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A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of Ropeginterferon alfa-2b-njft (P1101) in Adult Patients with Essential Thrombocythemia
Full description
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.
Ropeginterferon alfa-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.
Enrollment
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Inclusion criteria
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):
a. Cytoreductive-naïve patients must be in need of cytoreductive treatment, defined as having at least one of the following:
i. Progressive leukocytosis and/or thrombocytosis
ii. Disease-related symptoms (i.e., pruritus, night sweats, fatigue)
iii. Vasomotor/microvascular disturbances, not responsive to aspirin (including headache, chest pain or erythromelalgia, etc.)
iv. High-risk (history of thrombosis at any age; or age >60 years with JAK2 mutation)
b. Patients previously exposed to HU will be classified as either:
i. Documented formal HU resistance or intolerance
ii. HU stopped without documented formal resistance/intolerance due to insufficient blood count control or toxicity. The last HU dose must be >7 days prior the first dose of P1101.
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 of birth control until 60 days following the last dose of the study drug, and females must agree to not breastfeed during the 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
Both ANA-naïve and ANA-pretreated subjects are eligible for the study, regardless of the reason to terminate ANA use
Exclusion criteria
Any subject requiring a legally authorized representative
Subjects who stopped prior interferon alfa therapy due to low efficacy or poor tolerability
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 of prior administration of 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 JAK inhibitors
Primary purpose
Allocation
Interventional model
Masking
64 participants in 1 patient group
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Central trial contact
Jewell Jessup, PhD
Data sourced from clinicaltrials.gov
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