A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of P1101 in Adults With ET

Running, not enrolling · Phase 2

Conditions studied: Essential Thrombocythemia

In brief

A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of Ropeginterferon alfa-2b-njft (P1101) in Adult Patients with Essential Thrombocythemia

Key facts

Study ID

NCT05482971

Run by

PharmaEssentia

People needed

91

Starts

2022-09-29

Expected to finish

2027-03-31

Last updated by the study team

2025-08-13

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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:
• Documented autoimmune disease at screening or in the history (e.g., thyroid dysfunction, hepatitis, idiopathic thrombocytopenic purpura, scleroderma, psoriasis, or any arthritis of autoimmune origin)
• Clinically relevant pulmonary infiltrates, pneumonia, and pneumonitis at screening that, in the Investigator's opinion, would jeopardize the safety of the subject or their compliance with the protocol
• Infections with systemic manifestations (e.g., bacterial, fungal, or human immunodeficiency virus [HIV], except hepatitis B [HBV] and/or hepatitis C [HCV],at screening)
• Evidence of severe retinopathy (e.g., cytomegalovirus retinitis [CMV], macular degeneration) or clinically relevant ophthalmological disorder (due to diabetes mellitus or hypertension)
• History or presence of clinically relevant depression
• Previous suicide attempts or at any risk of suicide at screening, in the judgment of the Investigator
• History or presence of clinically significant neurologic diseases
• History of any malignancy within 5 years (except adequately treated nonmelanoma skin cancer, prostate cancer status post resection with an undetectable prostate-specific antigen [PSA], curative treated in-situ cancer of the cervix, ductal carcinoma in-situ [DCIS] of the breast, Stage 1 Grade 1 endometrial carcinoma, or other solid tumors including lymphomas [without bone marrow involvement] curatively treated with no evidence of disease for ≥2 years prior to study)
• History of alcohol or drug abuse within the last year
• History or evidence of any other MPN
• 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

Where it is running

• University of Alabama at Birmingham — Birmingham, Alabama, United States
• City of Hope National Medical Center — Duarte, California, United States
• Marin Cancer Care — Greenbrae, California, United States
• USC Norris Comprehensive Cancer Center — Los Angeles, California, United States
• Yale University School of Medicine - Yale Cancer Center — New Haven, Connecticut, United States
• Georgetown University Medical Center — Washington D.C., District of Columbia, United States
• The Winship Cancer Institute Emory University — Atlanta, Georgia, United States
• Fort Wayne Medical Oncology and Hematology — Fort Wayne, Indiana, United States
• Mercy Health - Paducah Medical Oncology and Hematology — Paducah, Kentucky, United States
• Tulane University Medical Center — New Orleans, Louisiana, United States
• Greater Baltimore Medical Center — Baltimore, Maryland, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Dana-Farber Cancer Institute — Boston, Massachusetts, United States
• Washington University School of Medicine - Division of Oncology — St Louis, Missouri, United States
• Cancer Care Specialists — Reno, Nevada, United States
• Astera HealthCare — East Brunswick, New Jersey, United States
• John Theurer Cancer Center At Hackensack UMC — Hackensack, New Jersey, United States
• Weill Medical College of Cornell University — New York, New York, United States
• Memorial Sloan Kettering Cancer Center — New York, New York, United States
• Stony Brook University Medical Center — Stony Brook, New York, United States
• Montefiore Medical Center — The Bronx, New York, United States
• University of North Carolina (UNC) - Lineberger Comprehensive Cancer Center — Chapel Hill, North Carolina, United States
• Duke University Medical Center — Durham, North Carolina, United States
• East Carolina University — Greenville, North Carolina, United States
• Regional Medical Oncology Center — Wilson, North Carolina, United States

Full record on ClinicalTrials.gov

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