Ropeginterferon Alfa-2b (P1101) vs. Anagrelide in Essential Thrombocythemia Patients With Hydroxyurea Resistance or Intolerance
NCT ID: NCT04285086
Last Updated: 2025-08-13
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
174 participants
INTERVENTIONAL
2020-08-25
2029-08-31
Brief Summary
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Detailed Description
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Available clinical data and experience with P1101 in PV shows that the compound, with proper dose modifications, is effective in controlling disease in a significant proportion of subjects with ET. Further, its increased serum half-life presents distinct advantages for ET treatment over that of standard IFN-α and other available PEG IFN-α therapy. This pivotal Phase 3 study will establish the efficacy and safety of P1101 in ET subjects.
In core study phase, the enrolled subjects will be randomized into two arms, the test arm is P1101, the control arm is ANA. The overall duration for each eligible patient is 14 months, including screening (1 month), treatment (12 months) and follow-up (1 month) period. Efficacy evaluations, safety assessments, and PK and immunogenicity evaluations of P1101 will be performed.
Evaluation of efficacy will include clinical laboratory assessments, allelic burden measurements of CALR, JAK-2, and MPL, spleen size measurements, bone marrow sampling, EQ-5D-3L, and MPN-SAF TSS completion.
Evaluation of safety will include assessing vital signs, clinical safety laboratory tests, physical examinations, ECG evaluation, heart ECHO, lung X-ray, ECOG performance status, ocular examination, and AEs.
Subjects who completed the end of treatment (EoT) and safety follow-up (EoS) visits of the SURPASS ET study and may benefit from P1101 therapy have the opportunity to receive P1101 treatment.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Ropeginterferon alfa-2b (P1101)
Pre-filled Syringe, Q2W, SC injection
Ropeginterferon alfa-2b
Ropeginterferon alfa-2b (P1101) dosage: from 250 mcg to 500 mcg
Anagrelide
Capsules, Daily, p.o.
Anagrelide
Anagrelide dosage: 0.5 mg per capsule, according to label and physician's judgement
Interventions
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Ropeginterferon alfa-2b
Ropeginterferon alfa-2b (P1101) dosage: from 250 mcg to 500 mcg
Anagrelide
Anagrelide dosage: 0.5 mg per capsule, according to label and physician's judgement
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Subjects diagnosed with high-risk ET (either older than 60 years and JAK2V617-positive at screening, or having disease-related thrombosis or hemorrhage in the past), diagnosed according to the World Health Organization (WHO) 2016 criteria
3. Subjects have received prior HU for ET, while the washout between the last dose of HU and randomization should not be shorter than 7 days
4. Interferon treatment-naïve, or anti-P1101 binding antibody negative at screening and the washout between last dose of interferon and randomization should not be shorter than 14 days.
5. Documented resistance/intolerance to prior HU for ET, referencing modified ELN criteria (Barosi, et al, 2007), whereby at least one of the following criteria is met:
Platelet count \>600 x 10\^9/L at ≥2 g/day (or ≥2.5 g/day if subject body weight \>80 kg) or maximally tolerated dose if \<2 g/day after at least 3 months of HU, or Platelet count \>400 x 10\^9/L and WBC count \<2.5 x 10\^9/L at any dose and any duration of HU, or Platelet count \>400 x 10\^9/L and hemoglobin (HGB) \<10 g/dL at any dose and any duration of HU, or Presence of HU-related toxicities at any dose and any duration of therapy (e.g., leg ulcers, mucocutaneous manifestations, pneumonitis, or HU-related fever), or Platelet count \>450 x 10\^9/L at any dose and any duration of HU. The actual dose and duration of HU must be recorded on the eCRF. Moreover, if patient received one dose of HU, the reason why subject was judged to be HU resistance/intolerance must be recorded on the eCRF.
6. Platelets \>450 x 10\^9/L at screening
7. WBC \>10 x 10\^9/L at screening
8. HGB ≥11 g/dL at screening for males and 10 g/dL at screening for females
9. Neutrophil count ≥1.0 x 10\^9/L at screening
10. Adequate hepatic function defined as bilirubin ≤1.5 x upper limit normal (ULN), prothrombin time (PT) (international normalized ratio, INR) ≤1.5 x ULN, albumin \>3.5 g/dL, alanine aminotransferase ≤2.0 x ULN, aspartate aminotransferase ≤2.0 x ULN at screening
11. Creatinine clearance ≥40 mL/min (by Cockcroft-Gault equation)
12. 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 28 days following the last dose of the study drug, and females must agree to not breastfeed during the study
13. Written informed consent obtained from the subject and ability for the subject to comply with the requirements of the study
Exclusion Criteria
2. Any contraindications or hypersensitivity to IFN-α or ANA and their excipients
3. Known risk factors for QT-prolongation (e.g., congenital long QT, known history of acquired QT-prolongations). Medications that can prolong QTc and induce hypokalemia will not be allowed in the study.
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 other 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, 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 judgement of the Investigator
7. History or presence of clinically significant neurologic diseases
8. History of any malignancy within 5 years (except Stage 0 chronic lymphocytic leukemia, basal cell, squamous cell, and superficial melanoma)
9. History of alcohol or drug abuse within the last year
10. History or evidence of any other MPN
11. History of splenectomy
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. Subjects with documented ANA resistance or intolerance (see Appendix 8 for definition).
18 Years
ALL
No
Sponsors
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PharmaEssentia
INDUSTRY
Responsible Party
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Principal Investigators
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Toshiaki Sato, MD/PhD
Role: STUDY_DIRECTOR
PharmaEssentia Japan K.K.
Craig Zimmerman, PhD
Role: STUDY_DIRECTOR
PharmaEssentia USA Corp.
Locations
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Washington University School of Medicine - Division of Oncology
St Louis, Missouri, United States
MD Anderson Cancer Center
Houston, Texas, United States
University of Utah
Salt Lake City, Utah, United States
St. Paul's Hospital
Vancouver, British Columbia, Canada
Princess Margaret Hospital
Toronto, Ontario, Canada
Jewish General Hospital
Montreal, Quebec, Canada
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China
Peking University People's Hospital
Beijing, Beijing Municipality, China
The First Affiliated Hospital, Chongqing Medical University
Chongqing, Chongqing Municipality, China
NanFang Hospital of Southern Medical University
Guangzhou, Guangdong, China
Union Hospital Tongji Medical College Huazhong University of Science and Technolog
Wuhan, Hubei, China
Zhongnan Hospital of Wuhan University
Wuhan, Hubei, China
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, China
Shengjing Hospital of China Medical University
Shenyang, Liaoning, China
Shaanxi Provincial People's Hospital
Xi'an, Shaanxi, China
Qilu Hospital of Shandong University
Jinan, Shandong, China
Ruijin Hospital affiliated to Shanghai Jiao Tong University school of Medicine
Shanghai, Shanghai Municipality, China
West China Hospital, Sichuan University
Chengdu, Sichuan, China
The Second Hospital of Tianjin Medical University
Tianjin, Tianjin Municipality, China
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, China
The First Affiliated Hospital, College of Medicine, Zhejiang University
Hangzhou, Zhejiang, China
Queen Mary Hospital
Hong Kong, , Hong Kong
Ehime University Hospital
Tōon, Ehime, Japan
Kitasato University Hospital
Sagamihara, Kanagawa, Japan
Mie University Hospital
Tsu, Mie-ken, Japan
University of Miyazaki Hospital
Miyazaki, Miyazaki, Japan
Kansai Medical University Hospital
Hirakata, Osaka, Japan
Kindai University Hospital
Sayama, Osaka, Japan
Osaka University Hospital
Suita, Osaka, Japan
Juntendo University Shizuoka Hospital
Izunokuni, Shizuoka, Japan
Juntendo University Hospital
Bunkyo City, Tokyo, Japan
Nippon Medical School Hospital
Bunkyo City, Tokyo, Japan
NTT Medical Center Tokyo
Shinagawa City, Tokyo, Japan
Tokyo Medical University Hospital
Shinjuku, Tokyo, Japan
University of Yamanashi Hospital
Chūō, Yamanashi, Japan
National University Hospital
Singapore, , Singapore
Singapore General Hospital
Singapore, , Singapore
Daegu Catholic University Hospital
Daegu, , South Korea
Seoul National University Hospital
Seoul, , South Korea
Severance Hospital, Yonsei University Health System
Seoul, , South Korea
SoonChunHyang University Seoul Hospital
Seoul, , South Korea
Samsung Medical Center
Seoul, , South Korea
Seoul St. Mary's Hospital, The Catholic University of Korea
Seoul, , South Korea
Korea University Guro Hospital
Seoul, , South Korea
Chia-Yi Christian Hospital
Chiayi City, Chiayi County, Taiwan
Chiayi Chang Gung Memorial Hospital
Chiayi City, Chiayi County, Taiwan
Kaohsiung Veterans General Hospital
Kaohsiung City, Kaohsiung City, Taiwan
E-Da Cancer Hospital
Kaohsiung City, Kaohsiung City, Taiwan
E-Da Hospital
Kaohsiung City, Kaohsiung City, Taiwan
Far Eastern Memorial Hospital
New Taipei City, New Taipei City, Taiwan
Tainan Municipal An-Nan Hospital
Tainan City, Tainan City, Taiwan
Chi Mei Medical Center
Tainan City, Tainan City, Taiwan
Chi-Mei Hospital - Liouying Branch
Tainan City, Tainan City, Taiwan
Shin Kong Wu Ho-Su Memorial Hospital
Taipei, Taipei City, Taiwan
Taipei Municipal Wan Fang Hospital
Taipei, Taipei City, Taiwan
Hualien Tzu Chi Hospital
Hualien City, , Taiwan
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, , Taiwan
Kaohsiung Chang Gung Memorial Hospital
Kaohsiung City, , Taiwan
China Medical University Hospital
Taichung, , Taiwan
National Cheng Kung University Hospital
Tainan City, , Taiwan
National Taiwan University Hospital
Taipei, , Taiwan
Mackay Memorial Hospital
Taipei, , Taiwan
Taipei Veterans General Hospital
Taipei, , Taiwan
Tri-Service General Hospital
Taipei, , Taiwan
Linkou Chang Gung Memorial Hospital
Taoyuan, , Taiwan
Countries
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References
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Verstovsek S, Komatsu N, Gill H, Jin J, Lee SE, Hou HA, Sato T, Qin A, Urbanski R, Shih W, Zagrijtschuk O, Zimmerman C, Mesa RA. SURPASS-ET: phase III study of ropeginterferon alfa-2b versus anagrelide as second-line therapy in essential thrombocythemia. Future Oncol. 2022 Sep;18(27):2999-3009. doi: 10.2217/fon-2022-0596. Epub 2022 Aug 4.
Other Identifiers
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P1101 ET
Identifier Type: -
Identifier Source: org_study_id
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