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

NCT05482971 | Active Not Recruiting Phase 2 FDA Drug

• 1 other identifier: EXCEED ET / A22-301
• Study Type: interventional
• Target: 91
• Locations: 2 countries
• Sites: 34

Brief 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

Conditions

Essential Thrombocythemia

Keywords

MPN; Myeloproliferative; Neoplasms; Myeloproliferative Neoplasms; Essential; Thrombocythemia; Besremi; Ropeginterferon Alfa-2b-njft; P1101; Essential Thrombocythemia

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (1)

Ropeginterferon alfa-2b (P1101)

EXPERIMENTAL

Pre-filled Syringe, Q2W, SC injection

Drug: Ropeginterferon alfa-2b-njft (P1101)

Interventions

Ropeginterferon alfa-2b-njft (P1101) DRUG

Ropeginterferon alfa-2b-njft (P1101)

Also known as: P1101

Ropeginterferon alfa-2b (P1101)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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.

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)

Sponsors & Collaborators

• PharmaEssentia: lead

Study Sites (34)

University of Alabama at Birmingham

Birmingham, Alabama, 35294, United States

Location

City of Hope National Medical Center

Duarte, California, 91010, United States

Location

Marin Cancer Care

Greenbrae, California, 94904, United States

Location

USC Norris Comprehensive Cancer Center

Los Angeles, California, 90033, United States

Location

Yale University School of Medicine - Yale Cancer Center

New Haven, Connecticut, 06510, United States

Location

Georgetown University Medical Center

Washington D.C., District of Columbia, 20057, United States

Location

The Winship Cancer Institute Emory University

Atlanta, Georgia, 30322, United States

Location

Fort Wayne Medical Oncology and Hematology

Fort Wayne, Indiana, 46804, United States

Location

Mercy Health - Paducah Medical Oncology and Hematology

Paducah, Kentucky, 42003, United States

Location

Tulane University Medical Center

New Orleans, Louisiana, 70112, United States

Location

Greater Baltimore Medical Center

Baltimore, Maryland, 21204, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

Washington University School of Medicine - Division of Oncology

St Louis, Missouri, 63110, United States

Location

Cancer Care Specialists

Reno, Nevada, 89511, United States

Location

Astera HealthCare

East Brunswick, New Jersey, 08816, United States

Location

John Theurer Cancer Center At Hackensack UMC

Hackensack, New Jersey, 07601, United States

Location

Weill Medical College of Cornell University

New York, New York, 10021, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

Stony Brook University Medical Center

Stony Brook, New York, 11794, United States

Location

Montefiore Medical Center

The Bronx, New York, 10467, United States

Location

University of North Carolina (UNC) - Lineberger Comprehensive Cancer Center

Chapel Hill, North Carolina, 27514, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

East Carolina University

Greenville, North Carolina, 27858, United States

Location

Regional Medical Oncology Center

Wilson, North Carolina, 27893, United States

Location

University of Tennessee Health Science Center

Memphis, Tennessee, 38103, United States

Location

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

University of Utah

Salt Lake City, Utah, 84132, United States

Location

University of Virginia - Emily Couric Cancer Center

Charlottesville, Virginia, 22903, United States

Location

Fred Hutchinson Cancer Research Center

Seattle, Washington, 98109, United States

Location

University of Calgary Tom Baker Cancer Centre

Calgary, Alberta, T2N 4N2, Canada

Location

St. Paul's Hospital - Providence Health Care

Vancouver, British Columbia, Canada

Location

Juravinski Cancer Centre

Hamilton, Ontario, Canada

Location

Princess Margaret Hospital

Toronto, Ontario, Canada

Location

Related Publications (1)

• Masarova L, Reeves BN, El Chaer F, Foltz L, Tashi T, Abu-Zeinah G, Lucas J, Halpern AB, Maze D, Qin A, Safah H, Lan F, O'Connell CL, Goel S, Rein L, Fang B, How J, Babu S, Li Z, Cerquozzi S, Oh ST, Hunter AM, Podoltsev N, Vachhani P, Yacoub A, Cunningham JM, Hillis C, Otoukesh S, Zagrijtschuk O, Castro H, Bose P. A multicenter study to assess efficacy, safety, and tolerability of ropeginterferon alfa-2b-njft in patients with essential thrombocythemia in the US and Canada: EXCEED-ET trial. Front Med (Lausanne). 2025 Apr 17;12:1548590. doi: 10.3389/fmed.2025.1548590. eCollection 2025.

  PMID: 40330782 DERIVED

MeSH Terms

Conditions

Thrombocythemia, Essential; Neoplasms; Myeloproliferative Disorders; Thrombocytosis

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Blood Platelet Disorders; Bone Marrow Diseases; Hemorrhagic Disorders

Study Officials

• Oleh Zagrijtschuk, MD, PhD

  PharmaEssentia Corporation

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 28, 2022
• First Posted: August 1, 2022
• Study Start: September 29, 2022
• Primary Completion: May 9, 2025
• Study Completion (Estimated): March 31, 2027
• Last Updated: August 13, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

CA (4); US (30)