A Study to Access Efficacy and Safety of P1101 in Chinese PV Patients Who Are Intolerant or Resistance to HU

NCT05485948 | Active Not Recruiting Phase 2

• 1 other identifier: A20-202
• Study Type: interventional
• Target: 49
• Locations: 1 country
• Sites: 14

Brief 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.

Conditions

Polycythemia Vera

Keywords

Ropeginterferon alpha-2b; Polycythemia Vera; Hydroxyurea; P1101

Outcome Measures

Primary Outcomes (1)

• The phlebotomy- or erythrocytapheresis-free CHR rate based on the central laboratory test results evaluation

  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.

  Week 24

Secondary Outcomes (1)

• The phlebotomy- or erythrocytapheresis-free CHR rate based on the evaluation of central laboratory test results

  Weeks 12, 36 and 52

Study Arms (1)

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.

Drug: Ropeginterferon alfa-2b

Interventions

Ropeginterferon alfa-2b DRUG

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.

Also known as: BESREMI

Treatment with P1101

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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);
• 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);

You may not qualify if:

• 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:
• 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;

Sponsors & Collaborators

• PharmaEssentia: lead

Study Sites (14)

Peking Union Medical College Hospital

Beijing, China

Location

Xiangya Hospital Central South University

Changsha, China

Location

The First Affiliated Hospital of Chongqing Medical Universit

Chongqing, China

Location

Nanfang Hospital affiliated to Southern Medical University

Guangzhou, China

Location

Anhui Provincial Hospital

Hefei, China

Location

Huashan Hospital affiliated to Fudan University

Shanghai, China

Location

Ruijin Hospital affiliated to Shanghai Jiaotong University

Shanghai, China

Location

Shenzhen Second People's Hospital

Shenzhen, China

Location

The First Affiliated Hospital of Soochow University

Suzhou, China

Location

Institute of Hematology &Blood Diseases Hospital ,Chinese Academy of medical science & Peking Union Medical College

Tianjin, China

Location

The Second Hospital of Tianjin Medical University

Tianjin, China

Location

Zhongnan Hospital affiliated to Wuhan University

Wuhan, China

Location

The First Affiliated Hospital Zhejiang University of Medicine

Zhejiang, China

Location

Henan Cancer Hospital

Zhengzhou, China

Location

Related Publications (2)

• Jin 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.

  PMID: 37344895 DERIVED

• Jin 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.

  PMID: 37129584 DERIVED

MeSH Terms

Conditions

Polycythemia Vera

Condition Hierarchy (Ancestors)

Bone Marrow Neoplasms; Hematologic Neoplasms; Neoplasms by Site; Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Myeloproliferative Disorders

Study Officials

• Jingjing Zhang

  PharmaEssentia

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 1, 2022
• First Posted: August 3, 2022
• Study Start: October 8, 2021
• Primary Completion: September 7, 2022
• Study Completion (Estimated): July 1, 2027
• Last Updated: August 3, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

CN (14)