Essential Thrombocytopenia Clinical Trial
Official title:
A Prospective, Single-center Clinical Trial of Pegylated Interferon Alfa-2b Versus Interferon Alfa Therapy in the Treatment of Childhood and Adolescent Essential Thrombocythemia
Objectives: To compare the efficacy and safety in childhood and adolescent patients (<20 years) diagnosed as essential thrombocythemia treated with the Pegylated Interferon Alfa-2b vs. Interferon Alfa. Study Design: A prospective, open-label, nonrandomized, single-center clinical trial
Status | Recruiting |
Enrollment | 40 |
Est. completion date | November 20, 2024 |
Est. primary completion date | October 20, 2024 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A to 19 Years |
Eligibility | Inclusion Criteria: - <20 years old - Male or Female - Diagnosis of essential thrombocythemia according to the 2016 WHO criteria. - Platelet count = 450 × 109 / L for more than 6 months(If the patient has JAK2 V617F, CALR or MPL gene mutation, the history may be less than 6 months) - Platelet count = 1000 × 109 / L or other therapeutic indications at screening. - The guardians has provided written informed consent prior to enrollment Exclusion Criteria: - Known to meet the criteria for primary myelofibrosis or polycythemia vera by 2016 WHO criteria - Presence of any life-threatening co-morbidity - Secondary thrombocytosis - Familial thrombocytosis - Resistance, or intolerance, or any contraindications to interferon - Interferon is used in the past 1 month before enrollment - Patients with previous or present thrombosis or active bleeding - WBC<4× 109 / L - HGB<110g/L - 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 diagnosed as diabetes with poor control - 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 |
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Institute of Hematology & Blood Diseases Hospital, China |
China,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Change in platelet count | Proportion of subjects with a continuous platelet count =600×109/L or decrease =50% (<1000×109/L ) (at least 12 weeks) from baseline during treatment will be evaluated. | From the start of study treatment (Day 1) up to the end of month 12 | |
Secondary | The complete hematologic response rates | To compare the complete hematologic response rates between different treatment groups | From the start of study treatment (Day 1) up to the end of month 12 | |
Secondary | Time to response in platelet count | Time to response in platelet count (<600×109/L) between different treatment groups | From the start of study treatment (Day 1) up to the end of month 12 | |
Secondary | Impact of therapy on key biomarkers | 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 (Day 1) up to the end of month 12 | |
Secondary | Incidence of major cardiovascular and thrombotic events | To estimate incidence of major cardiovascular and thrombotic events (defined as cardiovascular death, myocardial infarction, stroke, transient ischemic attack, pulmonary embolism, Budd Chiari syndrome, deep vein thrombosis, and any other clinically relevant thrombotic event) while on active treatment or observation following end of treatment between different treatment groups | From the start of study treatment (Day 1) up to the end of month 12 | |
Secondary | Incidence of development of myelodysplastic disorders, myelofibrosis, or leukemic transformation. | To estimate incidence of development of myelodysplastic disorders, myelofibrosis, or leukemic transformation between different treatment groups | From the start of study treatment (Day 1) up to the end of month 12 | |
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. | 12 months | |
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 (Day 1) up to the end of month 12 | |
Secondary | Impact of therapy on bone marrow histopathology (selectable) | To compare the proportion of subjects that display change on bone marrow histopathology | From the start of study treatment (Day 1) up to the end of month 12 | |
Secondary | Impact of therapy on cytogenetic abnormalities (selectable) | To compare the proportion of subjects that display change on cytogenetic abnormalities. | From the start of study treatment (Day 1) up to the end of month 12 | |
Secondary | Death while on active treatment or observation following end of treatment | To compare the incidence of death while on active treatment or observation following end of treatment | From the start of study treatment (Day 1) up to the end of month 12 | |
Secondary | Change in platelet count | Proportion of subjects with a continuous platelet count <1000×109/L in those with platelet count =1000×109/L before treatment (at least 12 weeks) will be evaluated. | From the start of study treatment (Day 1) up to the end of month 12 |
Status | Clinical Trial | Phase | |
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Recruiting |
NCT05395507 -
Pegylated Interferon Alfa-2b Versus Interferon Alfa Therapy in Adult Essential Thrombocythemia
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Phase 2 |