Polycythemia VeraPhase 2NCT05485948
A Study to Access Efficacy and Safety of P1101 in Chinese PV Patients Who Are Intolerant or Resistance to HU
A Phase 2 study testing Ropeginterferon alfa-2b in people with Polycythemia Vera, run by PharmaEssentia at 14 sites in China.
Trial summary
This study is a phase II single-arm study designed to evaluate the efficacy and safety of P1101 in Chinese PV patients who are intolerance or resistance to HU. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Polycythemia Vera
- What is being tested?
- Ropeginterferon alfa-2b
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 49 participants
- Where
- 14 sitesChina
- Study dates
- 8 Oct 2021to Jul 2027
- Phase
- Phase 2
- 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 diagnosed with PV according to the 2016 World Health Organization (WHO) criteria;
- According to the 2020 Guidelines of Chinese Society of Clinical Oncology (CSCO) on Diagnosis and Treatment of Hematological Malignancies, PV patients who are HU resistant or intolerant must meet at least one of the following criteria;
- Drug resistance: 3 months of treatment at HU doses above 2 g/d
- Phlebotomy is still required to maintain Hct <45%;
- Failure to control the bone marrow proliferation (such as platelet count >400x10^9/L and white blood cell count >10x10^9/L);
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- spleen shrinkage of less than >50%;
- Intolerance
- At the minimum dose of HU required to achieve complete or partial clinical hematologic response, the absolute neutrophil count (ANC) <1x10^9/L or PLT <100x10^9/L or HGB <100 g/L;
- At any dose of HU treatment, the patient develops lower limb ulcers or other intolerable non-hematologic toxicity, such as skin mucosal manifestations (dark skin, teeth or nails; oral ulcers, mucositis; skin ulcers, rash, and other symptoms), gastrointestinal complaints (nausea, loss of appetite, indigestion, vomiting, abdominal pain, constipation, and other symptoms), pneumonia, fever, etc.
- Have not received interferon therapy previously; or have negative anti-P1101 binding antibody at screening, and the washout time between the last dose of interferon and the first dose of the study drug should not be shorter than 14 days;
- 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;
- Hemoglobin (HGB) ≥10 g/dL for females, and hemoglobin (HGB) ≥11 g/dL for males at screening;
- Neutrophil count ≥1.5x10^9/L at screening;
- Creatinine clearance rate ≥40 mL/min at screening (according to the Cockcroft-Gault formula);
- Males and females of childbearing potential, as well as all the females with a menopause duration of less than 2 years, must consent to use acceptable contraceptive methods within 28 days after the last dose of the study drug;
- The patient or the patient's guardian signs the written informed consent, and the patient is able to comply with the study requirements.
Who cannot join
- Patients with symptomatic splenomegaly;
- Any contraindications to interferon α or hypersensitivity to interferon α;
- With severe or serious diseases that the investigator determines may affect the patient's participation in this study;
- History of major organ transplantation;
- Pregnant or breastfeeding women;
- Patients with any other diseases that the investigator determines will affect the study results or may weaken the compliance to protocol, including but not limited to:
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- Prior or current autoimmune thyroid disease, but patients with oral thyroxine replacement therapy could be enrolled;
- Other documented autoimmune diseases (such as hepatitis, immune thrombocytopenia [ITP], scleroderma, psoriasis or any autoimmune arthritis);
- Clinically significant pulmonary infiltration, infectious pneumonia and non-infectious pneumonia, or a past history of interstitial pneumonia at screening;
- Active infection with systemic manifestations (e.g., presence of bacteria, fungi and human immunodeficiency virus [HIV], excluding hepatitis B [HBV] and/or hepatitis C [HCV] at screening);
- With evidence of severe retinopathy (e.g., cytomegalovirus [CMV]-induced retinitis, macular degeneration) or clinically significant eye diseases (due to diabetes or hypertension);
- With clinically significant depression or a history of depression;
- Previously had suicidal attempts or has any risk for suicidal tendency at screening.
- Poorly controlled diabetes;
- Thromboembolic complications caused by PV and active abdomina hemorrhage;
- History of any malignancy tumors (except stage 0 chronic lymphocytic leukemia [CLL], cured basal cell carcinoma, squamous cell carcinoma and superficial melanoma) in the past 5 years;
- History of alcohol or drug abuse in the past year;
- History or evidence of post polycythemia vera-myelofibrosis (PPV-MF), essential thrombocythemia, or any non-PV MPN;
- Presence of blast cells in the peripheral blood in the past 3 months;
- Use any investigational drugs or investigational drug combinations within 4 weeks before the first dose of the study drug, or have not yet recovered from the effects caused by any previously administered investigational drug.
- Unless with contraindications, acetylsalicylic acid will be considered as background treatment of this study. The following are contraindications to low-dose acetylsalicylic acid: history of allergies to Bayaspirin or any preparations of salicylic acid, peptic ulcer, bleeding tendency, aspirin-induced asthma (asthma attack caused by non-steroidal anti-inflammatory drugs, etc.) and its history. If any contraindications are found, other prophylactic anti-thrombotic drugs can be used.
Trial locations and contacts
14 sites in 1 country.
14 sites in 1 country
China14 sites
Peking Union Medical College Hospital
Beijing
Xiangya Hospital Central South University
Changsha
The First Affiliated Hospital of Chongqing Medical Universit
Chongqing
Nanfang Hospital affiliated to Southern Medical University
Guangzhou
Anhui Provincial Hospital
Hefei
Huashan Hospital affiliated to Fudan University
Shanghai
Ruijin Hospital affiliated to Shanghai Jiaotong University
Shanghai
Shenzhen Second People's Hospital
Shenzhen
The First Affiliated Hospital of Soochow University
Suzhou
Institute of Hematology &Blood Diseases Hospital ,Chinese Academy of medical science & Peking Union Medical College
Tianjin
The Second Hospital of Tianjin Medical University
Tianjin
Zhongnan Hospital affiliated to Wuhan University
Wuhan
The First Affiliated Hospital Zhejiang University of Medicine
Zhejiang
Henan Cancer Hospital
Zhengzhou
About this study
Official title: A Phase II Single-Arm Multicenter Study to Access Efficacy and Safety of P1101 in Chinese Polycythemia Vera Patients Who Are Intolerant or Resistance to Hydroxyurea
This study is a phase II single-arm study designed to evaluate the efficacy and safety of P1101 in Chinese PV patients who are intolerance or resistance to HU.
Read the detailed description
Eligible subjects will be treated with P1101 at a starting dose of 250 µg at Week 0, a medium dose of 350 µg at Week 2, a target dose of 500 µg at Week 4, and a maintenance dose of 500 µg from the subsequent week until Week 52. At Week 24 (Month 6), the primary study endpoint, i.e., the phlebotomy- or erythrocytapheresis-free CHR rate, will be analyzed.
For subjects who switch from previous HU treatment to P1101 (for subjects who are currently receiving HU treatment), the dose should be gradually reduced during the screening period (adjusted by the investigator based on clinical practice); the HU treatment should be ended within up to 4 weeks of P1101 treatment, and HU should be prohibited after 4 weeks of P1101 treatment.
During the treatment period, subject visits are scheduled for once every 2 weeks. The end of treatment (EOT) visit will be performed at Week 52 or early termination of the study, and the end of study (EOS) visit will be performed at 28 days after the EOT visit.
Data analyses will be performed after all subjects complete 24 weeks of treatment, and a clinical trial report written, and the new drug application is submitted accordingly. An end-of-study statistical analysis will be performed after all the subjects have completed all visits as per protocol.
- Conditions
- Polycythemia Vera
- Being tested
- Ropeginterferon alfa-2b
- Keywords
- Ropeginterferon alpha-2b, Polycythemia Vera, Hydroxyurea, P1101
- Medical subject headings
- Polycythemia Vera
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ropeginterferon alfa-2bDrug
Also called BESREMI
Initial dose of 250 µg at Week 0, a medium dose of 350 µg at Week 2, a target dose of 500 µg at Week 4, and a maintenance dose of 500 µg until Week 52.
| Study group | Role | What they receive |
|---|---|---|
| Treatment with P1101 | Experimental | Subjects who meet all the inclusion criteria and do not meet any of the exclusion criteria will start treatment with P1101. The study drug will be subcutaneously injected once every 2 weeks, with the target dose being 500 µg. Subjects will receive an initial dose of 250 µg at Week 0, a medium dose of 350 µg at Week 2, a target dose of 500 µg at Week 4, and a maintenance dose of 500 µg from the subsequent week until Week 52. If the dose needs to be adjusted due to safety or tolerability consideration, it is allowed to be adjusted to the previous dose, but the target dose is preferred to be maintained during the treatment period. Receives: Drug: Ropeginterferon alfa-2b |
What is being measured
The primary outcome is the main question the study is designed to answer.
The phlebotomy- or erythrocytapheresis-free CHR rate based on the central laboratory test results evaluation
- Hct <45% without the use of phlebotomy or erythrocytapheresis (without the use of phlebotomy or erythrocytapheresis within the previous 3 months);
- PLT count≤400x10^9/L;
- WBC count <10x10^9/L.
Measured: Week 24
Secondary outcomes (1)
The phlebotomy- or erythrocytapheresis-free CHR rate based on the evaluation of central laboratory test results
- Hct <45% without the use of phlebotomy or erythrocytapheresis (without the use of phlebotomy or erythrocytapheresis within the previous 3 months);
- PLT count≤400x10^9/L;
- WBC count <10x10^9/L.
Measured: Weeks 12, 36 and 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Aug 2022
- Study start8 Oct 2021
- Primary completion7 Sep 2022
- Study completionJul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Polycythemia VeraStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 49 participants (actual)
Sponsor and investigators
- Lead sponsor
- PharmaEssentia
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jingjing Zhang, Study director, PharmaEssentia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05485948
- Sponsor's protocol ID
- A20-202
- Status last verified
- Jul 2025
- First submitted
- 1 Aug 2022
- Last updated
- 3 Aug 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Qin A, Shimoda K, Suo S, Fu R, Kirito K, Wu D, Liao J, Chen H, Wu L, Su X, Gao Y, Sato T, Li Y, Zhang J, Shen W, Wang W, Zhang L, Jin J, Komatsu N. Population Pharmacokinetics-Pharmacodynamics and Exposure-Response of Ropeginterferon Alfa-2b in Chinese and Japanese Patients With Polycythemia Vera. Pharmacol Res Perspect. 2025 Jun;13(3):e70109. doi: 10.1002/prp2.70109.
PubMed 40313042Jin J, Zhang L, Qin A, Wu D, Shao Z, Bai J, Chen S, Duan M, Zhou H, Xu N, Zhang S, Zuo X, Du X, Wang L, Li P, Zhang X, Li Y, Zhang J, Wang W, Shen W, Zagrijtschuk O, Urbanski R, Sato T, Xiao Z. A new dosing regimen of ropeginterferon alfa-2b is highly effective and tolerable: findings from a phase 2 study in Chinese patients with polycythemia vera. Exp Hematol Oncol. 2023 Jun 21;12(1):55. doi: 10.1186/s40164-023-00415-0.
PubMed 37344895Jin J, Qin A, Zhang L, Shen W, Wang W, Zhang J, Li Y, Wu D, Xiao Z. A phase II trial to assess the efficacy and safety of ropeginterferon alpha-2b in Chinese patients with polycythemia vera. Future Oncol. 2023 Apr;19(11):753-761. doi: 10.2217/fon-2022-1141. Epub 2023 May 2.
PubMed 37129584
Study at a glance
From the registry record, last updated by the sponsor on 3 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Single-Arm Multicenter Study to Access Efficacy and Safety of P1101 in Chinese Polycythemia Vera Patients Who Are Intolerant or Resistance to Hydroxyurea
- ClinicalTrials.gov ID
- NCT05485948
- Acronym / sponsor ID
- A20-202
- Conditions
- Polycythemia Vera
- Keywords
- Ropeginterferon alpha-2b, Polycythemia Vera, Hydroxyurea, P1101
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 49 participants (actual)
- Lead sponsor
- PharmaEssentiaIndustry (pharma, biotech, devices)
- Study start
- 8 Oct 2021 (actual)
- Primary completion
- 7 Sep 2022 (actual)
- Study completion
- Jul 2027 (estimated)
- First posted
- 3 Aug 2022 (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 study is a phase II single-arm study designed to evaluate the efficacy and safety of P1101 in Chinese PV patients who are intolerance or resistance to HU.
Detailed description
Eligible subjects will be treated with P1101 at a starting dose of 250 µg at Week 0, a medium dose of 350 µg at Week 2, a target dose of 500 µg at Week 4, and a maintenance dose of 500 µg from the subsequent week until Week 52. At Week 24 (Month 6), the primary study endpoint, i.e., the phlebotomy- or erythrocytapheresis-free CHR rate, will be analyzed.
For subjects who switch from previous HU treatment to P1101 (for subjects who are currently receiving HU treatment), the dose should be gradually reduced during the screening period (adjusted by the investigator based on clinical practice); the HU treatment should be ended within up to 4 weeks of P1101 treatment, and HU should be prohibited after 4 weeks of P1101 treatment.
During the treatment period, subject visits are scheduled for once every 2 weeks. The end of treatment (EOT) visit will be performed at Week 52 or early termination of the study, and the end of study (EOS) visit will be performed at 28 days after the EOT visit.
Data analyses will be performed after all subjects complete 24 weeks of treatment, and a clinical trial report written, and the new drug application is submitted accordingly. An end-of-study statistical analysis will be performed after all the subjects have completed all visits as per protocol.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment with P1101 | Experimental | Drug: Ropeginterferon alfa-2bSubjects who meet all the inclusion criteria and do not meet any of the exclusion criteria will start treatment with P1101. The study drug will be subcutaneously injected once every 2 weeks, with the target dose being 500 µg. Subjects will receive an initial dose of 250 µg at Week 0, a medium dose of 350 µg at Week 2, a target dose of 500 µg at Week 4, and a maintenance dose of 500 µg from the subsequent week until Week 52. If the dose needs to be adjusted due to safety or tolerability consideration, it is allowed to be adjusted to the previous dose, but the target dose is preferred to be maintained during the treatment period. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Male or female patients aged ≥18 years at the time of signing the informed consent form;
- Patients diagnosed with PV according to the 2016 World Health Organization (WHO) criteria;
- According to the 2020 Guidelines of Chinese Society of Clinical Oncology (CSCO) on Diagnosis and Treatment of Hematological Malignancies, PV patients who are HU resistant or intolerant must meet at least one of the following criteria;
- Have not received interferon therapy previously; or have negative anti-P1101 binding antibody at screening, and the washout time between the last dose of interferon and the first dose of the study drug should not be shorter than 14 days;
- 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;
Key exclusion criteria (21 listed)
- Patients with symptomatic splenomegaly;
- Any contraindications to interferon α or hypersensitivity to interferon α;
- With severe or serious diseases that the investigator determines may affect the patient's participation in this study;
- History of major organ transplantation;
- Pregnant or breastfeeding women;
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: BESREMIInitial dose of 250 µg at Week 0, a medium dose of 350 µg at Week 2, a target dose of 500 µg at Week 4, and a maintenance dose of 500 µg until Week 52. | Drug | Treatment with P1101 |
Primary outcome
The phlebotomy- or erythrocytapheresis-free CHR rate based on the central laboratory test results evaluation
Time frame: Week 24
1. Hct <45% without the use of phlebotomy or erythrocytapheresis (without the use of phlebotomy or erythrocytapheresis within the previous 3 months); 2. PLT count≤400x10^9/L; 3. WBC count <10x10^9/L.
1 secondary outcome
The phlebotomy- or erythrocytapheresis-free CHR rate based on the evaluation of central laboratory test results
Time frame: Weeks 12, 36 and 52
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 3 Aug 2025.
- Derived from this study: PubMed 40313042Qin A, Shimoda K, Suo S, Fu R, Kirito K, Wu D, Liao J, Chen H, Wu L, Su X, Gao Y, Sato T, Li Y, Zhang J, Shen W, Wang W, Zhang L, Jin J, Komatsu N. Population Pharmacokinetics-Pharmacodynamics and Exposure-Response of Ropeginterferon Alfa-2b in Chinese and Japanese Patients With Polycythemia Vera. Pharmacol Res Perspect. 2025 Jun;13(3):e70109. doi: 10.1002/prp2.70109.
- Derived from this study: PubMed 37344895Jin J, Zhang L, Qin A, Wu D, Shao Z, Bai J, Chen S, Duan M, Zhou H, Xu N, Zhang S, Zuo X, Du X, Wang L, Li P, Zhang X, Li Y, Zhang J, Wang W, Shen W, Zagrijtschuk O, Urbanski R, Sato T, Xiao Z. A new dosing regimen of ropeginterferon alfa-2b is highly effective and tolerable: findings from a phase 2 study in Chinese patients with polycythemia vera. Exp Hematol Oncol. 2023 Jun 21;12(1):55. doi: 10.1186/s40164-023-00415-0.
- Derived from this study: PubMed 37129584Jin J, Qin A, Zhang L, Shen W, Wang W, Zhang J, Li Y, Wu D, Xiao Z. A phase II trial to assess the efficacy and safety of ropeginterferon alpha-2b in Chinese patients with polycythemia vera. Future Oncol. 2023 Apr;19(11):753-761. doi: 10.2217/fon-2022-1141. Epub 2023 May 2.
- Individual participant dataSharing planned: No
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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.
Jingjing Zhang study director
PharmaEssentia
14 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ropeginterferon alfa-2b in people with Polycythemia Vera, run by PharmaEssentia at 14 sites in China.
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 17 inclusion and 21 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 3 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in China. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
The phlebotomy- or erythrocytapheresis-free CHR rate based on the central laboratory test results evaluation (time frame: Week 24). 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 NCT05485948, which also lists 3 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 3 Aug 2025.