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Essential ThrombocythemiaPhase 3SURPASS ETNCT04285086

Ropeginterferon Alfa-2b (P1101) vs. Anagrelide in Essential Thrombocythemia Patients With Hydroxyurea Resistance or Intolerance

A Phase 3 study testing Ropeginterferon alfa-2b and Anagrelide in people with Essential Thrombocythemia, run by PharmaEssentia at 67 sites in 8 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial summary

This is a Phase 3 open-label, multicenter, randomized, active-controlled study designed to compare the efficacy and safety and tolerability of P1101 compared with ANA after 12 months of treatment as second-line therapy for subjects with ET who have had a suboptimal or failed response to HU. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Essential Thrombocythemia
What is being tested?
Ropeginterferon alfa-2b and Anagrelide
Could I receive a placebo?
No, everyone receives an active treatment
How many people
174 participants
Where
67 sitesin 8 countries
Study dates
25 Aug 2020to 31 Aug 2029
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Male or female subjects ≥18 years old
  • 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
  • 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
  • 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.
  • 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.
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  • Platelets >450 x 10^9/L at screening
  • WBC >10 x 10^9/L at screening
  • HGB ≥11 g/dL at screening for males and 10 g/dL at screening for females
  • Neutrophil count ≥1.0 x 10^9/L at screening
  • 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
  • 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 28 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

Who cannot join

  • Any subject requiring a legally authorized representative
  • Any contraindications or hypersensitivity to IFN-α or ANA and their excipients
  • 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.
  • 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
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  • 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:
  • 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, 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 judgement of the Investigator
  • History or presence of clinically significant neurologic diseases
  • History of any malignancy within 5 years (except Stage 0 chronic lymphocytic leukemia, basal cell, squamous cell, and superficial melanoma)
  • History of alcohol or drug abuse within the last year
  • History or evidence of any other MPN
  • History of splenectomy
  • 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
  • Subjects with documented ANA resistance or intolerance (see Appendix 8 for definition).

Trial locations and contacts

67 sites in 8 countries.

67 sites in 8 countries

Taiwan21 sites

  • Chia-Yi Christian Hospital

    Chiayi City, Chiayi County, 60002

  • Chiayi Chang Gung Memorial Hospital

    Chiayi City, Chiayi County, 61363

  • Hualien Tzu Chi Hospital

    Hualien City, 97002

  • Kaohsiung Veterans General Hospital

    Kaohsiung City, Kaohsiung City, 81362

  • E-Da Cancer Hospital

    Kaohsiung City, Kaohsiung City, 82445

  • E-Da Hospital

    Kaohsiung City, Kaohsiung City, 82445

  • Kaohsiung Medical University Chung-Ho Memorial Hospital

    Kaohsiung City, 80756

  • Kaohsiung Chang Gung Memorial Hospital

    Kaohsiung City, 83301

  • Far Eastern Memorial Hospital

    New Taipei City, New Taipei City, 22060

  • China Medical University Hospital

    Taichung, 40447

  • Tainan Municipal An-Nan Hospital

    Tainan, Tainan City, 70965

  • Chi Mei Medical Center

    Tainan, Tainan City, 71004

  • Chi-Mei Hospital - Liouying Branch

    Tainan, Tainan City, 73657

  • National Cheng Kung University Hospital

    Tainan, 70403

  • Shin Kong Wu Ho-Su Memorial Hospital

    Taipei, Taipei City, 11101

  • Taipei Municipal Wan Fang Hospital

    Taipei, Taipei City, 11696

  • National Taiwan University Hospital

    Taipei, 10002

  • Mackay Memorial Hospital

    Taipei, 10449

  • Taipei Veterans General Hospital

    Taipei, 11217

  • Tri-Service General Hospital

    Taipei, 11449

  • Linkou Chang Gung Memorial Hospital

    Taoyuan City, 33305

Other trials in Taiwan

China15 sites

  • Peking Union Medical College Hospital

    Beijing, Beijing Municipality

  • Peking University People's Hospital

    Beijing, Beijing Municipality

  • West China Hospital, Sichuan University

    Chengdu, Sichuan

  • The First Affiliated Hospital, Chongqing Medical University

    Chongqing, Chongqing Municipality

  • NanFang Hospital of Southern Medical University

    Guangzhou, Guangdong

  • The First Affiliated Hospital, College of Medicine, Zhejiang University

    Hangzhou, Zhejiang

  • Qilu Hospital of Shandong University

    Jinan, Shandong

  • Ruijin Hospital affiliated to Shanghai Jiao Tong University school of Medicine

    Shanghai, Shanghai Municipality

  • Shengjing Hospital of China Medical University

    Shenyang, Liaoning

  • The First Affiliated Hospital of Soochow University

    Suzhou, Jiangsu

  • The Second Hospital of Tianjin Medical University

    Tianjin, Tianjin Municipality, 300211

  • Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences

    Tianjin, Tianjin Municipality

  • Union Hospital Tongji Medical College Huazhong University of Science and Technolog

    Wuhan, Hubei

  • Zhongnan Hospital of Wuhan University

    Wuhan, Hubei

  • Shaanxi Provincial People's Hospital

    Xi'an, Shaanxi

Other trials in China

Japan13 sites

  • Juntendo University Hospital

    Bunkyo City, Tokyo, 113-8431

  • Nippon Medical School Hospital

    Bunkyo City, Tokyo, 113-8603

  • University of Yamanashi Hospital

    Chūō, Yamanashi, 409-3898

  • Kansai Medical University Hospital

    Hirakata, Osaka, 573-1191

  • Juntendo University Shizuoka Hospital

    Izunokuni, Shizuoka, 410-2295

  • University of Miyazaki Hospital

    Miyazaki, Miyazaki, 889-1692

  • Kitasato University Hospital

    Sagamihara, Kanagawa, 252-0329

  • Kindai University Hospital

    Sayama, Osaka, 589-8511

  • NTT Medical Center Tokyo

    Shinagawa City, Tokyo, 141-0022

  • Tokyo Medical University Hospital

    Shinjuku, Tokyo, 160-0023

  • Osaka University Hospital

    Suita, Osaka, 565-0871

  • Ehime University Hospital

    Tōon, Ehime, 791-0204

  • Mie University Hospital

    Tsu, Mie-ken, 514-8507

Other trials in Japan

South Korea8 sites

  • Daegu Catholic University Hospital

    Daegu, 30566

  • Gachon University Gil Medical Center

    Incheon, 21565

  • Seoul National University Hospital

    Seoul, 03080

  • Severance Hospital, Yonsei University Health System

    Seoul, 03722

  • SoonChunHyang University Seoul Hospital

    Seoul, 04401

  • Samsung Medical Center

    Seoul, 06351

  • Seoul St. Mary's Hospital, The Catholic University of Korea

    Seoul, 06591

  • Korea University Guro Hospital

    Seoul, 08308

Other trials in South Korea

United States4 sites

  • MD Anderson Cancer Center

    Houston, Texas, 77030

  • University of Utah

    Salt Lake City, Utah, 84112

  • Mayo Clinic - Scottsdale

    Scottsdale, Arizona, 85259

  • Washington University School of Medicine - Division of Oncology

    St Louis, Missouri, 63110

Other trials in United States

Canada3 sites

  • Jewish General Hospital

    Montreal, Quebec, H3T 1E2

  • Princess Margaret Hospital

    Toronto, Ontario, M5G 2C1

  • St. Paul's Hospital

    Vancouver, British Columbia, V6Z 1Y6

Other trials in Canada

Singapore2 sites

  • National University Hospital

    Singapore, 119074

  • Singapore General Hospital

    Singapore, 169608

Other trials in Singapore

Hong Kong1 site

  • Queen Mary Hospital

    Hong Kong

Other trials in Hong Kong

About this study

Official title: A Phase 3, Open-Label, Multicenter, Randomized, Active-controlled Study to Assess PK and Compare the Efficacy, Safety, and Tolerability of P1101 vs Anagrelide as 2nd Line Therapy for Essential Thrombocythemia (SURPASS ET): The Core Study and Its Extension Study.

This is a Phase 3 open-label, multicenter, randomized, active-controlled study designed to compare the efficacy and safety and tolerability of P1101 compared with ANA after 12 months of treatment as second-line therapy for subjects with ET who have had a suboptimal or failed response to HU.

Read the detailed description

PharmaEssentia Corporation is developing a pegylated (PEG) IFN-α product, P1101, for the treatment of ET.

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.

Being tested
Ropeginterferon alfa-2b, Anagrelide
Keywords
Essential Thrombocythemia, Ropeginterferon, P1101, PharmaEssentia, MPN, Myeloproliferative neoplasms, ET, JAK2, CALR, MPL, IFN
Medical subject headings
Thrombocythemia, Essential, Myeloproliferative Disorders

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Ropeginterferon alfa-2bBiological

    Also called P1101

    Ropeginterferon alfa-2b (P1101) dosage: from 250 mcg to 500 mcg

  • AnagrelideDrug

    Anagrelide dosage: 0.5 mg per capsule, according to label and physician's judgement

Study groups in NCT04285086
Study groupRoleWhat they receive
Ropeginterferon alfa-2b (P1101)ExperimentalPre-filled Syringe, Q2W, SC injection
Receives: Biological: Ropeginterferon alfa-2b
AnagrelideActive comparatorCapsules, Daily, p.o.
Receives: Drug: Anagrelide

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Peripheral blood count remission

    platelets ≤400 x 10^9/L AND white blood cells (WBC) <9.5 x 10^9/L

    Measured: month 9 and month 12

  2. Improvement or non-progression in disease-related signs

    splenomegaly

    Measured: month 9 and month 12

  3. Large symptoms improvement or maintain non-progression

    based on the Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)

    Measured: month 9 and month 12

  4. Absence of hemorrhagic or thrombotic events

    absence of hemorrhagic or thrombotic events

    Measured: month 9 and month 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Feb 2020
  2. Study start25 Aug 2020
  3. Primary completion12 Nov 2024
  4. Study completion31 Aug 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Experimental Drug (Biological): Ropeginterferon alfa-2b (P1101), Q2W, SC injection Control Drug: Anagrelide, capsules, daily, p.o.
Masking
None (open label)
Enrolment
174 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04285086
Sponsor's protocol ID
P1101 ET
Acronym
SURPASS ET
Status last verified
Jul 2026
First submitted
23 Feb 2020
Last updated
22 Jul 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Mesa R, Gill H, Zhang L, Jin J, Kirito K, Komatsu N, Qin A, Xiao Z, Tashi T, Shimoda K, Ohishi K, Chen S, Zuo X, Shirane S, Hu Y, Zhang S, Wang Y, Takenaka K, Ichii M, Xu N, Shih LY, Lim KH, Lee SE, Bae SH, Teo WZY, Maze D, Oh ST, Bose P, Sato T, Zagrijtschuk O, Lin S, Shih WJ, Mascarenhas J, Masarova L; SURPASS-ET Study Group. Ropeginterferon alfa-2b in hydroxyurea-intolerant or hydroxyurea-refractory essential thrombocythaemia (SURPASS ET): a multicentre, open-label, randomised, active-controlled, phase 3 study. Lancet Haematol. 2025 Nov;12(11):e862-e875. doi: 10.1016/S2352-3026(25)00264-9.

    PubMed 41193116

  • 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.

    PubMed 35924546

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Ropeginterferon alfa-2b and Anagrelide in people with Essential Thrombocythemia, run by PharmaEssentia at 67 sites in 8 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 14 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 22 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 67 sites in Taiwan, China, Japan, South Korea, United States and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Peripheral blood count remission (time frame: month 9 and month 12). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04285086, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jul 2026.

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