Efficacy and Safety of Ropeginterferon Alfa 2b (P1101) for Patients With Polycythemia Vera

NCT06290765 | Not Yet Recruiting Phase 4

• 1 other identifier: A23-401
• Study Type: interventional
• Target: 70
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is a randomized, open-label, multicenter, two-arm study to assess the efficacy and safety of ropeginterferon alfa-2b for patients with PV. The entire study period is 60 weeks, including a main treatment phase (32 weeks), an extension treatment phase (24 weeks), and a safety follow-up phase (four weeks). However, the study may be extended for additional period of treatment after Week 60 pending the primary endpoint analysis at Week 32. Approximately 70 patients with PV will be enrolled.

Conditions

Polycythemia Vera; Myeloproliferative Neoplasm

Keywords

PV; MPNs; Ropeginterferon alfa-2b; P1101; Hct; Phlebotomy; JAK2

Outcome Measures

Primary Outcomes (1)

• The proportion of patients whose Hct is maintained without phlebotomy eligibility from Week 20 through Week 32.

  Phlebotomy eligibility is defined as either: a confirmed Hct ≥45% that is at least 3% higher than the baseline Hct, a confirmed Hct ≥48%, OR a confirmed Hct ≥45% according to the standard of care for phlebotomy at the institution regardless of the magnitude of the increase compared with the baseline.

  From Week 20 through Week 32

Study Arms (2)

Ropeginterferon alfa-2b group

EXPERIMENTAL

Ropeginterferon alfa-2b subcutaneously (SC) every two weeks (± 3 days), target optimal dose of 500 µg.

Drug: Ropeginterferon alfa-2b; Procedure: Phlebotomy and aspirin

Control group

OTHER

Patients will continuously receive the same therapy as s/he received for PV indication prior to screening.

Procedure: Phlebotomy and aspirin

Interventions

Ropeginterferon alfa-2b DRUG

Ropeginterferon alfa-2b subcutaneously (SC) every two weeks (± 3 days), 250 µg at Day 1, 350 µg at Week 2, and target optimal dose of 500 µg at Week 4. Phlebotomy should be conducted if confirmed Hct ≥48%, or confirmed Hct ≥45% that is ≥3% higher than baseline Hct value.

Also known as: P1101

Ropeginterferon alfa-2b group

Phlebotomy and aspirin PROCEDURE

Phlebotomy should be conducted if confirmed Hct ≥48%, or confirmed Hct ≥45% that is ≥3% higher than baseline Hct value, or confirmed Hct ≥45% according to the standard of care for phlebotomy at the institution regardless of the magnitude of the increase compared with the baseline. The same standard or criteria for phlebotomy eligibility should be applied for patients during the study at each study site or institution.

Also known as: Phlebotomy and aspirin plus the other cytoreductive agents

Control group; Ropeginterferon alfa-2b group

Eligibility Criteria

• Age: 18 Years - 59 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Age ≥18 years at the time of informed consent (or other age required by local regulations);
• PV according to the World Health Organization (WHO) 2016 or 2022 Criteria;
• At least 3 phlebotomies within 24 weeks or at least 5 phlebotomies within 52 weeks prior to screening due to inadequate control of Hct value;
• Have the following hematological values immediately prior to randomization at baseline:
• Hematocrit \<45%, and
• WBC ≥4× 109/L, and
• Platelets ≥100 × 109/L;
• Eastern Cooperative Oncology Group (ECOG) performance status 0, 1 or 2.
• Patients receiving cytoreductive therapy must be on a stable dose or minimal dose adjustments for at least 24 weeks before screening and with no planned dose change;
• Patients who are not receiving cytoreductive therapy must have been discontinued from any prior cytoreductive therapy for at least 24 weeks before screening and have recovered from any adverse events;
• 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;
• Written informed consent obtained from the patient or the patient's legal representative, and ability for the patient to comply with the study requirements.

You may not qualify if:

• Patients requiring phlebotomy at Hct levels ˂45% according to Investigator judgement;
• Clinically significant thrombosis (e.g., deep vein thrombosis or splenic vein thrombosis) or PV-related bleeding within 2 months prior to randomization;
• Post-PV myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) (Tefferi et al 2013, Barosi et al 2008);
• Contraindication to pegylated interferon or its excipients;
• known resistance or intolerance to interferon based therapies, as judged by Investigator;
• Documented autoimmune disease (e.g., thyroid dysfunction, idiopathic thrombocytopenic purpura (ITP), scleroderma, psoriasis, or any arthritis of autoimmune origin). Patients with well-managed thyroid disease by oral hormonal replacement therapy could be enrolled;
• Pulmonary infiltrates, pneumonia, and pneumonitis at screening that, in the Investigator's opinion, would jeopardize the safety or compliance with the protocol;
• Infections with systemic manifestations, e.g., bacterial, fungal, or human immunodeficiency virus (HIV), except inactive carriers of hepatitis B (HBV) and/or hepatitis C (HCV) at screening; inactive HBV carrier is defined as the presence of HBsAg and anti-Hepatitis B e-antigen (anti-HBe) antibody, HBV DNA ˂2000 IU/ml, and normal ALT (Invernizz et al 2016); inactive HCV carrier is defined as the presence of HCV RNA but has normal ALT or with no clinically significant symptom as judged by investigator;
• Any investigational drug less than 6 weeks prior to randomization or not recovered from the effects of prior administration of any investigational agent;
• History or presence of depression requiring treatment with antidepressant;
• Previous suicide attempts or at any risk of suicide at screening, in the judgement of the Investigator;
• Any significant morbidity or abnormality which may interfere with the study participation;
• Pregnant or lactating females;
• History of alcohol abuse or drug abuse within the last year;
• Evidence of severe retinopathy (e.g. cytomegalovirus retinitis, macular degeneration) or clinically relevant ophthalmological disorder (due to diabetes mellitus or hypertension);

Sponsors & Collaborators

• PharmaEssentia: lead

MeSH Terms

Conditions

Polycythemia Vera; Myeloproliferative Disorders

Interventions

Phlebotomy; Aspirin

Condition Hierarchy (Ancestors)

Bone Marrow Neoplasms; Hematologic Neoplasms; Neoplasms by Site; Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Blood Specimen Collection; Specimen Handling; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Punctures; Therapeutics; Surgical Procedures, Operative; Investigative Techniques; Salicylates; Hydroxybenzoates; Phenols; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals

Central Study Contacts

Sandy Kan

CONTACT

+886-2-26557688; sandy_kan@pharmaessentia.com

Evan Huang

CONTACT

+886-26557688; evan_huang@pharmaessentia.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 26, 2024
• First Posted: March 4, 2024
• Study Start: February 1, 2026
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): June 30, 2027
• Last Updated: July 31, 2025

Record last verified: 2025-07