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

July 28, 2025 updated by: PharmaEssentia

Efficacy and Safety of Ropeginterferon Alfa 2b (P1101) for Patients With Polycythemia Vera - A Randomized Open Label Global Multicenter Study (PARADIGM-PV)

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.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

70

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Age ≥18 years at the time of informed consent (or other age required by local regulations);
  2. PV according to the World Health Organization (WHO) 2016 or 2022 Criteria;
  3. 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;

  4. Have the following hematological values immediately prior to randomization at baseline:

    1. Hematocrit <45%, and
    2. WBC ≥4× 109/L, and
    3. Platelets ≥100 × 109/L;
  5. Eastern Cooperative Oncology Group (ECOG) performance status 0, 1 or 2.
  6. 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;
  7. 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;
  8. 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;
  9. Written informed consent obtained from the patient or the patient's legal representative, and ability for the patient to comply with the study requirements.

Exclusion Criteria:

  1. Patients requiring phlebotomy at Hct levels ˂45% according to Investigator judgement;
  2. Clinically significant thrombosis (e.g., deep vein thrombosis or splenic vein thrombosis) or PV-related bleeding within 2 months prior to randomization;
  3. 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);
  4. Contraindication to pegylated interferon or its excipients;
  5. known resistance or intolerance to interferon based therapies, as judged by Investigator;
  6. 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;
  7. Pulmonary infiltrates, pneumonia, and pneumonitis at screening that, in the Investigator's opinion, would jeopardize the safety or compliance with the protocol;
  8. 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;
  9. Any investigational drug less than 6 weeks prior to randomization or not recovered from the effects of prior administration of any investigational agent;
  10. History or presence of depression requiring treatment with antidepressant;
  11. Previous suicide attempts or at any risk of suicide at screening, in the judgement of the Investigator;
  12. Any significant morbidity or abnormality which may interfere with the study participation;
  13. Pregnant or lactating females;
  14. History of alcohol abuse or drug abuse within the last year;
  15. Evidence of severe retinopathy (e.g. cytomegalovirus retinitis, macular degeneration) or clinically relevant ophthalmological disorder (due to diabetes mellitus or hypertension);
  16. Significant liver (AST or ALT > 2.5 times ULN) or renal disease (creatinine > 2 mg/ml);
  17. History of major organ transplantation;
  18. History or presence of clinically significant neurologic diseases, e.g., uncontrolled severe seizure disorder;
  19. History of malignant disease, including solid tumors and hematological malignancies (except basal cell and squamous cell carcinomas of the skin and carcinoma in situ of the cervix that have been completely excised and are considered cured) within the last 3 years.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Ropeginterferon alfa-2b group
Ropeginterferon alfa-2b subcutaneously (SC) every two weeks (± 3 days), target optimal dose of 500 µg.
Drug: Ropeginterferon alfa-2b

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.

Other Names:
  • P1101
Procedure: Phlebotomy and aspirin
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.
Other Names:
  • Phlebotomy and aspirin plus the other cytoreductive agents
Other: Control group
Patients will continuously receive the same therapy as s/he received for PV indication prior to screening.
Procedure: Phlebotomy and aspirin
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.
Other Names:
  • Phlebotomy and aspirin plus the other cytoreductive agents

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The proportion of patients whose Hct is maintained without phlebotomy eligibility from Week 20 through Week 32.
Time Frame: 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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

PharmaEssentia

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

February 1, 2026

Primary Completion (Estimated)

December 31, 2026

Study Completion (Estimated)

June 30, 2027

Study Registration Dates

First Submitted

January 26, 2024

First Submitted That Met QC Criteria

February 29, 2024

First Posted (Actual)

March 4, 2024

Study Record Updates

Last Update Posted (Actual)

July 31, 2025

Last Update Submitted That Met QC Criteria

July 28, 2025

Last Verified

July 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • A23-401

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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