Back to resultsCombined Steroid and Cyclosporin as First-line Treatment in Adults With Primary Immune Thrombocytopenia
Primary Purpose
Immune Thrombocytopenia
Status
Not yet recruiting
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
Cyclosporine Oral Product
Prednisone
Sponsored by
About this trial
This is an interventional treatment trial for Immune Thrombocytopenia
Eligibility Criteria
Inclusion Criteria:
- Newly-diagnosed, treatment-naive primary ITP;
- Platelet counts <30×10^9/L;
- Platelet counts < 50×10^9/L and significant bleeding symptoms (WHO bleeding scale 2 or above);
- Willing and able to sign written informed consent.
Exclusion Criteria:
- Pregnant or lactating women;
- Secondary ITP (have a known diagnosis of connective tissue diseases, malignancy, active infection, HIV infections or hepatitis B virus or hepatitis C virus infections);
- Received first-line and second-line ITP specific treatments (e.g., steroids, intravenous immunoglobulin, TPO-RAs, rhTPO, rituximab, etc);
- Received drugs affecting the platelet counts within 6 months before the screening visit (e.g., chemotherapy, anticoagulants, etc);
- Severe medical condition (lung, heart, hepatic or renal disorder);
- Patients who are deemed unsuitable for the study by the investigator.
Sites / Locations
- Peking University Insititute of Hematology, Peking University People's Hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Active Comparator
Arm Label
Cyclosporin plus Steroid
Standard steroid
Arm Description
Cyclosporin: started orally 1 mg/kg/d in two divided doses for 1 week, increased to 1.5 mg/kg/d for 1week, further increased to 2.5 mg/kg/d and then continued for 24 weeks. After 26 weeks of cyclosporin treatment, the dose for patients who achieved complete response was reduced by 25 mg/d every 2 weeks to ensure continuing the lowest dose that achieved the targeted serum level of cyclosporin and a safe platelet count. Standard regimen of steroid for a total of 10 weeks: 1 mg per kilogram of body weight for 2 weeks followed by 40 mg daily for 2 weeks, 20 mg daily for 2 weeks, 10 mg daily for 2 weeks, 5 mg daily for 1 week and 5 mg every other day for the final week.
Standard regimen for a total of 10 weeks: 1 mg per kilogram of body weight for 2 weeks followed by 40 mg daily for 2 weeks, 20 mg daily for 2 weeks, 10 mg daily for 2 weeks, 5 mg daily for 1 week and 5 mg every other day for the final week.
Outcomes
Primary Outcome Measures
Treatment failure
Nonresponse or loss of response for those who had achieved overall response (assessed in a time-to-event analysis). Overall response was defined as platelet count ≥ 30,000 per cubic millimeter and at least 2-fold increase of the baseline count and absence of bleeding.
Secondary Outcome Measures
Number of patients with side effects
Number of patients with Medication adverse events.
Number of patients with bleeding
Number of patients with bleeding complication (WHO bleeding score)
Sustained response
The maintenance of platelet count ≥ 30 x 10^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 6-month follow-up.
Overall response (OR)
Platelet count ≥ 30,000 per cubic millimeter and at least 2-fold increase of the baseline count and absence of bleeding.
Complete response (CR)
Platelet count > 100,000 per cubic millimeter and absence of bleeding.
Time to response
The time from starting treatment to time of achievement of CR or OR
Duration of response
time from OR until loss of response or until the last follow-up visit
Remission
a durable platelet count ≥30×10^9/L without bleeding up to 12 months from randomization.
Rescue therapy
any new medical intervention taken to increase the platelet count or prevent bleeding events or an increase in the dose of concomitant treatments
Associated factors of treatment failure, OR, SR and remission
Factors that are associated with treatment failure, OR, SR and remission
Full Information
NCT ID
NCT05459649
First Posted
July 10, 2022
Last Updated
July 14, 2022
Sponsor
Peking University People's Hospital
1. Study Identification
Unique Protocol Identification Number
NCT05459649
Brief Title
Combined Steroid and Cyclosporin as First-line Treatment in Adults With Primary Immune Thrombocytopenia
Official Title
A Multicenter Randomized Trial of First Line Treatment for Newly Diagnosed Immune Thrombocytopenia: Standard Steroid Treatment Versus Combined Steroid and Cyclosporin
Study Type
Interventional
2. Study Status
Record Verification Date
July 2022
Overall Recruitment Status
Not yet recruiting
Study Start Date
July 20, 2022 (Anticipated)
Primary Completion Date
July 20, 2024 (Anticipated)
Study Completion Date
July 20, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Peking University People's Hospital
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
Randomized, open-label, multicenter study to compare the efficacy and safety of cyclosporin plus standard steroid compared to standard steroid monotherapy for the first-line treatment of adults with primary immune thrombocytopenia (ITP).
Detailed Description
The investigators are undertaking a parallel group, multicenter, randomized controlled trial of 253 adults with ITP in China. Patients were randomized to cyclosporin plus standard steroid compared to standard steroid monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Immune Thrombocytopenia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
Outcomes Assessor
Allocation
Randomized
Enrollment
253 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Cyclosporin plus Steroid
Arm Type
Experimental
Arm Description
Cyclosporin: started orally 1 mg/kg/d in two divided doses for 1 week, increased to 1.5 mg/kg/d for 1week, further increased to 2.5 mg/kg/d and then continued for 24 weeks. After 26 weeks of cyclosporin treatment, the dose for patients who achieved complete response was reduced by 25 mg/d every 2 weeks to ensure continuing the lowest dose that achieved the targeted serum level of cyclosporin and a safe platelet count.
Standard regimen of steroid for a total of 10 weeks: 1 mg per kilogram of body weight for 2 weeks followed by 40 mg daily for 2 weeks, 20 mg daily for 2 weeks, 10 mg daily for 2 weeks, 5 mg daily for 1 week and 5 mg every other day for the final week.
Arm Title
Standard steroid
Arm Type
Active Comparator
Arm Description
Standard regimen for a total of 10 weeks: 1 mg per kilogram of body weight for 2 weeks followed by 40 mg daily for 2 weeks, 20 mg daily for 2 weeks, 10 mg daily for 2 weeks, 5 mg daily for 1 week and 5 mg every other day for the final week.
Intervention Type
Drug
Intervention Name(s)
Cyclosporine Oral Product
Other Intervention Name(s)
Prednisone
Intervention Description
A combination of cyclosporin with standard steroid in newly diagnosed ITP patients: cyclosporin was started orally 1 mg/kg/d in two divided doses for 1 week, increased to 1.5 mg/kg/d for 1week, further increased to 2.5 mg/kg/d and then continued for 24 weeks. The therapeutic serum level of cyclosporin was 75 to 150 ug/L. After 26 weeks of cyclosporin treatment, the dose for patients who achieved complete response was reduced by 25 mg/d every 2 weeks to ensure continuing the lowest dose that achieved the targeted serum level of cyclosporin and a safe platelet count; standard steroid regimen included orally prednisone for a total of 10 weeks: 1 mg per kilogram of body weight for 2 weeks followed by 40 mg daily for 2 weeks, 20 mg daily for 2 weeks, 10 mg daily for 2 weeks, 5 mg daily for 1 week and 5 mg every other day for the final week.
Intervention Type
Drug
Intervention Name(s)
Prednisone
Intervention Description
Standard steroid in newly diagnosed ITP patients: orally prednisone for a total of 10 weeks: 1 mg per kilogram of body weight for 2 weeks followed by 40 mg daily for 2 weeks, 20 mg daily for 2 weeks, 10 mg daily for 2 weeks, 5 mg daily for 1 week and 5 mg every other day for the final week.
Primary Outcome Measure Information:
Title
Treatment failure
Description
Nonresponse or loss of response for those who had achieved overall response (assessed in a time-to-event analysis). Overall response was defined as platelet count ≥ 30,000 per cubic millimeter and at least 2-fold increase of the baseline count and absence of bleeding.
Time Frame
From date of randomization until 2 years or the end of follow-up
Secondary Outcome Measure Information:
Title
Number of patients with side effects
Description
Number of patients with Medication adverse events.
Time Frame
From date of randomization until 2 years or the end of follow-up
Title
Number of patients with bleeding
Description
Number of patients with bleeding complication (WHO bleeding score)
Time Frame
From date of randomization until 2 years or the end of follow-up
Title
Sustained response
Description
The maintenance of platelet count ≥ 30 x 10^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 6-month follow-up.
Time Frame
From date of randomization until 2 years or the end of follow-up
Title
Overall response (OR)
Description
Platelet count ≥ 30,000 per cubic millimeter and at least 2-fold increase of the baseline count and absence of bleeding.
Time Frame
From date of randomization until 2 years or the end of follow-up
Title
Complete response (CR)
Description
Platelet count > 100,000 per cubic millimeter and absence of bleeding.
Time Frame
From date of randomization until 2 years or the end of follow-up
Title
Time to response
Description
The time from starting treatment to time of achievement of CR or OR
Time Frame
From date of randomization until 2 years or the end of follow-up
Title
Duration of response
Description
time from OR until loss of response or until the last follow-up visit
Time Frame
From date of randomization until 2 years or the end of follow-up
Title
Remission
Description
a durable platelet count ≥30×10^9/L without bleeding up to 12 months from randomization.
Time Frame
at 12-month follow-up
Title
Rescue therapy
Description
any new medical intervention taken to increase the platelet count or prevent bleeding events or an increase in the dose of concomitant treatments
Time Frame
From date of randomization until 2 years or the end of follow-up
Title
Associated factors of treatment failure, OR, SR and remission
Description
Factors that are associated with treatment failure, OR, SR and remission
Time Frame
From date of randomization until 2 years or the end of follow-up
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Newly-diagnosed, treatment-naive primary ITP;
Platelet counts <30×10^9/L;
Platelet counts < 50×10^9/L and significant bleeding symptoms (WHO bleeding scale 2 or above);
Willing and able to sign written informed consent.
Exclusion Criteria:
Pregnant or lactating women;
Secondary ITP (have a known diagnosis of connective tissue diseases, malignancy, active infection, HIV infections or hepatitis B virus or hepatitis C virus infections);
Received first-line and second-line ITP specific treatments (e.g., steroids, intravenous immunoglobulin, TPO-RAs, rhTPO, rituximab, etc);
Received drugs affecting the platelet counts within 6 months before the screening visit (e.g., chemotherapy, anticoagulants, etc);
Severe medical condition (lung, heart, hepatic or renal disorder);
Patients who are deemed unsuitable for the study by the investigator.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Xiao-Hui Zhang, MD
Phone
+8613522338836
Email
zhangxh100@sina.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Xiao-Hui Zhang, MD
Organizational Affiliation
Peking University People's Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Peking University Insititute of Hematology, Peking University People's Hospital
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100010
Country
China
12. IPD Sharing Statement
Learn more about this trial
Combined Steroid and Cyclosporin as First-line Treatment in Adults With Primary Immune Thrombocytopenia
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