Myeloproliferative Neoplasms Clinical Trial

A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of P1101 in Adults With ET

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

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Full 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.

View Eligibility Criteria

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

Study is for people with:

Myeloproliferative Neoplasms

Phase:

Phase 2

Estimated Enrollment:

64

Study ID:

NCT05482971

Recruitment Status:

Recruiting

Sponsor:

PharmaEssentia

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There are 31 Locations for this study

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University of Alabama at Birmingham
Birmingham Alabama, 35294, United States More Info
Mitch Shea
Contact
[email protected]
City of Hope National Medical Center
Duarte California, 91010, United States More Info
Jewell Jessup, PhD
Contact
800-999-2449
[email protected]
Marin Cancer Care
Greenbrae California, 94904, United States More Info
Jaime Chang
Contact
415-925-5040
[email protected]
USC Norris Comprehensive Cancer Center
Los Angeles California, 90033, United States More Info
Jewell Jessup, PhD
Contact
800-999-2449
[email protected]
Yale University School of Medicine - Yale Cancer Center
New Haven Connecticut, 06510, United States More Info
Anne Caldwell
Contact
203-785-3465
[email protected]
Georgetown University Medical Center
Washington District of Columbia, 20057, United States More Info
Jennifer Steffen
Contact
[email protected]
The Winship Cancer Institute Emory University
Atlanta Georgia, 30322, United States More Info
Shannon Gleason
Contact
404-778-4334
[email protected]
Fort Wayne Medical Oncology and Hematology
Fort Wayne Indiana, 46804, United States More Info
Melissa Parker
Contact
[email protected]
Mercy Health - Paducah Medical Oncology and Hematology
Paducah Kentucky, 42003, United States More Info
Barbie Warner
Contact
270-441-4343
[email protected]
Tulane University Medical Center
New Orleans Louisiana, 70112, United States More Info
Greater Baltimore Medical Center
Baltimore Maryland, 21204, United States More Info
Steven Schmitt
Contact
443-849-3285
[email protected]
Massachusetts General Hospital
Boston Massachusetts, 02114, United States More Info
Jewell Jessup, PhD
Contact
800-999-2449
[email protected]
Dana-Farber Cancer Institute
Boston Massachusetts, 02215, United States More Info
Morgan Johnson
Contact
[email protected]
Washington University School of Medicine - Division of Oncology
Saint Louis Missouri, 63110, United States More Info
Karyn Gordon
Contact
314-362-0156
[email protected]
Cancer Care Specialists
Reno Nevada, 89511, United States More Info
Layla Tapia
Contact
775-329-0222
[email protected]
Astera HealthCare
East Brunswick New Jersey, 08816, United States More Info
Stephanie Ortiz
Contact
732-390-7750
[email protected]
John Theurer Cancer Center At Hackensack UMC
Hackensack New Jersey, 07601, United States More Info
Jason Brecher
Contact
551-996-5274
[email protected]
Montefiore Medical Center
Bronx New York, 10467, United States More Info
Noelle Townsend
Contact
718-430-2377
[email protected]
Weill Medical College of Cornell University
New York New York, 10021, United States More Info
Jewell Jessup, PhD
Contact
800-999-2449
[email protected]
Memorial Sloan Kettering Cancer Center
New York New York, 10065, United States More Info
Jewell Jessup, PhD
Contact
800-999-2449
[email protected]
Stony Brook University Medical Center
Stony Brook New York, 11794, United States More Info
Zita Makselyte
Contact
631-638-0844
[email protected]
University of North Carolina (UNC) - Lineberger Comprehensive Cancer Center
Chapel Hill North Carolina, 27514, United States More Info
Jewell Jessup, PhD
Contact
800-999-2449
[email protected]
Duke University Medical Center
Durham North Carolina, 27710, United States More Info
Peggy Alton
Contact
919-684-9220
[email protected]
East Carolina University
Greenville North Carolina, 27858, United States More Info
Toria Wilson
Contact
[email protected]
Regional Medical Oncology Center
Wilson North Carolina, 27893, United States More Info
Ines Hernandez
Contact
252-991-5261
[email protected]
University of Tennessee Health Science Center
Memphis Tennessee, 38103, United States More Info
Suzhen Gong
Contact
[email protected]
MD Anderson Cancer Center
Houston Texas, 77030, United States More Info
Mary Ann Richie
Contact
713-794-5478
[email protected]
University of Utah
Salt Lake City Utah, 84132, United States More Info
Braxton Smith
Contact
801-213-8431
[email protected]
University of Virginia - Emily Couric Cancer Center
Charlottesville Virginia, 22903, United States More Info
Ellie Gorham
Contact
434-297-5726
[email protected]
Fred Hutchinson Cancer Research Center
Seattle Washington, 98109, United States More Info
Jewell Jessup, PhD
Contact
800-999-2449
[email protected]
University of Calgary Tom Baker Cancer Centre
Calgary Alberta, T2N 4, Canada More Info
Jewell Jessup, PhD
Contact
800-999-2449
[email protected]
St. Paul's Hospital - Providence Health Care
Vancouver British Columbia, , Canada More Info
Tathiana Ruiz
Contact
6046822344
[email protected]
Juravinski Cancer Centre
Hamilton Ontario, , Canada More Info
Christopher Hillis
Contact
905-387-9495
[email protected]
Princess Margaret Hospital
Toronto Ontario, , Canada More Info
Nina Dimson
Contact
[email protected]

How clear is this clinincal trial information?

Study is for people with:

Myeloproliferative Neoplasms

Phase:

Phase 2

Estimated Enrollment:

64

Study ID:

NCT05482971

Recruitment Status:

Recruiting

Sponsor:


PharmaEssentia

How clear is this clinincal trial information?

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