Trial Title:
A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of P1101 in Adults With ET
NCT ID:
NCT05482971
Condition:
Essential Thrombocythemia
Conditions: Official terms:
Thrombocytosis
Thrombocythemia, Essential
Conditions: Keywords:
MPN
Myeloproliferative
Neoplasms
Myeloproliferative Neoplasms
Essential
Thrombocythemia
Besremi
Ropeginterferon Alfa-2b-njft
P1101
Essential Thrombocythemia
Study type:
Interventional
Study phase:
Phase 2
Overall status:
Active, not recruiting
Study design:
Allocation:
N/A
Intervention model:
Single Group Assignment
Primary purpose:
Treatment
Masking:
None (Open Label)
Intervention:
Intervention type:
Drug
Intervention name:
Ropeginterferon alfa-2b-njft (P1101)
Description:
Ropeginterferon alfa-2b-njft (P1101)
Arm group label:
Ropeginterferon alfa-2b (P1101)
Other name:
P1101
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
Detailed description:
PharmaEssentia is developing a pegylated (PEG) IFN-α product, P1101, for the treatment of
Essential Thrombocythemia (ET) as lack of disease modifying therapies in essential ET
constitutes a serious issue in modern hematology.
Ropeginterferon alfa-2b-njft (P1101) may represent an effective, well-tolerated treatment
with the ability to provide a deeper response and superior control of important blood
parameters with the potential to alter the course of the disease and prevent progression
to post-ET myelofibrosis (MF) and/or secondary acute myeloid leukemia (sAML).
Ropeginterferon alfa-2b-njft (P1101) is currently being evaluated in comparison to ANA in
the ongoing global Phase 3 clinical study, SURPASS ET.
Enrolled patients will receive P1101 over 13 months followed by an extension period.
Criteria for eligibility:
Criteria:
Inclusion Criteria:
1. Male and female subjects ≥18 years old.
2. Subjects diagnosed with ET according to the World Health Organization (WHO) 2016
criteria.
3. 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.
4. 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.
5. Creatinine clearance ≥40 mL/min (by Cockcroft-Gault equation).
6. 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.
7. Written informed consent obtained from the subject and ability for the subject to
comply with the requirements of the study.
8. Platelet count >450 × 109/L at screening
9. Both ANA-naïve and ANA-pretreated subjects are eligible for the study, regardless of
the reason to terminate ANA use
Exclusion Criteria:
1. Any subject requiring a legally authorized representative
2. Subjects who stopped prior interferon alfa therapy due to low efficacy or poor
tolerability
3. Any contraindications or hypersensitivity to IFN-α and/or its excipients
4. 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
5. History of major organ transplantation
6. Pregnant or lactating females
7. 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:
1. 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)
2. 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
3. Infections with systemic manifestations (e.g., bacterial, fungal, or human
immunodeficiency virus [HIV], except hepatitis B [HBV] and/or hepatitis C
[HCV],at screening)
4. Evidence of severe retinopathy (e.g., cytomegalovirus retinitis [CMV], macular
degeneration) or clinically relevant ophthalmological disorder (due to diabetes
mellitus or hypertension)
5. History or presence of clinically relevant depression
6. Previous suicide attempts or at any risk of suicide at screening, in the
judgment of the Investigator
7. History or presence of clinically significant neurologic diseases
8. 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)
9. History of alcohol or drug abuse within the last year
10. History or evidence of any other MPN
8. Use of any investigational drug <4 weeks prior to the first dose of study drug or
not recovered from effects of prior administration of any investigational agent
9. Presence of more than one driver mutation (e.g., V617F JAK2 and CALR, CALR and MPL,
V617F JAK2 and MPL)
10. Prior use of JAK inhibitors
Gender:
All
Minimum age:
18 Years
Maximum age:
N/A
Healthy volunteers:
No
Locations:
Facility:
Name:
University of Alabama at Birmingham
Address:
City:
Birmingham
Zip:
35294
Country:
United States
Facility:
Name:
City of Hope National Medical Center
Address:
City:
Duarte
Zip:
91010
Country:
United States
Facility:
Name:
Marin Cancer Care
Address:
City:
Greenbrae
Zip:
94904
Country:
United States
Facility:
Name:
USC Norris Comprehensive Cancer Center
Address:
City:
Los Angeles
Zip:
90033
Country:
United States
Facility:
Name:
Yale University School of Medicine - Yale Cancer Center
Address:
City:
New Haven
Zip:
06510
Country:
United States
Facility:
Name:
Georgetown University Medical Center
Address:
City:
Washington
Zip:
20057
Country:
United States
Facility:
Name:
The Winship Cancer Institute Emory University
Address:
City:
Atlanta
Zip:
30322
Country:
United States
Facility:
Name:
Fort Wayne Medical Oncology and Hematology
Address:
City:
Fort Wayne
Zip:
46804
Country:
United States
Facility:
Name:
Mercy Health - Paducah Medical Oncology and Hematology
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:
Greater Baltimore Medical Center
Address:
City:
Baltimore
Zip:
21204
Country:
United States
Facility:
Name:
Massachusetts General Hospital
Address:
City:
Boston
Zip:
02114
Country:
United States
Facility:
Name:
Dana-Farber Cancer Institute
Address:
City:
Boston
Zip:
02215
Country:
United States
Facility:
Name:
Washington University School of Medicine - Division of Oncology
Address:
City:
Saint Louis
Zip:
63110
Country:
United States
Facility:
Name:
Cancer Care Specialists
Address:
City:
Reno
Zip:
89511
Country:
United States
Facility:
Name:
Astera HealthCare
Address:
City:
East Brunswick
Zip:
08816
Country:
United States
Facility:
Name:
John Theurer Cancer Center At Hackensack UMC
Address:
City:
Hackensack
Zip:
07601
Country:
United States
Facility:
Name:
Montefiore Medical Center
Address:
City:
Bronx
Zip:
10467
Country:
United States
Facility:
Name:
Weill Medical College of Cornell University
Address:
City:
New York
Zip:
10021
Country:
United States
Facility:
Name:
Memorial Sloan Kettering Cancer Center
Address:
City:
New York
Zip:
10065
Country:
United States
Facility:
Name:
Stony Brook University Medical Center
Address:
City:
Stony Brook
Zip:
11794
Country:
United States
Facility:
Name:
University of North Carolina (UNC) - Lineberger Comprehensive Cancer Center
Address:
City:
Chapel Hill
Zip:
27514
Country:
United States
Facility:
Name:
Duke University Medical Center
Address:
City:
Durham
Zip:
27710
Country:
United States
Facility:
Name:
East Carolina University
Address:
City:
Greenville
Zip:
27858
Country:
United States
Facility:
Name:
Regional Medical Oncology Center
Address:
City:
Wilson
Zip:
27893
Country:
United States
Facility:
Name:
University of Tennessee Health Science Center
Address:
City:
Memphis
Zip:
38103
Country:
United States
Facility:
Name:
MD Anderson Cancer Center
Address:
City:
Houston
Zip:
77030
Country:
United States
Facility:
Name:
University of Utah
Address:
City:
Salt Lake City
Zip:
84132
Country:
United States
Facility:
Name:
University of Virginia - Emily Couric Cancer Center
Address:
City:
Charlottesville
Zip:
22903
Country:
United States
Facility:
Name:
Fred Hutchinson Cancer Research Center
Address:
City:
Seattle
Zip:
98109
Country:
United States
Facility:
Name:
University of Calgary Tom Baker Cancer Centre
Address:
City:
Calgary
Zip:
T2N 4N2
Country:
Canada
Facility:
Name:
St. Paul's Hospital - Providence Health Care
Address:
City:
Vancouver
Country:
Canada
Facility:
Name:
Juravinski Cancer Centre
Address:
City:
Hamilton
Country:
Canada
Facility:
Name:
Princess Margaret Hospital
Address:
City:
Toronto
Country:
Canada
Start date:
September 29, 2022
Completion date:
March 31, 2027
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/NCT05482971