Back to resultsPegylated Interferon Alfa-2b Versus Interferon Alfa Therapy in Childhood and Adolescent 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 Thrombocythemia, Childhood, Interferon Alfa, Pegylated Interferon Alfa-2b, Adolescent
Eligibility Criteria
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
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 twice a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available, and the specific dose will be determined by the researchers.
Pegylated Interferon Alfa-2b, with an initial dose of 135 ug once a week (body surface area < 1.73 m2) or 180 ug once a week ( body surface area≥1.73 m2).
Outcomes
Primary Outcome Measures
Change in platelet count
Proportion of subjects with a continuous platelet count ≤600×109/L or decrease ≥50% (<1000×109/L ) from baseline during treatment will be evaluated.
Secondary Outcome Measures
The complete hematologic response rates
To compare the complete hematologic response rates between different treatment groups
Time to response in platelet count
Time to response in platelet count (<600×109/L) between different treatment groups
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.
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
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
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.
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.
Impact of therapy on bone marrow histopathology
To compare the proportion of subjects that display change on bone marrow histopathology
Impact of therapy on cytogenetic abnormalities
To compare the proportion of subjects that display change on cytogenetic abnormalities.
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
Change in platelet count
Proportion of subjects with a continuous platelet count <1000×109/L during treatment will be evaluated.
Full Information
NCT ID
NCT04226950
First Posted
December 10, 2019
Last Updated
August 5, 2022
Sponsor
Institute of Hematology & Blood Diseases Hospital, China
1. Study Identification
Unique Protocol Identification Number
NCT04226950
Brief Title
Pegylated Interferon Alfa-2b Versus Interferon Alfa Therapy in Childhood and Adolescent Essential Thrombocythemia
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
Study Type
Interventional
2. Study Status
Record Verification Date
August 2022
Overall Recruitment Status
Recruiting
Study Start Date
January 10, 2020 (Actual)
Primary Completion Date
July 20, 2023 (Anticipated)
Study Completion Date
September 20, 2023 (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 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
Detailed Description
This is a prospective, open-label, nonrandomized, single-center clinical trial between Interferon Alfa and Pegylated Interferon Alfa-2b in childhood and adolescent essential thrombocythemia (<20 years).
Patients will be divided into the following two treatment groups: 1. Recombinant Interferon Alpha, with an initial dose of 300 wu twice a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available, and the specific dose will be determined by the researchers; 2. Pegylated Interferon Alfa-2b, with an initial dose of 135 ug once a week (body surface area < 1.73 m2) or 180 ug once a week ( body surface area≥1.73 m2).
The current drug therapies and possible risks of Pegylated Interferon Alfa-2b and Interferon Alfa in the treatment of childhood and adolescent essential thrombocythemia will be fully introduced to the guardians (childhood patients) or patients (adolescent patients) by the researchers. Then the patients will be divided into one of the two groups according to the guardians' (childhood patients) or patients' (adolescent patients) will.
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 Thrombocythemia, Childhood, Interferon Alfa, Pegylated Interferon Alfa-2b, Adolescent
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
40 (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 twice a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available, and the specific dose will be determined by the researchers.
Arm Title
Pegylated Interferon Alfa-2b
Arm Type
Experimental
Arm Description
Pegylated Interferon Alfa-2b, with an initial dose of 135 ug once a week (body surface area < 1.73 m2) or 180 ug once a week ( body surface area≥1.73 m2).
Intervention Type
Drug
Intervention Name(s)
Recombinant Interferon Alpha
Intervention Description
Recombinant Interferon Alpha, with an initial dose of 300 wu twice a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available, and the specific dose will be determined by the researchers;
Intervention Type
Drug
Intervention Name(s)
Pegylated interferon alfa-2b
Intervention Description
Pegylated Interferon Alfa-2b, with an initial dose of 135 ug once a week (body surface area < 1.73 m2) or 180 ug once a week ( body surface area≥1.73 m2).
Primary Outcome Measure Information:
Title
Change in platelet count
Description
Proportion of subjects with a continuous platelet count ≤600×109/L or decrease ≥50% (<1000×109/L ) from baseline during treatment will be evaluated.
Time Frame
From the start of study treatment (Day 1) up to the end of month 12
Secondary Outcome Measure Information:
Title
The complete hematologic response rates
Description
To compare the complete hematologic response rates between different treatment groups
Time Frame
From the start of study treatment (Day 1) up to the end of month 12
Title
Time to response in platelet count
Description
Time to response in platelet count (<600×109/L) between different treatment groups
Time Frame
From the start of study treatment (Day 1) up to the end of month 12
Title
Impact of therapy on key biomarkers
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 (Day 1) up to the end of month 12
Title
Incidence of major cardiovascular and thrombotic events
Description
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
Time Frame
From the start of study treatment (Day 1) up to the end of month 12
Title
Incidence of development of myelodysplastic disorders, myelofibrosis, or leukemic transformation.
Description
To estimate incidence of development of myelodysplastic disorders, myelofibrosis, or leukemic transformation between different treatment groups
Time Frame
From the start of study treatment (Day 1) up to the end of month 12
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
12 months
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 (Day 1) up to the end of month 12
Title
Impact of therapy on bone marrow histopathology
Description
To compare the proportion of subjects that display change on bone marrow histopathology
Time Frame
From the start of study treatment (Day 1) up to the end of month 12
Title
Impact of therapy on cytogenetic abnormalities
Description
To compare the proportion of subjects that display change on cytogenetic abnormalities.
Time Frame
From the start of study treatment (Day 1) up to the end of month 12
Title
Death while on active treatment or observation following end of treatment
Description
To compare the incidence of death while on active treatment or observation following end of treatment
Time Frame
From the start of study treatment (Day 1) up to the end of month 12
Title
Change in platelet count
Description
Proportion of subjects with a continuous platelet count <1000×109/L during treatment will be evaluated.
Time Frame
From the start of study treatment (Day 1) up to the end of month 12
10. Eligibility
Sex
All
Maximum Age & Unit of Time
19 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Rongfeng Fu, MD
Phone
+862223909009
Email
furongfeng@ihcams.ac.cn
First Name & Middle Initial & Last Name or Official Title & Degree
Lei Zhang, MD
Phone
+862223909240
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
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Lei Zhang, MD
Phone
+862223909240
Email
zhanglei1@ihcams.ac.cn
12. IPD Sharing Statement
Learn more about this trial
Pegylated Interferon Alfa-2b Versus Interferon Alfa Therapy in Childhood and Adolescent Essential Thrombocythemia
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