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

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

Study Overview

• Status: Active, not recruiting
• Conditions: Essential Thrombocythemia
• Intervention / Treatment: Drug: Ropeginterferon alfa-2b-njft (P1101)

Detailed 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.

• Study Type: Interventional
• Enrollment (Actual): 91
• Phase: Phase 2

Contacts and Locations

Study Locations

• Canada
  • Alberta
    • Calgary, Alberta, Canada, T2N 4N2
      • University of Calgary Tom Baker Cancer Centre
  • British Columbia
    • Vancouver, British Columbia, Canada
      • St. Paul's Hospital - Providence Health Care
  • Ontario
    • Hamilton, Ontario, Canada
      • Juravinski Cancer Centre
    • Toronto, Ontario, Canada
      • Princess Margaret Hospital
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35294
      • University of Alabama at Birmingham
  • California
    • Duarte, California, United States, 91010
      • City of Hope National Medical Center
    • Greenbrae, California, United States, 94904
      • Marin Cancer Care
    • Los Angeles, California, United States, 90033
      • Usc Norris Comprehensive Cancer Center
  • Connecticut
    • New Haven, Connecticut, United States, 06510
      • Yale University School of Medicine - Yale Cancer Center
  • District of Columbia
    • Washington, District of Columbia, United States, 20057
      • Georgetown University Medical Center
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • The Winship Cancer Institute Emory University
  • Indiana
    • Fort Wayne, Indiana, United States, 46804
      • Fort Wayne Medical Oncology and Hematology
  • Kentucky
    • Paducah, Kentucky, United States, 42003
      • Mercy Health - Paducah Medical Oncology and Hematology
  • Louisiana
    • New Orleans, Louisiana, United States, 70112
      • Tulane University Medical Center
  • Maryland
    • Baltimore, Maryland, United States, 21204
      • Greater Baltimore Medical Center
  • Massachusetts
    • Boston, Massachusetts, United States, 02215
      • Dana-Farber Cancer Institute
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital
  • Missouri
    • Saint Louis, Missouri, United States, 63110
      • Washington University School of Medicine - Division of Oncology
  • Nevada
    • Reno, Nevada, United States, 89511
      • Cancer Care Specialists
  • New Jersey
    • East Brunswick, New Jersey, United States, 08816
      • Astera HealthCare
    • Hackensack, New Jersey, United States, 07601
      • John Theurer Cancer Center at Hackensack UMC
  • New York
    • Bronx, New York, United States, 10467
      • Montefiore Medical Center
    • New York, New York, United States, 10065
      • Memorial Sloan Kettering Cancer Center
    • New York, New York, United States, 10021
      • Weill Medical College of Cornell University
    • Stony Brook, New York, United States, 11794
      • Stony Brook University Medical Center
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27514
      • University of North Carolina (UNC) - Lineberger Comprehensive Cancer Center
    • Durham, North Carolina, United States, 27710
      • Duke University Medical Center
    • Greenville, North Carolina, United States, 27858
      • East Carolina University
    • Wilson, North Carolina, United States, 27893
      • Regional Medical Oncology Center
  • Tennessee
    • Memphis, Tennessee, United States, 38103
      • University of Tennessee Health Science Center
  • Texas
    • Houston, Texas, United States, 77030
      • MD Anderson Cancer Center
  • Utah
    • Salt Lake City, Utah, United States, 84132
      • University of Utah
  • Virginia
    • Charlottesville, Virginia, United States, 22903
      • University of Virginia - Emily Couric Cancer Center
  • Washington
    • Seattle, Washington, United States, 98109
      • Fred Hutchinson Cancer Research Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Male and female subjects ≥18 years old.
2. Subjects diagnosed with ET according to the World Health Organization (WHO) 2016 criteria.

3. 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.

4. 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.
5. Creatinine clearance ≥40 mL/min (by Cockcroft-Gault equation).
6. 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.
7. Written informed consent obtained from the subject and ability for the subject to comply with the requirements of the study.
8. Platelet count >450 × 109/L at screening
9. Both ANA-naïve and ANA-pretreated subjects are eligible for the study, regardless of the reason to terminate ANA use

Exclusion Criteria:

1. Any subject requiring a legally authorized representative
2. Subjects who stopped prior interferon alfa therapy due to low efficacy or poor tolerability
3. Any contraindications or hypersensitivity to IFN-α and/or its excipients
4. 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
5. History of major organ transplantation
6. Pregnant or lactating females

7. 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:

   1. 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)
   2. 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
   3. Infections with systemic manifestations (e.g., bacterial, fungal, or human immunodeficiency virus [HIV], except hepatitis B [HBV] and/or hepatitis C [HCV],at screening)
   4. Evidence of severe retinopathy (e.g., cytomegalovirus retinitis [CMV], macular degeneration) or clinically relevant ophthalmological disorder (due to diabetes mellitus or hypertension)
   5. History or presence of clinically relevant depression
   6. Previous suicide attempts or at any risk of suicide at screening, in the judgment of the Investigator
   7. History or presence of clinically significant neurologic diseases
   8. 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)
   9. History of alcohol or drug abuse within the last year
   10. History or evidence of any other MPN
8. 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
9. Presence of more than one driver mutation (e.g., V617F JAK2 and CALR, CALR and MPL, V617F JAK2 and MPL)
10. Prior use of JAK inhibitors

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Ropeginterferon alfa-2b (P1101); Pre-filled Syringe, Q2W, SC injection	Drug: Ropeginterferon alfa-2b-njft (P1101); Ropeginterferon alfa-2b-njft (P1101); Other Names: P1101

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To assess efficacy of ropeginterferon alfa-2b-njft (P1101) in adult USA/Canadian patients with ET	Efficacy will be based on peripheral blood count remission as defined by hematocrit (HCT) <45%, white blood cell (WBC) count ≤10 × 109/L, and platelets (PLT) ≤ 400 × 109/L in at least 80% of bi-weekly measurements for a consecutive 32-wek period during the 52-week core study treatment period.	12 months

Collaborators and Investigators

• Sponsor: PharmaEssentia
• Investigators: Study Director: Oleh Zagrijtschuk, MD, PhD, PharmaEssentia Corporation

Study record dates

Study Major Dates

• Study Start (Actual): September 29, 2022
• Primary Completion (Actual): May 9, 2025
• Study Completion (Estimated): March 31, 2027

Study Registration Dates

• First Submitted: July 28, 2022
• First Submitted That Met QC Criteria: July 28, 2022
• First Posted (Actual): August 1, 2022

Study Record Updates

• Last Update Posted (Actual): August 13, 2025
• Last Update Submitted That Met QC Criteria: August 8, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Keywords: MPN; Myeloproliferative; Neoplasms; Myeloproliferative Neoplasms; Essential; Thrombocythemia; Besremi; Ropeginterferon Alfa-2b-njft; P1101; Essential Thrombocythemia
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders; Bone Marrow Diseases; Hemorrhagic Disorders; Blood Platelet Disorders; Myeloproliferative Disorders; Thrombocytosis; Thrombocythemia, Essential
• Other Study ID Numbers: EXCEED ET / A22-301

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No