

Phase 1

• Conditions: The myeloproliferative disorders essential thrombocythaemia (ET) and polycythaemia vera (PV).; MedDRA version: 14.1Level: LLTClassification code 10043550Term: ThrombocythemiaSystem Organ Class: 100000004851; MedDRA version: 14.1Level: LLTClassification code 10036058Term: PolycythemiaSystem Organ Class: 100000004851; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2010-019501-41-GB
• Lead Sponsor: Consorzio Mario Negri Sud

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2012-11-14
• Last Update Posted: 20 July 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 612

Inclusion Criteria

- A diagnosis (<3 years prior to entry in the sudy) of Essential Thrombocythaemia (ET) or Polycythaemia Vera (PV) made in accordance with the WHO (2008) criteria. - Never treated with cytoreductive drugs except hydroxyurea for up to 3 months maximum (phlebotomy, aspirin allowed, anagrelide allowed). - Age: > 18 years (no upper limit). - Ability and willingness to comply with all study requirements. - Signed informed consent to participate in this study. - Willing to participate in associated correlative science biomarker study. - Serum creatinine < 1.5 x upper limit of normal. - AST and ALT < 2 x upper limit of normal. - Total Bilirubin within normal limits. - No known PNH (paroxysmal nocturnal hemoglobinuria) clone. - No concurrent hormonal oral contraceptive use. - Patients must have high risk disease as defined below: High risk PV ANY ONE of the following: 1)age >60 years; 2)Previous documented thrombosis, erythromelalgia or migraine (severe, recurrent, requiring medications, and felt to be secondary to the MPN) either after diagnosis or within 10 years before diagnosis and considered to be disease related; 3)significant splenomegaly (>5 cm below the left costal margin) or symptomatic splenomegaly (splenic infarcts or requiring analgesia); 4)platelets > 1000 x 109/L; 5)diabetes or hypertension requiring pharmacological therapy for > 6 months. High risk ET ANY ONE of the following: 1)age > 60 years; 2)platelet count > 1500 x 109/L; 3)previous documented thrombosis, erythromelalgia or migraine headaches (severe, recurrent, requiring medications, and felt to be secondary to the MPN) either after diagnosis or within 10 years before diagnosis and considered to be disease related; 4)previous hemorrhage related to ET; 5)Diabetes or hypertension requiring pharmacological therapy for > 6 months.
Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 32
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 32

Exclusion Criteria

- Any contraindications to Pegylated interferon or Hydroxyurea (listed below). - Presence of any life-threatening co-morbidity. - History of active substance or alcohol abuse within the last year. - Subjects who are pregnant, lactating or of reproductive potential and not practicing an effective means of contraception. - History of psychiatric disorder (e.g. depression). - History of autoimmune disorder (e.g. hepatitis). - Hypersensitivity to IFN-a HIV, HBV, or systemic infection. - Evidence of severe retinopathy (e.g. CMV retinitis, macular degeneration) or clinically relevant ophthalmological disorder (e.g. due to diabetes mellitus or hypertension). - History or other evidence of decompensated liver disease. - Splanchnic vein thrombosis (includes Budd-Chiari, portal vein, splenic and mesenteric thrombosis). - History or other evidence of chronic pulmonary disease associated with functional limitation. - Thyroid dysfunction not adequately controlled. - Any investigational drug <6 weeks prior to the first dose of study drug. - Neutrophil count <1.5 x 109/L. - JAK2 exon 12 mutation. - Patients should not meet criteria for post PV or post ET-MF - No previous exposure to any formulation of pegylated interferon. - Subjects with any other medical condition, which in the opinion of the investigator would compromise the results of the study by deleterious effects of treatment.

Study & Design

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