Back to resultsThe Combination of Tacrolimus and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia
Primary Purpose
Immune Thrombocytopenia
Status
Recruiting
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
TAC
Danazol
Sponsored by
About this trial
This is an interventional treatment trial for Immune Thrombocytopenia focused on measuring Immune Thrombocytopenia, tacrolimus, danazol
Eligibility Criteria
Inclusion Criteria:
- Primary immune thrombocytopenia (ITP);
- 18 years older;
- Platelet count of less than 30×10^9/L at enrollment;
- Patients who did not achieve a sustained response to treatment with full-dose corticosteroids for a minimum duration of 4 weeks or who relapsed during steroid-tapering or after its discontinuation;
- Patients who were receiving other maintenance regimens, primarily corticosteroids, ciclosporin, or mycophenolate mofetil, were also eligible if the dose of treatment had been stable in the past month and the dose was expected to be stable after enrolment and remained unchanged at least for the first 4 weeks of study until initial response was assessed, unless severe adverse events were suspected.
Exclusion Criteria:
- Secondary ITP (e.g., patients with HIV, HBV, HCV, Helicobacter pylori infection or SLE);
- Congestive heart failure, severe arrhythmia;
- Nursing or pregnant women;
- ALT or AST levels ≥ 3× the upper limit of the normal threshold;
- Creatinine or serum bilirubin levels ≥ 1.5× the upper limit;
- Active or previous malignancy ;
- Patients who had received danazol treatment or did not respond to danazol;
- Patients unable to have routine blood tests because of reasons such as insufficient time.
Sites / Locations
- Peking University Insititute of Hematology, Peking University People's HospitalRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Active Comparator
Arm Label
tacrolimus and Danazol
Danazol
Arm Description
Tacrolimus is given at a dose of 0.03mg/kg·d, and the dose is adjusted to maintain the trough concentration of tacrolimus at approximately 3-5 ng/mL for 12 weeks.
Danazol is given at 200mg bid for 12 weeks.
Outcomes
Primary Outcome Measures
Sustained response
Sustained response was defines as the maintenance of platelet count ≥ 30 x 10^9/L, and at least 2-fold increase of the baseline count, and the absence of bleeding, and no need for rescue medication at the 6-month follow-up.
Secondary Outcome Measures
Complete response
Complete response was defines as the maintenance of platelet count ≥ 100 x 10^9/L, and at least 2-fold increase of the baseline count, and the absence of bleeding, and no need for rescue medication.
Response
Response was defines as the maintenance of platelet count ≥ 30 x 10^9/L, and at least 2-fold increase of the baseline count, and the absence of bleeding, and no need for rescue medication.
Time to response
Time to response was defined as the time from starting treatment to the time to achieve the response.
Duration of response
Duration of response was measured from the achievement of response to the loss of response.
Adverse events
Adverse events were scaled according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Full Information
NCT ID
NCT05471050
First Posted
July 17, 2022
Last Updated
July 20, 2022
Sponsor
Peking University People's Hospital
1. Study Identification
Unique Protocol Identification Number
NCT05471050
Brief Title
The Combination of Tacrolimus and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia
Official Title
The Combination of Tacrolimus and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia: A Randomized, Controlled, Open-label Trial
Study Type
Interventional
2. Study Status
Record Verification Date
July 2022
Overall Recruitment Status
Recruiting
Study Start Date
March 2, 2022 (Actual)
Primary Completion Date
March 1, 2023 (Anticipated)
Study Completion Date
June 1, 2023 (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
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
Randomized, open-label, multicenter study to compare the efficacy and safety of combination of tacrolimus and danazol versus danazol for the treatment of adults with steroid-resistant/relapse immune thrombocytopenia (ITP).
Detailed Description
The investigators are undertaking a randomized controlled trial of 120 adults with steroid-resistant/relapse ITP in China. Patients were randomized to tacrolimus plus danazol and danazol 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
Keywords
Immune Thrombocytopenia, tacrolimus, danazol
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Model Description
The investigators are undertaking a randomized controlled trial of 120 adults with steroid-resistant/ relapse ITP in China. Patients were randomized to tacrolimus plus danazol and danazol monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.
Masking
None (Open Label)
Allocation
Randomized
Enrollment
120 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
tacrolimus and Danazol
Arm Type
Experimental
Arm Description
Tacrolimus is given at a dose of 0.03mg/kg·d, and the dose is adjusted to maintain the trough concentration of tacrolimus at approximately 3-5 ng/mL for 12 weeks.
Arm Title
Danazol
Arm Type
Active Comparator
Arm Description
Danazol is given at 200mg bid for 12 weeks.
Intervention Type
Drug
Intervention Name(s)
TAC
Intervention Description
Tacrolimus is given at a dose of 0.03mg/kg·d, and the dose is adjusted to maintain the trough concentration of tacrolimus at approximately 3-5 ng/mL for 12 weeks.
Intervention Type
Drug
Intervention Name(s)
Danazol
Intervention Description
Danazol is given at 200mg bid for 12 weeks.
Primary Outcome Measure Information:
Title
Sustained response
Description
Sustained response was defines as the maintenance of platelet count ≥ 30 x 10^9/L, and at least 2-fold increase of the baseline count, and the absence of bleeding, and no need for rescue medication at the 6-month follow-up.
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Complete response
Description
Complete response was defines as the maintenance of platelet count ≥ 100 x 10^9/L, and at least 2-fold increase of the baseline count, and the absence of bleeding, and no need for rescue medication.
Time Frame
6 months
Title
Response
Description
Response was defines as the maintenance of platelet count ≥ 30 x 10^9/L, and at least 2-fold increase of the baseline count, and the absence of bleeding, and no need for rescue medication.
Time Frame
6 months
Title
Time to response
Description
Time to response was defined as the time from starting treatment to the time to achieve the response.
Time Frame
6 months
Title
Duration of response
Description
Duration of response was measured from the achievement of response to the loss of response.
Time Frame
6 months
Title
Adverse events
Description
Adverse events were scaled according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Time Frame
6 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Primary immune thrombocytopenia (ITP);
18 years older;
Platelet count of less than 30×10^9/L at enrollment;
Patients who did not achieve a sustained response to treatment with full-dose corticosteroids for a minimum duration of 4 weeks or who relapsed during steroid-tapering or after its discontinuation;
Patients who were receiving other maintenance regimens, primarily corticosteroids, ciclosporin, or mycophenolate mofetil, were also eligible if the dose of treatment had been stable in the past month and the dose was expected to be stable after enrolment and remained unchanged at least for the first 4 weeks of study until initial response was assessed, unless severe adverse events were suspected.
Exclusion Criteria:
Secondary ITP (e.g., patients with HIV, HBV, HCV, Helicobacter pylori infection or SLE);
Congestive heart failure, severe arrhythmia;
Nursing or pregnant women;
ALT or AST levels ≥ 3× the upper limit of the normal threshold;
Creatinine or serum bilirubin levels ≥ 1.5× the upper limit;
Active or previous malignancy ;
Patients who had received danazol treatment or did not respond to danazol;
Patients unable to have routine blood tests because of reasons such as insufficient time.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Xiao-Hui Zhang, MD
Phone
+8613522338836
Email
zhangxh@bjmu.edu.cn
First Name & Middle Initial & Last Name or Official Title & Degree
Xuan Cai, MD
Phone
+8618811729606
Email
2206385254@qq.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Xiao-Hui Zhang, MD
Organizational Affiliation
Peking University People's Hospital, Peking University Insititute of Hematology
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
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Xiao-hui Zhang, MD
Phone
+8613522338836
Email
zhangxh@bjmu.edu.cn
12. IPD Sharing Statement
Learn more about this trial
The Combination of Tacrolimus and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia
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