Essential Thrombocytopenia Clinical Trial
Official title:
A Prospective, Multicenter, Randomized Controlled Clinical Trial of Pegylated Interferon Alfa-2b Versus Interferon Alfa Therapy in the Treatment of Adult Essential Thrombocythemia
Objectives: To compare the efficacy and safety in Adult patients (≥18 years) diagnosed as essential thrombocythemia treated with the Pegylated Interferon Alfa-2b vs. Interferon Alfa. Study Design: A prospective, open-label, multicenter, randomized controlled clinical trial.
Status | Recruiting |
Enrollment | 194 |
Est. completion date | June 30, 2025 |
Est. primary completion date | March 31, 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | Inclusion Criteria: - =18 years old. - Male or Female. - Diagnosis of essential thrombocythemia according to the 2016 World Health Organization criteria. - Those who have not use interferon within 4 weeks before the first medication. - Patients with indications for cytoreductive therapy. - Men and women with reproductive potential, as well as all women with menopause less than 2 years, must agree to use acceptable contraceptive methods until 28 days after the last dose of study drug, and women must agree not to breastfeed during the study period. - Voluntary written informed consent. Exclusion Criteria: - Resistance, or intolerance, or any contraindications to interferon. - Patients with active thrombosis or active bleeding. - Neutrophil count < 1.0x10^9/L. - Hemoglobin < 11g/dL for male, or < 10g/dL for female. - Poor control of thyroid dysfunction. - Patients with a prior malignancy within the last 3 years. - Patients with severe cardiac or pulmonary dysfunction. - Severe renal damage (creatinine clearance < 30 ml / min). - Severe liver dysfunction (ALT or AST > 2.5×ULN). - Patients with hepatitis B virus, hepatitis C virus replication or HIV infection. - Patients with a history of drug / alcohol abuse (within 2 years before the study). - Patients that have participated in other experimental researches within one month before enrollment. - History of psychiatric disorder. - Any other circumstances that the investigator considers that the patient is not suitable to participate in the trial. |
Country | Name | City | State |
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China | Institute of Hematology & Blood Diseases Hospital | Tianjin | Tianjin |
Lead Sponsor | Collaborator |
---|---|
Institute of Hematology & Blood Diseases Hospital, China |
China,
Type | Measure | Description | Time frame | Safety issue |
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Other | Changes of immune cell subgroups | The proportion of T lymphocyte, B lymphocyte and dendritic cell subsets and the changes of gene expression profile of these cells will be analyzed before and after treatment. | From the start of study treatment (Week 0) up to the end of Week 52. | |
Primary | Complete hematological remission (CHR) rates | The CHR rates defined as European Leukemia Net will be compared between the two groups. | From the start of study treatment (Week 0) up to the end of Week 52 | |
Secondary | CHR rates at week 24 and 36 | The CHR rates at week 24 and 36 will be compared between the two groups | From the start of study treatment (Week 0) up to the end of Week 24 and Week 36, respectively | |
Secondary | Time to CHR | The time of reaching CHR will be compared between the two groups | From the start of study treatment (Week 0) up to the end of Week 52. | |
Secondary | The proportion of patients crossed to the contralateral group | The proportion of patients crossed to the contralateral group will be compared between the two groups | From the start of study treatment (Week 0) up to the end of Week 52. | |
Secondary | The CHR rates after crossover | The CHR rates within 52 weeks after crossover | From the start of study treatment (Week 0) up to the end of Week 52. | |
Secondary | Impact of therapy on driver mutations | To compare the proportion of subjects that display change on key biomarkers of the disease- JAK2V617F, CALR, MPL mutations. | From the start of study treatment (Week 0) up to the end of Week 52. | |
Secondary | Impact of therapy on non-driver mutations | To compare the proportion of subjects that display change on key non-driver biomarkers of the disease-DNMT3A, ASXL1, TET2 or other mutations. | From the start of study treatment (Week 0) up to the end of Week 52. | |
Secondary | Change of splenomegaly | To compare the proportion of subject with improvement and no progress rate of splenomegaly between the two groups. | From the start of study treatment (Week 0) up to the end of Week 52. | |
Secondary | Change of bone marrow pathology | To compare the rate of patients with improvement and no progress rate of bone marrow pathology between the two groups | From the start of study treatment (Week 0) up to the end of Week 52. | |
Secondary | The incidence of major thrombotic events | To compare the incidence of major thrombotic events between the two groups. | From the start of study treatment (Week 0) up to the end of Week 52. | |
Secondary | The incidence of major bleeding events | To compare the incidence of major bleeding events between the two groups. | From the start of study treatment (Week 0) up to the end of Week 52. | |
Secondary | The incidence of progressing to bone marrow fibrosis | The incidence of progressing to bone marrow fibrosis will be compared between the two groups | From the start of study treatment (Week 0) up to the end of Week 52. | |
Secondary | The incidence of progressing to acute leukemia | The incidence of progressing to acute leukemia will be compared between the two groups | From the start of study treatment (Week 0) up to the end of Week 52. | |
Secondary | Change in Myeloproliferative Neoplasm Symptom Assessment Form total symptom score | To compare the proportion of subjects that display change in Myeloproliferative Neoplasm Symptom Assessment Form total symptom score (0-100 scores, higher scores mean a worse outcome) between different treatment groups. | From the start of study treatment (Week 0) up to the end of Week 52. | |
Secondary | Change of quality of life | Compare the rate of patients with improvement in quality of life between the two groups (assessed by EORTC quality of life scale QLQ-C30 V3.0 questionnaire). | From the start of study treatment (Week 0) up to the end of Week 52. | |
Secondary | Change of microcirculation disturbance | The rate of patients with improvement in microcirculation disturbance (such as pruritus, headache, dizziness, chest tightness, erythematous limb pain and limb paresthesia) will be compared between the two groups | From the start of study treatment (Week 0) up to the end of Week 52. | |
Secondary | Specific pre-defined toxicity | To compare incidence of specific pre-defined toxicity including fatigue, flu-like symptoms, dizziness, injection site necrosis, dyspnea, pain, depression, blurred Vision, insomnia, anorexia, weight Loss, weakness, pruritis, sweating, fever, decreased Libido, hot Flashes, flushing. | From the start of study treatment (Week 0) up to the end of Week 52. |
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT04226950 -
Pegylated Interferon Alfa-2b Versus Interferon Alfa Therapy in Childhood and Adolescent Essential Thrombocythemia
|
Phase 2 |