A Study to Assess Efficacy, Safety, and Tolerability of P1101 in Adult Patients With PV

September 22, 2023 updated by: PharmaEssentia

A Phase IIIb, Randomized, Open-Label, Parallel Group, Multicenter Study to Assess Efficacy, Safety, and Tolerability of Two Dosing Regimens of Ropeginterferon Alfa-2b-njft (P1101) in Adult Patients With Polycythemia Vera (PV)

A Study to Assess Efficacy, Safety, and Tolerability of P1101 in Adult Patients with PV

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Polycythemia vera (PV) is the most common type of chronic myeloproliferative neoplasm (MPN), with an annual reported incidence of up to 2.6/100,000. This is a long-term debilitating and life-threatening disease because it is associated with the risk of thrombosis, bleeding, and progression to myelofibrosis (MF) and secondary acute myeloid leukemia (sAML)

Ropeginterferon alfa-2b-njft (P1101), which gained US marketing authorization in November 2021, is the only interferon alfa approved for the treatment of PV.

This study aims to evaluate the efficacy, tolerability, and safety of ropeginterferon alfa-2b-njft (P1101) in US and Canadian PV patients, utilizing an optimized dosing regimen.

Study Type

Interventional

Enrollment (Estimated)

100

Phase

  • Phase 3

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 Locations

  • Canada
    • Alberta
      • Calgary, Alberta, Canada
    • British Columbia
      • Vancouver, British Columbia, Canada
    • Ontario
      • Hamilton, Ontario, Canada
        • Not yet recruiting
        • Juravinski Cancer Center - Hamilton Health Sciences
        • Contact:
          • Christopher Hillis, PhD
          • Phone Number: 905-387-9495
          • Email: hillis@hhsc.ca
      • Ottawa, Ontario, Canada, K1H 8L6
        • Not yet recruiting
        • The Ottawa Hospital
        • Contact:
      • Toronto, Ontario, Canada
      • Toronto, Ontario, Canada, M5B 1W8
  • United States
    • Florida
      • Jacksonville, Florida, United States, 32207
    • Indiana
      • Fort Wayne, Indiana, United States, 46804
        • Recruiting
        • Fort Wayne Medical Oncology and Hematology
        • Contact:
    • Kansas
      • Westwood, Kansas, United States, 66205
        • Recruiting
        • University of Kansas Medical Center
        • Contact:
    • Kentucky
      • Paducah, Kentucky, United States, 42003
        • Recruiting
        • Mercy Health
        • Contact:
    • Louisiana
      • New Orleans, Louisiana, United States, 70112
        • Recruiting
        • Tulane University Medical Center
        • Contact:
    • Maryland
      • Bethesda, Maryland, United States, 20817
        • Recruiting
        • American Oncology Partners of Maryland PA (Center for Cancer & Blood Disorders)
        • Contact:
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Recruiting
        • Washington University School of Medicine
        • Contact:
    • New Jersey
      • East Brunswick, New Jersey, United States, 08816
    • New York
      • Bronx, New York, United States, 10467
      • New York, New York, United States, 10029
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • Not yet recruiting
        • University of North Carolina Lineberger Comprehensive Cancer Center
        • Contact:
      • Greenville, North Carolina, United States, 27834
        • Not yet recruiting
        • East Carolina University
        • Contact:
      • High Point, North Carolina, United States, 27265
    • Tennessee
      • Memphis, Tennessee, United States, 38103
        • Not yet recruiting
        • University of Tennessee Health Science Center
        • Contact:
    • Texas
      • Houston, Texas, United States, 77030
        • Not yet recruiting
        • MD Anderson
        • Contact:
    • Utah
      • Salt Lake City, Utah, United States, 841312
    • Virginia
      • Charlottesville, Virginia, United States, 22903
        • Recruiting
        • University of Virginia - Emily Couric Cancer Center
        • Contact:

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

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Male or female subjects aged ≥18 years at the time of signing the informed consent form
  2. Subjects diagnosed with PV according to the 2008 or 2016 World Health Organization (WHO) criteria
  3. Subjects with good liver function at screening, which is defined as total bilirubin ≤1.5 × upper limit of normal (ULN), international normalized ratio (INR) ≤1.5 × ULN, albumin >3.5 g/dL, alanine aminotransferase (ALT) ≤2.0 × ULN, and aspartate aminotransferase (AST) ≤2.0 × ULN
  4. Hemoglobin (HGB) ≥10 g/dL for females, and HGB ≥11 g/dL for males at screening
  5. Neutrophil count ≥1.5 × 10^9/L at screening
  6. Creatinine clearance rate ≥40 mL/min at screening (according to the Cockcroft-Gault formula)
  7. 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
  8. Written informed consent obtained from the subject and ability for the subject to comply with the requirements of the study

Exclusion Criteria:

  1. Any contraindications to interferon alfa or hypersensitivity to interferon alfa
  2. Subjects who stopped prior to interferon alfa therapy due to low efficacy or poor tolerability
  3. Subjects with severe or serious diseases that the Investigator determines may affect the subject's participation in this study
  4. History of major organ transplantation
  5. Pregnant or breastfeeding women

  6. Subjects with any other diseases that the Investigator determines will affect the study results or may weaken the compliance to protocol, including but not limited to:

    1. Prior or current autoimmune thyroid disease (clinical symptoms of hyper- or hypo-thyroidism), except subjects with controlled thyroid replacement therapy, could be enrolled
    2. Other documented autoimmune diseases (such as hepatitis, immune thrombocytopenia [ITP], scleroderma, psoriasis, or any autoimmune arthritis)
    3. Clinically significant pulmonary infiltration, infectious pneumonia, and non-infectious pneumonia, or a past history of interstitial pneumonia at screening
    4. Active infection with systemic manifestations (e.g., presence of bacteria, fungi, and/or human immunodeficiency virus [HIV] at screening, excluding hepatitis B [HBV] and/or hepatitis C [HCV] at screening)
    5. Evidence of severe retinopathy (e.g., cytomegalovirus [CMV]-induced retinitis, macular degeneration) or clinically significant eye diseases (due to diabetes or hypertension)
    6. History or presence of clinically relevant depression per Investigator's judgment
    7. Previously had suicidal attempts or has any risk for suicidal tendency at screening
    8. Poorly controlled diabetes defined as HbA1c >8.0% for at least 1 year
    9. Active thromboembolic complications caused by PV and abdominal hemorrhage in the active phase
    10. History of any malignancy within 5 years (except adequately treated non-melanoma 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)
    11. History of alcohol or drug abuse in the past year
    12. History or evidence of post-polycythemia vera-myelofibrosis (PPV-MF), essential thrombocythemia, or any non-PV MPN
    13. Presence of blast cells in the peripheral blood in the past 12 weeks
  7. Use any investigational drug <4 weeks prior to the first dose of study drug, or not recovered from effects of prior administration of any investigational drug
  8. Any subject requiring a legally authorized representative

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: P1101 250-350-500mcg
Pre-filled Syringe, Q2W starting at 250-350-500, SC injection
Drug: P1101 (Ropeginterferon alfa-2b-njft)
Ropeginterferon alfa-2b-njft
Active Comparator: Ropeginterferon alfa-2b-njft
Pre-filled Syringe, Q2W starting at 100 up to 500 (50mcg increases), SC injection
Drug: Ropeginterferon alfa-2b-njft (P1101)
Ropeginterferon alfa-2b-njft

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Compare efficacy, safety, and tolerability of P1101 utilizing 250-350-500 mcg compared to the current labeled dosing through assessing the proportion of subjects that are in a complete hematologic response at Week 24.
Time Frame: 24 weeks
CHR is defined as hematocrit (HCT) <45%, white blood cell (WBC) count <10 × 10^9/L, platelets (PLT) ≤400 × 10^9/L in the absence of phlebotomy in the previous 12 weeks.
24 weeks

Collaborators and Investigators

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

Sponsor

PharmaEssentia

Investigators

  • Study Director: Ray Urbanski, MD, PhD, PharmaEssentia USA Corporation

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 (Actual)

October 26, 2022

Primary Completion (Estimated)

January 11, 2024

Study Completion (Estimated)

December 28, 2025

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)

September 25, 2023

Last Update Submitted That Met QC Criteria

September 22, 2023

Last Verified

September 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ECLIPSE PV / A22-203

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

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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