Myeloproliferative Neoplasms Clinical Trial
A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of P1101 in Adults With ET
A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of Ropeginterferon alfa-2b-njft (P1101) in Adult Patients with Essential Thrombocythemia
PharmaEssentia is developing a pegylated (PEG) IFN-α product, P1101, for the treatment of Essential Thrombocythemia (ET) as lack of disease modifying therapies in essential ET constitutes a serious issue in modern hematology.
Ropeginterferonalfa-2b-njft (P1101) may represent an effective, well-tolerated treatment with the ability to provide a deeper response and superior control of important blood parameters with the potential to alter the course of the disease and prevent progression to post-ET myelofibrosis (MF) and/or secondary acute myeloid leukemia (sAML). Ropeginterferon alfa-2b-njft (P1101) is currently being evaluated in comparison to ANA in the ongoing global Phase 3 clinical study, SURPASS ET..
Enrolled patients will receive P1101 over 13 months followed by an extension period.
Male and female subjects ≥18 years old.
Subjects diagnosed with ET according to the World Health Organization (WHO) 2016 criteria.
Subjects that are cytoreductive treatment-naïve, or pre-exposed to HU and/or ANA, as specified below (according to Investigator's judgment and documented in the patient's medical record):
Adequate hepatic function defined as bilirubin ≤1.5 × upper limit normal (ULN), prothrombin time (PT) (international normalized ratio, [INR]) ≤1.5 x ULN, albumin >3.5 g/dL, alanine aminotransferase (ALT) ≤2.0 x ULN, aspartate aminotransferase ≤2.0 x ULN at screening.
Creatinine clearance ≥40 mL/min (by Cockcroft-Gault equation).
Males and females of childbearing potential, as well as all women <2 years after the onset of menopause, must agree to use an acceptable form birth control until 60 days following last dose study drug, and females not breastfeed during study.
Written informed consent obtained from the subject and ability for the subject to comply with the requirements of the study.
Platelet count >450 × 109/L at screening
Any subject requiring a legally authorized representative
Any contraindications or hypersensitivity to IFN-α and/or its excipients
Co-morbidity with severe or serious condition that, in the Investigator's opinion, would jeopardize the safety of the subject or their compliance with the protocol, including significant cardiac disease (including New York Heart Association Class III-IV congestive heart failure and clinically significant arrhythmias) and pulmonary hypertension
History of major organ transplantation
Pregnant or lactating females
Subjects with any significant medical conditions that, in the opinion of the Investigator, would compromise the results of the study or may impair compliance with the requirements of the protocol, including but not limited to:
Use of any investigational drug <4 weeks prior to the first dose of study drug or not recovered from effects administration any investigational agent
Presence of more than one driver mutation (e.g., V617F JAK2 and CALR, CALR and MPL, V617F JAK2 and MPL)
Prior use of Ruxolitinib
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