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

Phase 2

Active, not recruiting

• Conditions: Essential Thrombocythemia
• Interventions: Drug: Ropeginterferon alfa-2b-njft (P1101)

• Registration Number: NCT05482971
• Lead Sponsor: PharmaEssentia

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.

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations
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Related News

Study Timeline

• Study First Submitted: 2022-07-28
• Study First Submitted QC: 2022-07-28
• Study First Posted: 2022-08-01
• Study Start: 2022-09-29
• Status Verified: 2024-10
• Last Update Submitted: 2024-10-21
• Last Update Posted: 2024-10-23
• Primary Completion: 2027-03-31
• Study Completion: 2027-03-31

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 91

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Ropeginterferon alfa-2b (P1101)	Ropeginterferon alfa-2b-njft (P1101)	Pre-filled Syringe, Q2W, SC injection

Primary Outcome Measures

Name	Time	Method
To assess efficacy of ropeginterferon alfa-2b-njft (P1101) in adult USA/Canadian patients with ET	12 months	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.

Trial Locations

Locations (34)

University of Alabama at Birmingham; 🇺🇸 Birmingham, Alabama, United States

City of Hope National Medical Center; 🇺🇸 Duarte, California, United States

Marin Cancer Care; 🇺🇸 Greenbrae, California, United States

USC Norris Comprehensive Cancer Center; 🇺🇸 Los Angeles, California, United States

Yale University School of Medicine - Yale Cancer Center; 🇺🇸 New Haven, Connecticut, United States

Georgetown University Medical Center; 🇺🇸 Washington, District of Columbia, United States

The Winship Cancer Institute Emory University; 🇺🇸 Atlanta, Georgia, United States

Fort Wayne Medical Oncology and Hematology; 🇺🇸 Fort Wayne, Indiana, United States

Mercy Health - Paducah Medical Oncology and Hematology; 🇺🇸 Paducah, Kentucky, United States

Tulane University Medical Center; 🇺🇸 New Orleans, Louisiana, United States

Greater Baltimore Medical Center; 🇺🇸 Baltimore, Maryland, United States

Massachusetts General Hospital; 🇺🇸 Boston, Massachusetts, United States

Dana-Farber Cancer Institute; 🇺🇸 Boston, Massachusetts, United States

Washington University School of Medicine - Division of Oncology; 🇺🇸 Saint Louis, Missouri, United States

Cancer Care Specialists; 🇺🇸 Reno, Nevada, United States

Astera HealthCare; 🇺🇸 East Brunswick, New Jersey, United States

John Theurer Cancer Center At Hackensack UMC; 🇺🇸 Hackensack, New Jersey, United States

Montefiore Medical Center; 🇺🇸 Bronx, New York, United States

Weill Medical College of Cornell University; 🇺🇸 New York, New York, United States

Memorial Sloan Kettering Cancer Center; 🇺🇸 New York, New York, United States

Stony Brook University Medical Center; 🇺🇸 Stony Brook, New York, United States

University of North Carolina (UNC) - Lineberger Comprehensive Cancer Center; 🇺🇸 Chapel Hill, North Carolina, United States

Duke University Medical Center; 🇺🇸 Durham, North Carolina, United States

East Carolina University; 🇺🇸 Greenville, North Carolina, United States

Regional Medical Oncology Center; 🇺🇸 Wilson, North Carolina, United States

University of Tennessee Health Science Center; 🇺🇸 Memphis, Tennessee, United States

MD Anderson Cancer Center; 🇺🇸 Houston, Texas, United States

University of Utah; 🇺🇸 Salt Lake City, Utah, United States

University of Virginia - Emily Couric Cancer Center; 🇺🇸 Charlottesville, Virginia, United States

Fred Hutchinson Cancer Research Center; 🇺🇸 Seattle, Washington, United States

University of Calgary Tom Baker Cancer Centre; 🇨🇦 Calgary, Alberta, Canada

St. Paul's Hospital - Providence Health Care; 🇨🇦 Vancouver, British Columbia, Canada

Juravinski Cancer Centre; 🇨🇦 Hamilton, Ontario, Canada

Princess Margaret Hospital; 🇨🇦 Toronto, Ontario, Canada