P1101 in Treating Patients With Early PMF or Overt PMF at Low or Intermediate-1 Risk

August 11, 2026 updated by: PharmaEssentia

A Randomized, Double-Blind, Placebo-Controlled Multicenter Phase III Study to Assess Efficacy and Safety of Ropeginterferon Alfa-2b (P1101) in Adult Patients With Pre-fibrotic/Early Primary Myelofibrosis (PMF) or Overt PMF at Low or Intermediate-1 Risk According to DIPSS Plus (HOPE-PMF): The Core Study and Its Extension Study

This is a phase 3 double-blind clinical trial arm to test Ropeginterferon alfa-2b (P1101) in adult patients with Primary Myelofibrosis (PMF) at early stage or low to medium risk.

Participants will receive the study drug/placebo bi-weekly and have an assessment visit every 4 weeks. The ratio of study drug to placebo group is 2:1.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

160

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5B 1W8
        • St. Michael's Hospital
      • Toronto, Ontario, Canada, M5G 2M9
        • Princess Margaret Hospital
    • Quebec
      • Montreal, Quebec, Canada
        • Jewish General Hospital
  • China
    • Beijing Municipality
      • Beijing, Beijing Municipality, China, 100044
        • Peking University People's Hospital
      • Beijing, Beijing Municipality, China, 100010
        • Peking Union Medical College Hospital
    • Chongqing Municipality
      • Chongqing, Chongqing Municipality, China
        • The Second Affiliated Hospital of AMU
    • Guangdong
      • Guangdong, Guangdong, China, 510410
        • Southern Hospital of Southern Medical University
    • Guangzhou
      • Guangzhou, Guangzhou, China, 510080
        • Guangdong Provincial People's Hospital
    • Heilongjiang
      • Harbin, Heilongjiang, China, 150010
        • Institute of Hematology and Oncology, Harbin The First Hospital
    • Henan
      • Zhengzhou, Henan, China, 450003
        • Henan Cancer Hospital
    • Hubei
      • Wuhan, Hubei, China
        • Union Hospital Tong Ji Medical College
    • Jiangsu
      • Nanjing, Jiangsu, China, 210024
        • Jiangsu Provincial Hospital
      • Suzhou, Jiangsu, China, 215005
        • The First Affiliated Hospital of Soochow University
    • Shandong
      • Jinan, Shandong, China, 250012
        • Qilu Hospital of Shandong University
    • Shanghai Municipality
      • Shanghai, Shanghai Municipality, China, 200030
        • Shanghai Sixth People's Hospital
      • Shanghai, Shanghai Municipality, China, 200020
        • Ruijin Hospital, Shanghai Jiaotong University School of Medicine
    • Sichuan
      • Chengdu, Sichuan, China, 610093
        • West China Hospital, Sichuan University
    • Tianjin Municipality
      • Tianjin, Tianjin Municipality, China, 301600
        • Hematology Hospital of Chinese Academy of Medical Sciences
    • Zhejiang
      • Hangzhou, Zhejiang, China, 310002
        • The First Affiliated Hospital, Zhejiang University School of Medicine
  • Hong Kong
    • Hong Kong
      • Hong Kong, Hong Kong, Hong Kong
        • Queen Mary Hospital
  • Japan
    • Chiba
      • Kamogawa, Chiba, Japan
        • Kameda General Hospital
    • Ehime
      • Matsuyama, Ehime, Japan
        • Ehime University Hospital
    • Hiroshima
      • Hiroshima, Hiroshima, Japan
        • Hiroshima Red Cross & Atomic-Bomb Survivors Hospital
    • Iwate
      • Morioka, Iwate, Japan
        • Iwate Prefectural Central Hospital
    • Kanagawa
      • Kamakura, Kanagawa, Japan
        • Shonan Kamakura General Hospital
    • Mie-ken
      • Tsu, Mie-ken, Japan
        • Mie University Hospital
    • Miyazaki
      • Miyazaki, Miyazaki, Japan
        • University of Miyazaki Hospital
    • Osaka
      • Hirakata, Osaka, Japan
        • Kansai Medical University Hospital
      • Osaka, Osaka, Japan
        • Osaka Metropolitan University Hospital
    • Shizuoka
      • Izunokuni, Shizuoka, Japan
        • Juntendo University Shizuoka Hospital
    • Tokyo
      • Tokyo, Tokyo, Japan
        • Juntendo University Hospital
      • Tokyo, Tokyo, Japan
        • Nerima Hikarigaoka Hospital
      • Tokyo, Tokyo, Japan
        • Nippon Medical School Hospital
      • Tokyo, Tokyo, Japan
        • Tokyo Medical University Hospital
    • Yamanashi
      • Chūō, Yamanashi, Japan, 409-3898
        • University of Yamanashi Hospital
  • Jordan
    • Amman Governorate
      • Amman, Amman Governorate, Jordan, 11941
        • King Hussein Cancer Center
  • Singapore
    • Singapore
      • Singapore, Singapore, Singapore
        • National University Hospital
      • Singapore, Singapore, Singapore
        • Parkway Cancer Center
      • Singapore, Singapore, Singapore
        • Singapore General Hospital
  • South Korea
    • Daegu
      • Daegu, Daegu, South Korea, 41944
        • Kyungpook National University Hospital
    • Gyeonggi-do
      • Seongnam-si, Gyeonggi-do, South Korea, 13620
        • Bundang Seoul National University Hospital
    • Jeollanam-do
      • Hwasun, Jeollanam-do, South Korea, 58128
        • Chonnam National University - Hwasun Hospital
    • Seoul
      • Seocho, Seoul, South Korea, 06591
        • Catholic University of Korea Seoul - St. Mary's Hospital
      • Seoul, Seoul, South Korea, 03080
        • Seoul National University Hospital
      • Seoul, Seoul, South Korea, 06351
        • Samsung Medical Center
      • Seoul, Seoul, South Korea, 03722
        • Severance Hospital - Shinchon
  • Taiwan
      • Chiayi City, Taiwan, 613016
        • Chang Gung Memorial Hospital - Chiayi
    • Kaohsiung
      • Kaohsiung City, Kaohsiung, Taiwan, 813414
        • Kaohsiung Veterans General Hospital
    • Keelung
      • Keelung, Keelung, Taiwan, 204
        • Chang Gung Memorial Hospital - Keelung
    • Taichung
      • Taichung, Taichung, Taiwan, 40447
        • China Medical University Hospital
    • Tainan
      • Tainan, Tainan, Taiwan, 710402
        • Chi Mei Medical Center
      • Tainan, Tainan, Taiwan, 736
        • Chi-Mei Hospital - Liouying Branch
    • Taipei
      • Taipei, Taipei, Taiwan, 100225
        • National Taiwan University Hospital
      • Taipei, Taipei, Taiwan, 11217
        • Taipei Veterans General Hospital
      • Taipei, Taipei, Taiwan, 111
        • Shin Kong Wu Ho-Su Memorial Hospital
      • Taipei, Taipei, Taiwan, 10449
        • Mackay Memorial Hospital - Taipei
      • Taipei, Taipei, Taiwan, 11259
        • Koo Foundation Sun Yat-Sen Cancer Center
      • Taipei, Taipei, Taiwan, 112
        • Cheng Hsin General Hospital
      • Taipei, Taipei, Taiwan, 116
        • Taipei Municipal Wan Fang Hospital
    • Taoyuan
      • Taoyuan, Taoyuan, Taiwan, 333
        • Chang Gung Memorial Hospital - Linkou
  • United States
    • California
      • Los Angeles, California, United States, 90033
        • USC Norris Comprehensive Cancer Center
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20007
        • Georgetown University Medical Center
    • Kansas
      • Kansas City, Kansas, United States, 66103
        • University of Kansas Medical Center (KUMC)
    • Maryland
      • Bethesda, Maryland, United States, 20817
        • American Oncology Partners of Maryland PA (Center for Cancer and Blood Disorders)
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Washington University School of Medicine
    • New York
      • New York, New York, United States, 10021
        • Weill Medical College of Cornell University
      • Stony Brook, New York, United States, 11794
        • Stony Brook University Medical Center
    • North Carolina
      • Charlotte, North Carolina, United States, 28204
        • Levine Cancer Institute
      • Durham, North Carolina, United States, 27710
        • Duke University
    • Ohio
      • Columbus, Ohio, United States, 43210
        • Ohio State University
    • Texas
      • Houston, Texas, United States, 77030
        • MD Anderson Cancer Center
    • Virginia
      • Richmond, Virginia, United States, 23284
        • Virginia Commonwealth University
    • Washington
      • Seattle, Washington, United States, 98195
        • University of Washington Medical Center - Fred Hutchinson Cancer Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Male or female patients aged ≥18 years at the time of signing the informed consent form;
  2. Patients with pre-fibrotic/early PMF (Pre-PMF) or overt primary myelofibrosis at low to intermediate-1 risk according to DIPSS plus, diagnosed according to WHO 2016 or 2022 classification;
  3. With good liver function at screening, which is defined as total bilirubin ≤1.5 × upper limit of normal (ULN), international normalized ratio (INR) ≤1.5 × ULN, albumin >3.5 g/dL, alanine aminotransferase (ALT) ≤2.0 × ULN, and aspartate aminotransferase (AST) ≤2.0 × ULN;
  4. Hgb ≥10.0 g/dL at screening;
  5. Neutrophil count ≥1.0 × 10^9/L at screening;
  6. Creatinine clearance rate ≥30 mL/min at screening (according to the Cockcroft-Gault formula);
  7. 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;
  8. Written informed consent obtained from the subject and ability for the subject to comply with the requirements of the study.

Exclusion Criteria:

  1. Any known contraindications to interferon α or hypersensitivity to interferon α;
  2. Patients with prior interferon therapy having poor tolerability or lack of efficacy to the previous interferon therapy per investigator's judgement;
  3. Patients with an ongoing cytoreduction (e.g., HU or IFN-α) at the time of screening if, in the Investigator's opinion, randomizing them into the placebo arm will lead to immediate rebound increase of peripheral blood counts and thus may jeopardize their health status;
  4. With severe or serious diseases that, in the Investigator's opinion, may affect the patient's participation in this study;
  5. History of major organ transplantation;
  6. Pregnant or breastfeeding women;
  7. Patients with any other diseases that will affect the study results or may weaken the compliance to protocol per the Investigator's judgment;
  8. Use any investigational drug <4 weeks prior to the first dose of study drug, or not recovered from effects of prior administration of any investigational drug.
  9. Eligible for JAK inhibitor therapy at screening.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Ropeginterferon alfa-2b (P1101)
Ropeginterferon alfa-2b (P1101) is administrated subcutaneously (SC) every two weeks (± 3 days) until 80 weeks.
Biological: Ropeginterferon alfa-2b

Pre-filled Syringe.

Dosage: up to 500mcg

Other Names:
  • P1101
Placebo Comparator: Placebo control
Placebo is administrated subcutaneously (SC) every two weeks (± 3 days) until 80 weeks.
Other: Placebo
Placebo is a look-alike substance with the intervention (Ropeginterferon alfa-2b) that contains no active drug.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants with Platelet count equal or less (≤) 400 × 10^9/L
Time Frame: 80 weeks
Platelet count ≤400 × 10^9/L is one of the criteria for clinically relevant complete hematologic response (CrCHR).
80 weeks
Number of Participants with White Blood Cells (WBC) count equal or less (≤) 10 × 10^9/L
Time Frame: 80 weeks
White Blood Cells (WBC) count ≤10 × 10^9/L is one of the criteria for clinically relevant complete hematologic response (CrCHR).
80 weeks
Number of Participants with Hemoglobin (Hgb) equal or greater (≧) 10.0 g/dL
Time Frame: 80 weeks
Peripheral blood: Hemoglobin (Hgb) ≧ 10.0 g/dL is one of the criteria for clinically relevant complete hematologic response (CrCHR).
80 weeks
Number of Participants absence of major thrombotic events
Time Frame: 80 weeks
The absence of major thrombotic events during the observation time frame is one of the criteria for clinically relevant complete hematologic response (CrCHR).
80 weeks
Number of Participants with no progression to secondary acute myeloid leukemia (AML).
Time Frame: 80 weeks
The absence of progression to secondary acute myeloid leukemia (AML) is one of the criteria for clinically relevant complete hematologic remission (CrCHR).
80 weeks
Number of Participants with no progression on the Total Symptom Score (TSS)
Time Frame: 80 weeks

The TSS score is utilized to evaluate clinical symptoms, which is based on the MFSAF Total Symptom Score (TSS) form v4.0.

No progression is defined as:

  • The participants who still have a TSS score equal to or less than (≤) 10 If the baseline score is ≤ 10.
  • The participants with a TSS score no increase than 50% If the baseline score is greater than (>) 10.
80 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

PharmaEssentia

Investigators

  • Study Director: Toshiaki Sato, MD/PhD, PharmaEssentia JP

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 18, 2025

Primary Completion (Estimated)

November 18, 2027

Study Completion (Estimated)

January 11, 2029

Study Registration Dates

First Submitted

June 10, 2024

First Submitted That Met QC Criteria

June 14, 2024

First Posted (Actual)

June 21, 2024

Study Record Updates

Last Update Posted (Actual)

August 13, 2026

Last Update Submitted That Met QC Criteria

August 11, 2026

Last Verified

July 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • A23-302

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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