Back to resultsPegylated Interferon Alfa-2b Versus Interferon Alfa Therapy in Adult Essential Thrombocythemia
Primary Purpose
Essential Thrombocytopenia
Status
Recruiting
Phase
Phase 4
Locations
China
Study Type
Interventional
Intervention
Recombinant Interferon Alpha
Pegylated interferon alfa-2b
Sponsored by
About this trial
This is an interventional treatment trial for Essential Thrombocytopenia focused on measuring Essential Thrombocytopenia, Interferon Alfa, Pegylated Interferon Alfa-2b, Efficacy, Safety
Eligibility Criteria
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.
Sites / Locations
- Institute of Hematology & Blood Diseases HospitalRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
Experimental
Arm Label
Recombinant Interferon Alpha
Pegylated Interferon Alfa-2b
Arm Description
Recombinant Interferon Alpha, with an initial dose of 300 wu three times a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available.
Pegylated Interferon Alfa-2b, with an initial dose of 135 ug at week 0 , and then 180 ug once a week from week 1 to week 52.
Outcomes
Primary Outcome Measures
Complete hematological remission (CHR) rates
The CHR rates defined as European Leukemia Net will be compared between the two groups.
Secondary Outcome Measures
CHR rates at week 24 and 36
The CHR rates at week 24 and 36 will be compared between the two groups
Time to CHR
The time of reaching CHR will be compared between the two groups
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
The CHR rates after crossover
The CHR rates within 52 weeks after crossover
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.
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.
Change of splenomegaly
To compare the proportion of subject with improvement and no progress rate of splenomegaly between the two groups.
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
The incidence of major thrombotic events
To compare the incidence of major thrombotic events between the two groups.
The incidence of major bleeding events
To compare the incidence of major bleeding events between the two groups.
The incidence of progressing to bone marrow fibrosis
The incidence of progressing to bone marrow fibrosis will be compared between the two groups
The incidence of progressing to acute leukemia
The incidence of progressing to acute leukemia will be compared between the two groups
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.
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).
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
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.
Full Information
NCT ID
NCT05395507
First Posted
May 10, 2022
Last Updated
July 18, 2022
Sponsor
Institute of Hematology & Blood Diseases Hospital, China
1. Study Identification
Unique Protocol Identification Number
NCT05395507
Brief Title
Pegylated Interferon Alfa-2b Versus Interferon Alfa Therapy in Adult Essential Thrombocythemia
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
Study Type
Interventional
2. Study Status
Record Verification Date
July 2022
Overall Recruitment Status
Recruiting
Study Start Date
June 1, 2022 (Actual)
Primary Completion Date
March 31, 2025 (Anticipated)
Study Completion Date
June 30, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Institute of Hematology & Blood Diseases Hospital, China
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
Brief Summary
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.
Detailed Description
This is a prospective, open-label, multicenter, randomized controlled clinical trial between Interferon Alfa and Pegylated Interferon Alfa-2b in adult essential thrombocythemia (≥18 years).
Patients will be randomly divided into the following two treatment groups: 1. Recombinant Interferon Alpha, with an initial dose of 300 wu three times a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available. 2. Pegylated Interferon Alfa-2b, with an initial dose of 135 ug at week 0 , and then 180 ug once a week from week 1 to week 52.
The dosage will be adjusted according to the results of laboratory examinations and patient tolerance. The patient will be transferred to the other group if intolerance or resistance occurs.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Essential Thrombocytopenia
Keywords
Essential Thrombocytopenia, Interferon Alfa, Pegylated Interferon Alfa-2b, Efficacy, Safety
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
194 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Recombinant Interferon Alpha
Arm Type
Active Comparator
Arm Description
Recombinant Interferon Alpha, with an initial dose of 300 wu three times a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available.
Arm Title
Pegylated Interferon Alfa-2b
Arm Type
Experimental
Arm Description
Pegylated Interferon Alfa-2b, with an initial dose of 135 ug at week 0 , and then 180 ug once a week from week 1 to week 52.
Intervention Type
Drug
Intervention Name(s)
Recombinant Interferon Alpha
Intervention Description
Recombinant Interferon Alpha, with an initial dose of 300 wu three times a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available.
Intervention Type
Drug
Intervention Name(s)
Pegylated interferon alfa-2b
Intervention Description
Pegylated Interferon Alfa-2b, with an initial dose of 135 ug at week 0 , and then 180 ug once a week from week 1 to week 52.
Primary Outcome Measure Information:
Title
Complete hematological remission (CHR) rates
Description
The CHR rates defined as European Leukemia Net will be compared between the two groups.
Time Frame
From the start of study treatment (Week 0) up to the end of Week 52
Secondary Outcome Measure Information:
Title
CHR rates at week 24 and 36
Description
The CHR rates at week 24 and 36 will be compared between the two groups
Time Frame
From the start of study treatment (Week 0) up to the end of Week 24 and Week 36, respectively
Title
Time to CHR
Description
The time of reaching CHR will be compared between the two groups
Time Frame
From the start of study treatment (Week 0) up to the end of Week 52.
Title
The proportion of patients crossed to the contralateral group
Description
The proportion of patients crossed to the contralateral group will be compared between the two groups
Time Frame
From the start of study treatment (Week 0) up to the end of Week 52.
Title
The CHR rates after crossover
Description
The CHR rates within 52 weeks after crossover
Time Frame
From the start of study treatment (Week 0) up to the end of Week 52.
Title
Impact of therapy on driver mutations
Description
To compare the proportion of subjects that display change on key biomarkers of the disease- JAK2V617F, CALR, MPL mutations.
Time Frame
From the start of study treatment (Week 0) up to the end of Week 52.
Title
Impact of therapy on non-driver mutations
Description
To compare the proportion of subjects that display change on key non-driver biomarkers of the disease-DNMT3A, ASXL1, TET2 or other mutations.
Time Frame
From the start of study treatment (Week 0) up to the end of Week 52.
Title
Change of splenomegaly
Description
To compare the proportion of subject with improvement and no progress rate of splenomegaly between the two groups.
Time Frame
From the start of study treatment (Week 0) up to the end of Week 52.
Title
Change of bone marrow pathology
Description
To compare the rate of patients with improvement and no progress rate of bone marrow pathology between the two groups
Time Frame
From the start of study treatment (Week 0) up to the end of Week 52.
Title
The incidence of major thrombotic events
Description
To compare the incidence of major thrombotic events between the two groups.
Time Frame
From the start of study treatment (Week 0) up to the end of Week 52.
Title
The incidence of major bleeding events
Description
To compare the incidence of major bleeding events between the two groups.
Time Frame
From the start of study treatment (Week 0) up to the end of Week 52.
Title
The incidence of progressing to bone marrow fibrosis
Description
The incidence of progressing to bone marrow fibrosis will be compared between the two groups
Time Frame
From the start of study treatment (Week 0) up to the end of Week 52.
Title
The incidence of progressing to acute leukemia
Description
The incidence of progressing to acute leukemia will be compared between the two groups
Time Frame
From the start of study treatment (Week 0) up to the end of Week 52.
Title
Change in Myeloproliferative Neoplasm Symptom Assessment Form total symptom score
Description
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.
Time Frame
From the start of study treatment (Week 0) up to the end of Week 52.
Title
Change of quality of life
Description
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).
Time Frame
From the start of study treatment (Week 0) up to the end of Week 52.
Title
Change of microcirculation disturbance
Description
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
Time Frame
From the start of study treatment (Week 0) up to the end of Week 52.
Title
Specific pre-defined toxicity
Description
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.
Time Frame
From the start of study treatment (Week 0) up to the end of Week 52.
Other Pre-specified Outcome Measures:
Title
Changes of immune cell subgroups
Description
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.
Time Frame
From the start of study treatment (Week 0) up to the end of Week 52.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Lei Zhang, MD
Phone
8602223909240
Email
zhanglei1@ihcams.ac.cn
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Lei Zhang, MD
Organizational Affiliation
Institute of Hematology & Blood Diseases Hospital, China
Official's Role
Principal Investigator
Facility Information:
Facility Name
Institute of Hematology & Blood Diseases Hospital
City
Tianjin
State/Province
Tianjin
ZIP/Postal Code
300020
Country
China
Individual Site Status
Recruiting
12. IPD Sharing Statement
Learn more about this trial
Pegylated Interferon Alfa-2b Versus Interferon Alfa Therapy in Adult Essential Thrombocythemia
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