A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of P1101 in Adults With ET
Primary Purpose
Essential Thrombocythemia
Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Ropeginterferon alfa-2b-njft (P1101)
Sponsored by
About this trial
This is an interventional treatment trial for Essential Thrombocythemia focused on measuring MPN, Myeloproliferative, Neoplasms, Myeloproliferative Neoplasms, Essential, Thrombocythemia, Besremi, Ropeginterferon Alfa-2b-njft, P1101, Essential Thrombocythemia
Eligibility Criteria
Inclusion Criteria:
- Male and female subjects ≥18 years old.
- Subjects diagnosed with ET according to the World Health Organization (WHO) 2016 criteria.
- 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):
- Interferon treatment-naïve.
- 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.
- Creatinine clearance ≥40 mL/min (by Cockcroft-Gault equation).
- 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.
- Written informed consent obtained from the subject and ability for the subject to comply with the requirements of the study.
- Platelet count >450 × 109/L at screening
Exclusion Criteria:
- Any subject requiring a legally authorized representative
- Any contraindications or hypersensitivity to IFN-α and/or its excipients
- 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
- History of major organ transplantation
- Pregnant or lactating females
- 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:
- 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
- Presence of more than one driver mutation (e.g., V617F JAK2 and CALR, CALR and MPL, V617F JAK2 and MPL)
- Prior use of Ruxolitinib
Sites / Locations
- University of Alabama at BirminghamRecruiting
- City of Hope National Medical Center
- Marin Cancer CareRecruiting
- USC Norris Comprehensive Cancer Center
- Yale University School of Medicine - Yale Cancer Center
- Georgetown University Medical Center
- The Winship Cancer Institute Emory University
- Fort Wayne Medical Oncology and HematologyRecruiting
- Mercy Health - Paducah Medical Oncology and HematologyRecruiting
- Tulane University Medical CenterRecruiting
- Greater Baltimore Medical CenterRecruiting
- Massachusetts General Hospital
- Dana-Farber Cancer Institute
- Washington University School of Medicine - Division of OncologyRecruiting
- Cancer Care SpecialistsRecruiting
- Astera HealthCareRecruiting
- John Theurer Cancer Center At Hackensack UMC
- Montefiore Medical CenterRecruiting
- Weill Medical College of Cornell University
- Memorial Sloan Kettering Cancer Center
- Stony Brook University Medical CenterRecruiting
- University of North Carolina (UNC) - Lineberger Comprehensive Cancer CenterRecruiting
- Duke University Medical CenterRecruiting
- East Carolina University
- Regional Medical Oncology Center
- University of Tennessee Health Science Center
- MD Anderson Cancer CenterRecruiting
- University of UtahRecruiting
- University of Virginia - Emily Couric Cancer CenterRecruiting
- Fred Hutchinson Cancer Research Center
- University of Calgary Tom Baker Cancer Centre
- St. Paul's Hospital - Providence Health CareRecruiting
- Juravinski Cancer CentreRecruiting
- Princess Margaret Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Ropeginterferon alfa-2b (P1101)
Arm Description
Pre-filled Syringe, Q2W, SC injection
Outcomes
Primary Outcome Measures
To assess efficacy of ropeginterferon alfa-2b-njft (P1101) in adult USA/Canadian patients with ET
Efficacy will be based on peripheral blood count remission as defined by hematocrit (HCT) <45%, white blood cell (WBC) count ≤10 × 109/L, and platelets (PLT) ≤ 400 × 109/L in at least 80% of bi-weekly measurements for a consecutive 32-wek period during the 52-week core study treatment period.
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05482971
Brief Title
A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of P1101 in Adults With ET
Official Title
A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of Ropeginterferon Alfa-2b-njft (P1101) in Adult Patients With Essential Thrombocythemia
Study Type
Interventional
2. Study Status
Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
September 29, 2022 (Actual)
Primary Completion Date
July 29, 2024 (Anticipated)
Study Completion Date
December 31, 2026 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
PharmaEssentia
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
Brief 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Essential Thrombocythemia
Keywords
MPN, Myeloproliferative, Neoplasms, Myeloproliferative Neoplasms, Essential, Thrombocythemia, Besremi, Ropeginterferon Alfa-2b-njft, P1101, Essential Thrombocythemia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
64 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Ropeginterferon alfa-2b (P1101)
Arm Type
Experimental
Arm Description
Pre-filled Syringe, Q2W, SC injection
Intervention Type
Drug
Intervention Name(s)
Ropeginterferon alfa-2b-njft (P1101)
Other Intervention Name(s)
P1101
Intervention Description
Ropeginterferon alfa-2b-njft (P1101)
Primary Outcome Measure Information:
Title
To assess efficacy of ropeginterferon alfa-2b-njft (P1101) in adult USA/Canadian patients with ET
Description
Efficacy will be based on peripheral blood count remission as defined by hematocrit (HCT) <45%, white blood cell (WBC) count ≤10 × 109/L, and platelets (PLT) ≤ 400 × 109/L in at least 80% of bi-weekly measurements for a consecutive 32-wek period during the 52-week core study treatment period.
Time Frame
12 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Male and female subjects ≥18 years old.
Subjects diagnosed with ET according to the World Health Organization (WHO) 2016 criteria.
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.
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.
Creatinine clearance ≥40 mL/min (by Cockcroft-Gault equation).
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.
Written informed consent obtained from the subject and ability for the subject to comply with the requirements of the study.
Platelet count >450 × 109/L at screening
Both ANA-naïve and ANA-pretreated subjects are eligible for the study, regardless of the reason to terminate ANA use
Exclusion Criteria:
Any subject requiring a legally authorized representative
Subjects who stopped prior interferon alfa therapy due to low efficacy or poor tolerability
Any contraindications or hypersensitivity to IFN-α and/or its excipients
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
History of major organ transplantation
Pregnant or lactating females
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:
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)
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
Infections with systemic manifestations (e.g., bacterial, fungal, or human immunodeficiency virus [HIV], except hepatitis B [HBV] and/or hepatitis C [HCV],at screening)
Evidence of severe retinopathy (e.g., cytomegalovirus retinitis [CMV], macular degeneration) or clinically relevant ophthalmological disorder (due to diabetes mellitus or hypertension)
History or presence of clinically relevant depression
Previous suicide attempts or at any risk of suicide at screening, in the judgment of the Investigator
History or presence of clinically significant neurologic diseases
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)
History of alcohol or drug abuse within the last year
History or evidence of any other MPN
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
Presence of more than one driver mutation (e.g., V617F JAK2 and CALR, CALR and MPL, V617F JAK2 and MPL)
Prior use of JAK inhibitors
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Jewell Jessup, PhD
Phone
1-800-999-2449
Email
clinicaltrials@pharmaessentia.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ray Urbanski, MD, PhD
Organizational Affiliation
PharmaEssentia USA Corporation
Official's Role
Study Director
Facility Information:
Facility Name
University of Alabama at Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35294
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Mitch Shea
Email
sheam@uab.edu
Facility Name
City of Hope National Medical Center
City
Duarte
State/Province
California
ZIP/Postal Code
91010
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jewell Jessup, PhD
Phone
800-999-2449
Email
clinicaltrials@pharmaessentia.com
Facility Name
Marin Cancer Care
City
Greenbrae
State/Province
California
ZIP/Postal Code
94904
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jaime Chang
Phone
415-925-5040
Email
jchang@marincancercare.com
Facility Name
USC Norris Comprehensive Cancer Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90033
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jewell Jessup, PhD
Phone
800-999-2449
Email
clinicaltrials@pharmaessentia.com
Facility Name
Yale University School of Medicine - Yale Cancer Center
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06510
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Anne Caldwell
Phone
203-785-3465
Email
anne.caldwell@yale.edu
Facility Name
Georgetown University Medical Center
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20057
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jennifer Steffen
Email
js4936@georgetown.edu
Facility Name
The Winship Cancer Institute Emory University
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Shannon Gleason
Phone
404-778-4334
Email
shannon.gleason@emory.edu
Facility Name
Fort Wayne Medical Oncology and Hematology
City
Fort Wayne
State/Province
Indiana
ZIP/Postal Code
46804
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Melissa Parker
Email
Melissa.parker@fwmoh.com
Facility Name
Mercy Health - Paducah Medical Oncology and Hematology
City
Paducah
State/Province
Kentucky
ZIP/Postal Code
42003
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Barbie Warner
Phone
270-441-4343
Email
BJWarner@mercy.com
Facility Name
Tulane University Medical Center
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70112
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Leta Ko
Phone
504-988-6120
Email
lko@tulane.edu
Facility Name
Greater Baltimore Medical Center
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21204
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Steven Schmitt
Phone
443-849-3285
Email
sschmitt@gbmc.org
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jewell Jessup, PhD
Phone
800-999-2449
Email
clinicaltrials@pharmaessential.com
Facility Name
Dana-Farber Cancer Institute
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Morgan Johnson
Email
morgan_johnson@dfci.harvard.edu
Facility Name
Washington University School of Medicine - Division of Oncology
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Karyn Gordon
Phone
314-362-0156
Email
kdgordon@wustl.edu
Facility Name
Cancer Care Specialists
City
Reno
State/Province
Nevada
ZIP/Postal Code
89511
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Layla Tapia
Phone
775-329-0222
Ext
230
Email
ltapia@ccsreno.com
Facility Name
Astera HealthCare
City
East Brunswick
State/Province
New Jersey
ZIP/Postal Code
08816
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Stephanie Ortiz
Phone
732-390-7750
Email
stephanie.ortiz@asterahealthcare.org
Facility Name
John Theurer Cancer Center At Hackensack UMC
City
Hackensack
State/Province
New Jersey
ZIP/Postal Code
07601
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jason Brecher
Phone
551-996-5274
Email
jason.brecher@hmhn.org
Facility Name
Montefiore Medical Center
City
Bronx
State/Province
New York
ZIP/Postal Code
10467
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Noelle Townsend
Phone
718-430-2377
Email
noelle.townsend@einsteinmed.edu
Facility Name
Weill Medical College of Cornell University
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jewell Jessup, PhD
Phone
800-999-2449
Email
clinicaltrials@pharmaessentia.com
Facility Name
Memorial Sloan Kettering Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jewell Jessup, PhD
Phone
800-999-2449
Email
clinicaltrials@pharmaessentia.com
Facility Name
Stony Brook University Medical Center
City
Stony Brook
State/Province
New York
ZIP/Postal Code
11794
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Zita Makselyte
Phone
631-638-0844
Email
Zita.Makselyte@stonybrookmedicine.edu
Facility Name
University of North Carolina (UNC) - Lineberger Comprehensive Cancer Center
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27514
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jewell Jessup, PhD
Phone
800-999-2449
Email
clinicaltrials@pharmaessentia.com
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Peggy Alton
Phone
919-684-9220
Email
peggy.alton@duke.edu
Facility Name
East Carolina University
City
Greenville
State/Province
North Carolina
ZIP/Postal Code
27858
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Toria Wilson
Email
wilsonto22@ecu.edu
Facility Name
Regional Medical Oncology Center
City
Wilson
State/Province
North Carolina
ZIP/Postal Code
27893
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ines Hernandez
Phone
252-991-5261
Email
ines.hernandez@regionaloncology.com
Facility Name
University of Tennessee Health Science Center
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38103
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Suzhen Gong
Email
sgong@uthsc.edu
Facility Name
MD Anderson Cancer Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Mary Ann Richie
Phone
713-794-5478
Email
marichie@mdanderson.org
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Braxton Smith
Phone
801-213-8431
Email
braxton.smith@hci.utah.edu
Facility Name
University of Virginia - Emily Couric Cancer Center
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22903
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ellie Gorham
Phone
434-297-5726
Email
eg3ap@hscmail.mcc.virginia.edu
Facility Name
Fred Hutchinson Cancer Research Center
City
Seattle
State/Province
Washington
ZIP/Postal Code
98109
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jewell Jessup, PhD
Phone
800-999-2449
Email
clinicaltrials@pharmaessentia.com
Facility Name
University of Calgary Tom Baker Cancer Centre
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T2N 4N2
Country
Canada
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jewell Jessup, PhD
Phone
800-999-2449
Email
clinicaltrials@pharmaessentia.com
Facility Name
St. Paul's Hospital - Providence Health Care
City
Vancouver
State/Province
British Columbia
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Tathiana Ruiz
Phone
6046822344
Ext
64987
Email
truiz1@providencehealth.bc.ca
Facility Name
Juravinski Cancer Centre
City
Hamilton
State/Province
Ontario
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Christopher Hillis
Phone
905-387-9495
Email
hillis@hhsc.ca
Facility Name
Princess Margaret Hospital
City
Toronto
State/Province
Ontario
Country
Canada
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Nina Dimson
Email
nina.dimson@uhn.ca
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of P1101 in Adults With ET
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