A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of P1101 in Adults With ET
Hematopoietic Malignancies
Leukemia
Unknown Primary
18 Years and older, Male and Female
EXCEED ET / A22-301 (primary)
NCI-2023-00605
Summary
A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of
Ropeginterferon alfa-2b-njft (P1101) in Adult Patients with Essential Thrombocythemia
Objectives
PharmaEssentia is developing a pegylated (PEG) IFN-a 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.
Eligibility
- 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
Treatment Sites in Georgia
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