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

Launched by XIAMEN AMOYTOP BIOTECH CO., LTD. · Aug 11, 2024

Keywords

ClinConnect Summary

This clinical trial is studying the effectiveness and safety of a medication called Peginterferon α-2b for patients with essential thrombocythemia (ET) who have not responded well to or cannot tolerate another treatment called hydroxyurea. Essential thrombocythemia is a condition where the body produces too many platelets, which can lead to complications like blood clots. The trial aims to enroll 27 participants aged 18 and older who have high-risk ET and have struggled with hydroxyurea treatment. Eligible participants will be randomly assigned to receive one of two doses of Peginterferon α-2b over a treatment period of nearly 48 weeks, followed by an extension period and a follow-up.

If you join the study, you will receive regular injections of Peginterferon α-2b and will be monitored closely for any side effects and how well the treatment works. To participate, you should not have any major health issues or other blood disorders, and you must meet specific criteria related to your previous treatments and current health status. This trial is currently recruiting participants, and it's important that anyone interested understands the study’s purpose and agrees to the requirements before signing up.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 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:
• • 1. 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);
• • 2. 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;
• • 3. 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:
• • 1. Male subjects: must agree to use reliable contraception from inform consent until 6 months following the last dose of the study drug.
• 2. Female subjects: Must meet at least one of the following conditions:
• • i) Women without childbearing potential; ii) Women of childbearing potential: no pregnant or breastfeed, negative in blood pregnancy test within 4 days prior to the first dosing, and must agree to use reliable contraception from inform consent until 6 months following the last dose of the study drug;
• • Subjects understand the objective, characteristic, method and possible adverse reactions of the study, voluntarily participate in this study, and sign informed consent.
• Exclusion Criteria:
• • 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;
• • Other situations that, in the investigator's opinion, not appropriate for inclusion.