Primary MyelofibrosisPhase 3NCT06468033
P1101 in Treating Patients With Early PMF or Overt PMF at Low or Intermediate-1 Risk
A Phase 3 study testing Ropeginterferon alfa-2b in people with Primary Myelofibrosis and Myeloproliferative Neoplasm, run by PharmaEssentia at 73 sites in 9 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Myelofibrosis, Myeloproliferative Neoplasm
- What is being tested?
- Ropeginterferon alfa-2b
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 160 participants
- Where
- 73 sitesin 9 countries
- Study dates
- 18 Jul 2025to 11 Jan 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 patients aged ≥18 years at the time of signing the informed consent form;
- 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;
- 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;
- Hgb ≥10.0 g/dL at screening;
- Neutrophil count ≥1.0 × 10^9/L at screening;
- Creatinine clearance rate ≥30 mL/min at screening (according to the Cockcroft-Gault formula);
- 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;
- Written informed consent obtained from the subject and ability for the subject to comply with the requirements of the study.
Who cannot join
- Any known contraindications to interferon α or hypersensitivity to interferon α;
- Patients with prior interferon therapy having poor tolerability or lack of efficacy to the previous interferon therapy per investigator\'s judgement;
- 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;
- With severe or serious diseases that, in the Investigator's opinion, may affect the patient's participation in this study;
- History of major organ transplantation;
- Pregnant or breastfeeding women;
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- Patients with any other diseases that will affect the study results or may weaken the compliance to protocol per the Investigator's judgment;
- 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.
- Eligible for JAK inhibitor therapy at screening.
Trial locations and contacts
73 sites in 9 countries.
73 sites in 9 countries
China16 sites
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100010
Peking University People's Hospital
Beijing, Beijing Municipality, 100044
West China Hospital, Sichuan University
Chengdu, Sichuan, 610093
The Second Affiliated Hospital of AMU
Chongqing, Chongqing Municipality
Southern Hospital of Southern Medical University
Guangdong, Guangdong, 510410
Guangdong Provincial People's Hospital
Guangzhou, Guangzhou, 510080
The First Affiliated Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310002
Institute of Hematology and Oncology, Harbin The First Hospital
Harbin, Heilongjiang, 150010
Qilu Hospital of Shandong University
Jinan, Shandong, 250012
Jiangsu Provincial Hospital
Nanjing, Jiangsu, 210024
Ruijin Hospital, Shanghai Jiaotong University School of Medicine
Shanghai, Shanghai Municipality, 200020
Shanghai Sixth People's Hospital
Shanghai, Shanghai Municipality, 200030
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, 215005
Hematology Hospital of Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, 301600
Union Hospital Tong Ji Medical College
Wuhan, Hubei
Henan Cancer Hospital
Zhengzhou, Henan, 450003
Japan15 sites
University of Yamanashi Hospital
Chūō, Yamanashi, 409-3898
Kansai Medical University Hospital
Hirakata, Osaka
Hiroshima Red Cross & Atomic-Bomb Survivors Hospital
Hiroshima, Hiroshima
Juntendo University Shizuoka Hospital
Izunokuni, Shizuoka
Shonan Kamakura General Hospital
Kamakura, Kanagawa
Kameda General Hospital
Kamogawa, Chiba
Ehime University Hospital
Matsuyama, Ehime
University of Miyazaki Hospital
Miyazaki, Miyazaki
Iwate Prefectural Central Hospital
Morioka, Iwate
Osaka Metropolitan University Hospital
Osaka, Osaka
Juntendo University Hospital
Tokyo, Tokyo
Nerima Hikarigaoka Hospital
Tokyo, Tokyo
Nippon Medical School Hospital
Tokyo, Tokyo
Tokyo Medical University Hospital
Tokyo, Tokyo
Mie University Hospital
Tsu, Mie-ken
Taiwan14 sites
Chang Gung Memorial Hospital - Chiayi
Chiayi City, 613016
Kaohsiung Veterans General Hospital
Kaohsiung City, Kaohsiung, 813414
Chang Gung Memorial Hospital - Keelung
Keelung, Keelung, 204
China Medical University Hospital
Taichung, Taichung, 40447
Chi Mei Medical Center
Tainan, Tainan, 710402
Chi-Mei Hospital - Liouying Branch
Tainan, Tainan, 736
National Taiwan University Hospital
Taipei, Taipei, 100225
Mackay Memorial Hospital - Taipei
Taipei, Taipei, 10449
Shin Kong Wu Ho-Su Memorial Hospital
Taipei, Taipei, 111
Taipei Veterans General Hospital
Taipei, Taipei, 11217
Koo Foundation Sun Yat-Sen Cancer Center
Taipei, Taipei, 11259
Cheng Hsin General Hospital
Taipei, Taipei, 112
Taipei Municipal Wan Fang Hospital
Taipei, Taipei, 116
Chang Gung Memorial Hospital - Linkou
Taoyuan, Taoyuan, 333
United States13 sites
American Oncology Partners of Maryland PA (Center for Cancer and Blood Disorders)
Bethesda, Maryland, 20817
Levine Cancer Institute
Charlotte, North Carolina, 28204
Ohio State University
Columbus, Ohio, 43210
Duke University
Durham, North Carolina, 27710
MD Anderson Cancer Center
Houston, Texas, 77030
University of Kansas Medical Center (KUMC)
Kansas City, Kansas, 66103
USC Norris Comprehensive Cancer Center
Los Angeles, California, 90033
Weill Medical College of Cornell University
New York, New York, 10021
Virginia Commonwealth University
Richmond, Virginia, 23284
University of Washington Medical Center - Fred Hutchinson Cancer Center
Seattle, Washington, 98195
Washington University School of Medicine
St Louis, Missouri, 63110
Stony Brook University Medical Center
Stony Brook, New York, 11794
Georgetown University Medical Center
Washington D.C., District of Columbia, 20007
South Korea7 sites
Kyungpook National University Hospital
Daegu, Daegu, 41944
Chonnam National University - Hwasun Hospital
Hwasun, Jeollanam-do, 58128
Catholic University of Korea Seoul - St. Mary's Hospital
Seocho, Seoul, 06591
Bundang Seoul National University Hospital
Seongnam-si, Gyeonggi-do, 13620
Seoul National University Hospital
Seoul, Seoul, 03080
Severance Hospital - Shinchon
Seoul, Seoul, 03722
Samsung Medical Center
Seoul, Seoul, 06351
Canada3 sites
Jewish General Hospital
Montreal, Quebec
St. Michael's Hospital
Toronto, Ontario, M5B 1W8
Princess Margaret Hospital
Toronto, Ontario, M5G 2M9
Singapore3 sites
National University Hospital
Singapore, Singapore
Parkway Cancer Center
Singapore, Singapore
Singapore General Hospital
Singapore, Singapore
About this study
Official title: 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.
- Conditions
- Primary Myelofibrosis, Myeloproliferative Neoplasm
- Being tested
- Ropeginterferon alfa-2b, Placebo
- Keywords
- P1101, Primary Myelofibrosis, PMF, Ropeginterferon, PharmaEssentia, Myeloproliferative neoplasms, MPN, Pre-fibrotic, Low or Intermediate-1 Risk
- Medical subject headings
- Primary Myelofibrosis, 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
Pre-filled Syringe.
Dosage: up to 500mcg
PlaceboOther
Placebo is a look-alike substance with the intervention (Ropeginterferon alfa-2b) that contains no active drug.
| Study group | Role | What they receive |
|---|---|---|
| Ropeginterferon alfa-2b (P1101) | Experimental | Ropeginterferon alfa-2b (P1101) is administrated subcutaneously (SC) every two weeks (± 3 days) until 80 weeks. Receives: Biological: Ropeginterferon alfa-2b |
| Placebo control | Placebo comparator | Placebo is administrated subcutaneously (SC) every two weeks (± 3 days) until 80 weeks. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Platelet count equal or less (≤) 400 × 10^9/L
Platelet count ≤400 × 10^9/L is one of the criteria for clinically relevant complete hematologic response (CrCHR).
Measured: 80 weeks
Number of Participants with White Blood Cells (WBC) count equal or less (≤) 10 × 10^9/L
White Blood Cells (WBC) count ≤10 × 10^9/L is one of the criteria for clinically relevant complete hematologic response (CrCHR).
Measured: 80 weeks
Number of Participants with Hemoglobin (Hgb) equal or greater (≧) 10.0 g/dL
Peripheral blood: Hemoglobin (Hgb) ≧ 10.0 g/dL is one of the criteria for clinically relevant complete hematologic response (CrCHR).
Measured: 80 weeks
Number of Participants absence of major thrombotic events
The absence of major thrombotic events during the observation time frame is one of the criteria for clinically relevant complete hematologic response (CrCHR).
Measured: 80 weeks
Number of Participants with no progression to secondary acute myeloid leukemia (AML).
The absence of progression to secondary acute myeloid leukemia (AML) is one of the criteria for clinically relevant complete hematologic remission (CrCHR).
Measured: 80 weeks
Number of Participants with no progression on the Total Symptom Score (TSS)
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 (\&amp;gt;) 10.
Measured: 80 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jun 2024
- Study start18 Jul 2025
- Primary completion18 Nov 2027 (estimated)
- Study completion11 Jan 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary MyelofibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 160 participants (actual)
Sponsor and investigators
- Lead sponsor
- PharmaEssentia
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Toshiaki Sato, MD/PhD, Study director, PharmaEssentia JP
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06468033
- Sponsor's protocol ID
- A23-302
- Status last verified
- Jul 2026
- First submitted
- 10 Jun 2024
- Last updated
- 13 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06468033
- Acronym / sponsor ID
- A23-302
- Conditions
- Primary Myelofibrosis, Myeloproliferative Neoplasm
- Keywords
- P1101, Primary Myelofibrosis, PMF, Ropeginterferon, PharmaEssentia, Myeloproliferative neoplasms, MPN, Pre-fibrotic
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 160 participants (actual)
- Lead sponsor
- PharmaEssentiaIndustry (pharma, biotech, devices)
- Study start
- 18 Jul 2025 (actual)
- Primary completion
- 18 Nov 2027 (estimated)
- Study completion
- 11 Jan 2029 (estimated)
- First posted
- 21 Jun 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ropeginterferon alfa-2b (P1101) | Experimental | Biological: Ropeginterferon alfa-2bRopeginterferon alfa-2b (P1101) is administrated subcutaneously (SC) every two weeks (± 3 days) until 80 weeks. |
| Placebo control | Placebo comparator | Other: PlaceboPlacebo is administrated subcutaneously (SC) every two weeks (± 3 days) until 80 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Male or female patients aged ≥18 years at the time of signing the informed consent form;
- 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;
- 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;
- Hgb ≥10.0 g/dL at screening;
- Neutrophil count ≥1.0 × 10^9/L at screening;
Key exclusion criteria (9 listed)
- Any known contraindications to interferon α or hypersensitivity to interferon α;
- Patients with prior interferon therapy having poor tolerability or lack of efficacy to the previous interferon therapy per investigator\'s judgement;
- 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;
- With severe or serious diseases that, in the Investigator's opinion, may affect the patient's participation in this study;
- History of major organ transplantation;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Ropeginterferon alfa-2bAlso: P1101Pre-filled Syringe. Dosage: up to 500mcg | Biological | Ropeginterferon alfa-2b (P1101) |
| PlaceboPlacebo is a look-alike substance with the intervention (Ropeginterferon alfa-2b) that contains no active drug. | Other | Placebo control |
Primary outcome
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).
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).
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).
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).
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).
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 (\&amp;gt;) 10.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Aug 2026.
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Study of Selinexor in Combination With Ruxolitinib in Myelofibrosis
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Study of Momelotinib in Combination With Luspatercept in Participants With Transfusion Dependent Myelofibrosis
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Toshiaki Sato, MD/PhD study director
PharmaEssentia JP
73 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Ropeginterferon alfa-2b in people with Primary Myelofibrosis and Myeloproliferative Neoplasm, run by PharmaEssentia at 73 sites in 9 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 8 inclusion and 9 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 13 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 73 sites in China, Japan, Taiwan, United States, South Korea and 4 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. Some participants in this study may receive a placebo. 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?
Number of Participants with Platelet count equal or less (≤) 400 × 10^9/L (time frame: 80 weeks). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 13 Aug 2026.