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Can a biologic drug outperform standard therapy for a rare blood cancer?

NCT ID NCT04285086

Disease control Sponsor: PharmaEssentia Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jul 22, 2026 · Last updated Jul 23, 2026 · Updated 1 time

Summary

This phase 3 trial compares the biologic drug P1101 (ropeginterferon alfa-2b) against the standard drug anagrelide in people with essential thrombocythemia, a rare blood cancer that causes too many platelets. The study enrolls adults who have not responded well to the first-line treatment hydroxyurea. Researchers are measuring whether P1101 can better control blood counts, reduce spleen size, ease symptoms, and prevent dangerous blood clots or bleeding over 12 months.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
a biologic drug called ropeginterferon alfa-2b (P1101) compared with the drug anagrelide
What this could lead to
If P1101 proves more effective than anagrelide, it could offer a better second-line treatment option for people with essential thrombocythemia who did not respond well to hydroxyurea.
What could go wrong
This is a phase 3 trial, but it is open-label (both doctors and patients know which treatment is given), which can introduce bias. The drug may cause side effects similar to other interferons, such as flu-like symptoms or mood changes.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

174 people

The number who actually took part.

Started

Aug 2020

Expected to finish

Aug 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Male or female subjects ≥18 years old 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: 1. Any subject requiring a legally authorized representative 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).

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Conditions

The condition(s) this trial relates to.

essential thrombocythemia Myeloproliferative Disorders

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Essential thrombocythemia

Contacts and locations

Locations

  • Chi Mei Medical Center

    Tainan, Tainan City, 71004, Taiwan

  • Chi-Mei Hospital - Liouying Branch

    Tainan, Tainan City, 73657, Taiwan

  • Chia-Yi Christian Hospital

    Chiayi City, Chiayi County, 60002, Taiwan

  • Chiayi Chang Gung Memorial Hospital

    Chiayi City, Chiayi County, 61363, Taiwan

  • China Medical University Hospital

    Taichung, 40447, Taiwan

  • Daegu Catholic University Hospital

    Daegu, 30566, South Korea

  • E-Da Cancer Hospital

    Kaohsiung City, Kaohsiung City, 82445, Taiwan

  • E-Da Hospital

    Kaohsiung City, Kaohsiung City, 82445, Taiwan

  • Ehime University Hospital

    Tōon, Ehime, 791-0204, Japan

  • Far Eastern Memorial Hospital

    New Taipei City, New Taipei City, 22060, Taiwan

  • Gachon University Gil Medical Center

    Incheon, 21565, South Korea

  • Hualien Tzu Chi Hospital

    Hualien City, 97002, Taiwan

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

    Tianjin, Tianjin Municipality, China

  • Jewish General Hospital

    Montreal, Quebec, H3T 1E2, Canada

  • Juntendo University Hospital

    Bunkyo City, Tokyo, 113-8431, Japan

  • Juntendo University Shizuoka Hospital

    Izunokuni, Shizuoka, 410-2295, Japan

  • Kansai Medical University Hospital

    Hirakata, Osaka, 573-1191, Japan

  • Kaohsiung Chang Gung Memorial Hospital

    Kaohsiung City, 83301, Taiwan

  • Kaohsiung Medical University Chung-Ho Memorial Hospital

    Kaohsiung City, 80756, Taiwan

  • Kaohsiung Veterans General Hospital

    Kaohsiung City, Kaohsiung City, 81362, Taiwan

  • Kindai University Hospital

    Sayama, Osaka, 589-8511, Japan

  • Kitasato University Hospital

    Sagamihara, Kanagawa, 252-0329, Japan

  • Korea University Guro Hospital

    Seoul, 08308, South Korea

  • Linkou Chang Gung Memorial Hospital

    Taoyuan City, 33305, Taiwan

  • MD Anderson Cancer Center

    Houston, Texas, 77030, United States

  • Mackay Memorial Hospital

    Taipei, 10449, Taiwan

  • Mayo Clinic - Scottsdale

    Scottsdale, Arizona, 85259, United States

  • Mie University Hospital

    Tsu, Mie-ken, 514-8507, Japan

  • NTT Medical Center Tokyo

    Shinagawa City, Tokyo, 141-0022, Japan

  • NanFang Hospital of Southern Medical University

    Guangzhou, Guangdong, China

  • National Cheng Kung University Hospital

    Tainan, 70403, Taiwan

  • National Taiwan University Hospital

    Taipei, 10002, Taiwan

  • National University Hospital

    Singapore, 119074, Singapore

  • Nippon Medical School Hospital

    Bunkyo City, Tokyo, 113-8603, Japan

  • Osaka University Hospital

    Suita, Osaka, 565-0871, Japan

  • Peking Union Medical College Hospital

    Beijing, Beijing Municipality, China

  • Peking University People's Hospital

    Beijing, Beijing Municipality, China

  • Princess Margaret Hospital

    Toronto, Ontario, M5G 2C1, Canada

  • Qilu Hospital of Shandong University

    Jinan, Shandong, China

  • Queen Mary Hospital

    Hong Kong, Hong Kong

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

    Shanghai, Shanghai Municipality, China

  • Samsung Medical Center

    Seoul, 06351, South Korea

  • Seoul National University Hospital

    Seoul, 03080, South Korea

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

    Seoul, 06591, South Korea

  • Severance Hospital, Yonsei University Health System

    Seoul, 03722, South Korea

  • Shaanxi Provincial People's Hospital

    Xi'an, Shaanxi, China

  • Shengjing Hospital of China Medical University

    Shenyang, Liaoning, China

  • Shin Kong Wu Ho-Su Memorial Hospital

    Taipei, Taipei City, 11101, Taiwan

  • Singapore General Hospital

    Singapore, 169608, Singapore

  • SoonChunHyang University Seoul Hospital

    Seoul, 04401, South Korea

  • St. Paul's Hospital

    Vancouver, British Columbia, V6Z 1Y6, Canada

  • Tainan Municipal An-Nan Hospital

    Tainan, Tainan City, 70965, Taiwan

  • Taipei Municipal Wan Fang Hospital

    Taipei, Taipei City, 11696, Taiwan

  • Taipei Veterans General Hospital

    Taipei, 11217, Taiwan

  • The First Affiliated Hospital of Soochow University

    Suzhou, Jiangsu, China

  • The First Affiliated Hospital, Chongqing Medical University

    Chongqing, Chongqing Municipality, China

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

    Hangzhou, Zhejiang, China

  • The Second Hospital of Tianjin Medical University

    Tianjin, Tianjin Municipality, 300211, China

  • Tokyo Medical University Hospital

    Shinjuku, Tokyo, 160-0023, Japan

  • Tri-Service General Hospital

    Taipei, 11449, Taiwan

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

    Wuhan, Hubei, China

  • University of Miyazaki Hospital

    Miyazaki, Miyazaki, 889-1692, Japan

  • University of Utah

    Salt Lake City, Utah, 84112, United States

  • University of Yamanashi Hospital

    Chūō, Yamanashi, 409-3898, Japan

  • Washington University School of Medicine - Division of Oncology

    St Louis, Missouri, 63110, United States

  • West China Hospital, Sichuan University

    Chengdu, Sichuan, China

  • Zhongnan Hospital of Wuhan University

    Wuhan, Hubei, China

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