Trial Title:
A Study to Assess Efficacy, Safety, and Tolerability of P1101 in Adult Patients With PV
NCT ID:
NCT05481151
Condition:
Polycythemia Vera
Conditions: Official terms:
Polycythemia Vera
Polycythemia
Conditions: Keywords:
Polycythemia Vera
Essential Thrombocythemia
P1101
Ropeginterferonalfa-2b-njft
Polycythemia
Vera
PharmaEssentia
Besremi
Ropeg
Study type:
Interventional
Study phase:
Phase 3
Overall status:
Active, not recruiting
Study design:
Allocation:
Randomized
Intervention model:
Parallel Assignment
Primary purpose:
Treatment
Masking:
None (Open Label)
Intervention:
Intervention type:
Drug
Intervention name:
P1101 (Ropeginterferon alfa-2b-njft)
Description:
Ropeginterferon alfa-2b-njft
Arm group label:
P1101 250-350-500mcg
Intervention type:
Drug
Intervention name:
Ropeginterferon alfa-2b-njft (P1101)
Description:
Ropeginterferon alfa-2b-njft
Arm group label:
Ropeginterferon alfa-2b-njft
Summary:
A Study to Assess Efficacy, Safety, and Tolerability of P1101 in Adult Patients with PV
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.
Criteria for eligibility:
Criteria:
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
Gender:
All
Minimum age:
18 Years
Maximum age:
N/A
Healthy volunteers:
No
Locations:
Facility:
Name:
Baptist MD Anderson
Address:
City:
Jacksonville
Zip:
32207
Country:
United States
Facility:
Name:
Fort Wayne Medical Oncology and Hematology
Address:
City:
Fort Wayne
Zip:
46804
Country:
United States
Facility:
Name:
University of Kansas Medical Center
Address:
City:
Westwood
Zip:
66205
Country:
United States
Facility:
Name:
Mercy Health
Address:
City:
Paducah
Zip:
42003
Country:
United States
Facility:
Name:
Tulane University Medical Center
Address:
City:
New Orleans
Zip:
70112
Country:
United States
Facility:
Name:
American Oncology Partners of Maryland PA (Center for Cancer & Blood Disorders)
Address:
City:
Bethesda
Zip:
20817
Country:
United States
Facility:
Name:
Washington University School of Medicine
Address:
City:
Saint Louis
Zip:
63110
Country:
United States
Facility:
Name:
Astera HealthCare
Address:
City:
East Brunswick
Zip:
08816
Country:
United States
Facility:
Name:
Montefiore Medical Center
Address:
City:
Bronx
Zip:
10467
Country:
United States
Facility:
Name:
Mount Sinai
Address:
City:
New York
Zip:
10029
Country:
United States
Facility:
Name:
University of North Carolina Lineberger Comprehensive Cancer Center
Address:
City:
Chapel Hill
Zip:
27599
Country:
United States
Facility:
Name:
East Carolina University
Address:
City:
Greenville
Zip:
27834
Country:
United States
Facility:
Name:
Wake Forest Baptist Medical Center
Address:
City:
High Point
Zip:
27265
Country:
United States
Facility:
Name:
University of Tennessee Health Science Center
Address:
City:
Memphis
Zip:
38103
Country:
United States
Facility:
Name:
MD Anderson
Address:
City:
Houston
Zip:
77030
Country:
United States
Facility:
Name:
University of Utah
Address:
City:
Salt Lake City
Zip:
841312
Country:
United States
Facility:
Name:
University of Virginia - Emily Couric Cancer Center
Address:
City:
Charlottesville
Zip:
22903
Country:
United States
Facility:
Name:
Tom Baker Cancer Centre
Address:
City:
Calgary
Country:
Canada
Facility:
Name:
St. Paul's Hospital
Address:
City:
Vancouver
Country:
Canada
Facility:
Name:
Juravinski Cancer Center - Hamilton Health Sciences
Address:
City:
Hamilton
Country:
Canada
Facility:
Name:
The Ottawa Hospital
Address:
City:
Ottawa
Zip:
K1H 8L6
Country:
Canada
Facility:
Name:
St. Michael's Hospital
Address:
City:
Toronto
Zip:
M5B 1W8
Country:
Canada
Facility:
Name:
Princess Margaret Hospital
Address:
City:
Toronto
Country:
Canada
Start date:
October 26, 2022
Completion date:
July 31, 2025
Lead sponsor:
Agency:
PharmaEssentia
Agency class:
Industry
Source:
PharmaEssentia
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT05481151