Peginterferon α-2b Injection for Hydroxyurea Resistant or Intolerant ET

NCT06552429 | Recruiting Phase 2

• 1 other identifier: P2b-4-3-002
• Study Type: interventional
• Target: 27
• Locations: 1 country
• Sites: 8

Brief Summary

This is a multicenter, randomized, open-label Phase 2 clinical study. It is aimed to enroll 27 essential thrombocytopenia (ET) patients who are resistant to or intolerant of hydroxyurea(HU). Eligible patients will be randomized to receive either Peginterferon α-2b 135 mcg or Peginterferon α-2b 180 mcg at a ratio of 1:2, and all subjects will go through a target treatment period (Weeks 1 \~ Week 48), an extension treatment period (Weeks 49 \~ Week 96) and a follow-up period (Weeks 97 \~ Week 100). Pharmacokinetics, safety, efficacy will be evaluated.

Conditions

Essential Thrombocythemia

Keywords

Essential Thrombocythemia; Peginterferon α-2b; hydroxyurea resistant; hydroxyurea intolerant

Outcome Measures

Primary Outcomes (7)

• Maximum concentration (Cmax)

  week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.

• Time to maximum concentration (Tmax)

  week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.

• Area under the plasma concentration-time curve

  week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.

• Apparent volume of distribution after oral administration (Vz/f)

  week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.

• Apparent plasma clearance (CL/F)

  week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.

• Plasma elimination half-life (t1/2)

  week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.

• Relationship between exposure and the effect (desired-effectiveness or undesirable-toxicity) in a pharmacokinetic model and pharmacodynamic model.

  up to 96 weeks.

Secondary Outcomes (17)

• Rate of complete hematological remission.

  Week 24, 36, 48, 60, 72, 84, 96.

• Platelet counts change from baseline.

  Week 12, 24, 36, 48, 60, 72, 84, 96.

• White blood cell counts change from baseline.

  Week 12, 24, 36, 48, 60, 72, 84, 96.

• Complete remission rate.

  Week 24, 36, 48, 60, 72, 84, 96.

• Time to complete remission from baseline.

  Week 48, 96.

Study Arms (2)

Peginterferon α-2b 135 mcg dose group

EXPERIMENTAL

Drug: Peginterferon α-2b injection

Peginterferon α-2b 180 mcg dose group

EXPERIMENTAL

Drug: Peginterferon α-2b injection

Interventions

Peginterferon α-2b injection DRUG

Peginterferon α-2b injection, 135 mcg, s.c., once a week, during the targeted treatment period (the first 48 week), peginterferon α-2b dose is depended on the patient's response and tolerability during the extension treatment (week 49 to week 96).

Peginterferon α-2b 135 mcg dose group

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 subjects, aged greater or equal to 18 years old at screening;
• Subjects diagnosed as high-risk ET according to the World Health Organization (WHO) 2016 criteria：1) who is older than 60 years and JAK2V617F positive at screening, 2) or who previously suffered from disease-related thrombosis or hemorrhage;
• Subjects who have previously received HU for ET, and the time interval between the last HU dose and the first dose of the study drug should not be less than 7 days;
• Interferon treatment-naïve, and for those who have previously received interferon the the time interval between the last dose of interferon and randomization should not be less than 1 month;
• Patients with confirmed hydroxyurea resistance or intolerant, as at least one of the following criteria is met:
• Platelet count remain greater than 600×10\^9 /L after at least 3 months of HU treatment at a dose ≥2g/d (dose ≥2.5 g/d if subject weight \> 80 kg);
• Platelet count greater than 400\*10\^9/L while white blood cell (WBC) count lower than 2.5\*10\^9/L, or platelet count greater than 400\*10\^9 /L while hemoglobin lower than 100 g/L at any dose of HU;
• Presence of HU-related toxicities at any dose of HU: e.g. ulcers in legs, or any unacceptable skin mucosal manifestations or fever;
• Platelet counts \> 450\*10\^9/L at screening;
• Neutrophil count ≥1.0\*10\^9/L at screening;
• Haemoglobin ≥11 g/dL at screening for males and 10 g/dL for females at screening;
• There is no serious function damage in liver and kidney: total bilirubin ≤1.5 upper limit of normal (ULN), alanine aminotransferase≤2.0 ULN, aspartate aminotransferase≤2.0 ULN, prothrombin time is prolonged by less than 4 seconds, Creatinine clearance ≥50 mL/min (according to Cockcroft-Gault formula) at screening;
• Both male and female subjects must agree take an appropriate contraceptive method, including:
• Male subjects: must agree to use reliable contraception from inform consent until 6 months following the last dose of the study drug.
• Female subjects: Must meet at least one of the following conditions:

You may not qualify if:

• History of any other myeloproliferative tumors, or evidence of the presence of any other myeloproliferative tumors;
• Contraindications or hypersensitivities to interferons of any of its excipients;
• Severe medical conditions or serious comorbidities that the investigators determined could jeopardize the safety or protocol adherence, e.g. New York Heart Association \[NYHA\] Class III-IV, congestive heart failure, symptomatic arrhythmias，pulmonary hypertension;
• History of major organ transplantation;
• Documented autoimmune disease or history of autoimmune disease at screening, e.g. medication un-controlled thyroid dysfunction, autoimmune hepatitis, idiopathic thrombocytopenic purpura, scleroderma, psoriasis, or any autoimmune arthritis;
• Clinically significant pulmonary infiltration, infectious pneumonia, and non-infectious pneumonia at screening that, in the investigator's opinion, would jeopardize the safety of the subject or their compliance with the protocol;
• Infection with systemic clinical manifestations at screening, e.g., bacteria, fungi, human immunodeficiency virus, excluding hepatitis B and/or C;
• Evidence of severe retinopathy, e.g., cytomegalovirus retinitis, symptomatic macular degeneration, or clinically significant eye disease, e.g. due to diabetes mellitus or hypertension;
• Diagnosed clinically significant depression or a history of depression and, in the investigator's opinion, previous suicide attempts or at any risk of suicide at screening;
• Diagnosed clinically significant neurological disease or a history of clinically significant neurological disease, except for a history of stable cerebral thrombosis or cerebral hemorrhage;
• History of any malignancy within 5 years (except stage 0 chronic lymphocytic leukemia, basal cell carcinoma, squamous cell carcinoma, and superficial melanoma);
• A history of alcohol or drug abuse within 1 year;
• Have used any investigational drug within 4 weeks prior to first dose of investigational drug, or not recovered from the effects of prior investigational drug administration;

Sponsors & Collaborators

• Xiamen Amoytop Biotech Co., Ltd.: lead

Study Sites (8)

Peking Union Hospital, Chinese Academy of Medical Sciences

Beijing, China

RECRUITING

Peking University People's Hospital

Beijing, China

RECRUITING

Union Hospital affiliated to Fujian Medical University

Fujian, China

RECRUITING

Nanfang Hospital, Southern Medical University

Guangzhou, China

RECRUITING

Harbin First Hospital

Harbin, China

RECRUITING

Henan Cancer Hospital

Henan, China

RECRUITING

Ruijin Hospital, Shanghai Jiao Tong University School of Medicine

Shanghai, China

RECRUITING

The First Affiliated Hospital of Zhejiang University School of Medicine

Zhejiang, China

RECRUITING

MeSH Terms

Conditions

Thrombocythemia, Essential

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombocytosis; Blood Platelet Disorders; Myeloproliferative Disorders; Bone Marrow Diseases; Hemorrhagic Disorders

Study Officials

• Lei Zhang

  Chinese Academy of Medical Sciences

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Lei Zhang

CONTACT

13502118379; zhanglei1@ihcams.ac.cn

Rongfeng Fu

CONTACT

13502118379; furongfeng@ihcams.ac.cn

Study Design

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

Study Record Dates

• First Submitted: July 27, 2024
• First Posted: August 14, 2024
• Study Start: August 29, 2024
• Primary Completion: October 26, 2025
• Study Completion (Estimated): September 1, 2027
• Last Updated: November 17, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

CN (8)