To hear about similar clinical trials, please enter your email below

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

Login to your account

Did you forget your password?