Back to resultsThe Combination of Atorvastatin, Acetylcysteine and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia
Primary Purpose
Immune Thrombocytopenia
Status
Unknown status
Phase
Phase 1
Locations
China
Study Type
Interventional
Intervention
atorvastatin
Acetylcysteine
Danazol
Sponsored by
About this trial
This is an interventional treatment trial for Immune Thrombocytopenia focused on measuring steroid-resistant, refractory, Atorvastatin, Acetylcysteine, Danazol
Eligibility Criteria
Inclusion Criteria:
- ITP confirmed by excluding other supervened causes of thrombocytopenia;
- 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;
- ECOG<2.
- EPCs in bone marrow less than 0.02%
Exclusion Criteria:
- Secondary immune thrombocytopenia (e.g., patients with HIV, HCV, Helicobacter pylori infection or patients with systemic lupus erythematosus)
- congestive heart failure
- severe arrhythmia
- nursing or pregnant women
- aspartate aminotransferase and alanine transaminase levels ≥ 3× the upper limit of the normal threshold criteria
- creatinine or serum bilirubin levels each 1.5 times or more than the normal range
- active or previous malignancy
- Unable to do blood routine test for the sake of time, distance, economic issues or other reasons.
Sites / Locations
- Peking University Insititute of Hematology, Peking University People's Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
atorvastatin, acetylcysteine & danazol
Arm Description
atorvastatin 20mg qd po plus acetylcysteine 400mg tid po plus danazol 200mg bid po for 12 weeks
Outcomes
Primary Outcome Measures
the sustained platelet response at the 6-month follow-up
The number of participants (responders) with platelet count >=30x10^9/L and at least a 2-fold increase in the baseline count (PR) or a platelet count >=100x10^9/L (CR) and the absence of bleeding, without rescue medication at 6-month follow-up.
Secondary Outcome Measures
overall response
The number of participants with platelet count >=30×10^9/L at least once and at least a doubling of the baseline platelet count without the administration of any other platelet increasing therapy.
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.
incidence of treatment-emergent adverse events
All patients were assessed for treatment-emergent adverse events every week during the first 8 weeks of treatment, and at 2-week intervals thereafter. Adverse events were scaled according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0
Full Information
NCT ID
NCT03460808
First Posted
March 5, 2018
Last Updated
March 5, 2018
Sponsor
Peking University People's Hospital
1. Study Identification
Unique Protocol Identification Number
NCT03460808
Brief Title
The Combination of Atorvastatin, Acetylcysteine and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia
Official Title
The Combination of Atorvastatin, Acetylcysteine and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia
Study Type
Interventional
2. Study Status
Record Verification Date
March 2018
Overall Recruitment Status
Unknown status
Study Start Date
March 10, 2018 (Anticipated)
Primary Completion Date
January 1, 2020 (Anticipated)
Study Completion Date
January 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
5. Study Description
Brief Summary
Single-arm, open-lable, multicentre study to compare the efficacy and safety of atorvastatin, acetylcysteine plus danazol with danazol monotherapy in patients with corticosteroidresistant/relapsed ITP.
Detailed Description
Immune thrombocytopenia (ITP) is a severe bleeding disorder. Approximately 2/3 of patients achieve remission from first-line therapies. However, the underlying mechanism of corticosteroid-resistant or relapsed ITP is not well understood; thus, treatment remains a great challenge. Atorvastatin was shown to enhance bone marrow endothelial cell function and N-acetylcysteine (NAC) was shown to inhibit PLT binding to endothelial cell.
A multicentre prospective study was performed in non-splenectomized ITP patients who were either resistant to a standard dose of corticosteroids or had relapsed. Patients were randomized to atorvastatin, acetylcysteine plus danazol with danazol monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study, in order to compare the efficacy and safety of atorvastatin, acetylcysteine plus danazol with danazol monotherapy in patients with corticosteroid-resistant/relapsed ITP.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Immune Thrombocytopenia
Keywords
steroid-resistant, refractory, Atorvastatin, Acetylcysteine, Danazol
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
200 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
atorvastatin, acetylcysteine & danazol
Arm Type
Experimental
Arm Description
atorvastatin 20mg qd po plus acetylcysteine 400mg tid po plus danazol 200mg bid po for 12 weeks
Intervention Type
Drug
Intervention Name(s)
atorvastatin
Intervention Description
Atorvastatin was used in combination with acetylcysteine and danazol.
Intervention Type
Drug
Intervention Name(s)
Acetylcysteine
Intervention Description
Acetylcysteine was used in combination with atorvastatin and danazol.
Intervention Type
Drug
Intervention Name(s)
Danazol
Other Intervention Name(s)
Danocrine, Cleregil, Danol
Intervention Description
Danazol was used in combination with atorvastatin and acetylcysteine or as the monotherapy
Primary Outcome Measure Information:
Title
the sustained platelet response at the 6-month follow-up
Description
The number of participants (responders) with platelet count >=30x10^9/L and at least a 2-fold increase in the baseline count (PR) or a platelet count >=100x10^9/L (CR) and the absence of bleeding, without rescue medication at 6-month follow-up.
Time Frame
From the start of study treatment (Day 1) up to the end of Month 6
Secondary Outcome Measure Information:
Title
overall response
Description
The number of participants with platelet count >=30×10^9/L at least once and at least a doubling of the baseline platelet count without the administration of any other platelet increasing therapy.
Time Frame
From the start of study treatment (Day 1) up to the end of Month 6
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
From the start of study treatment (Day 1) up to the end of Year 2
Title
duration of response
Description
Duration of response was measured from the achievement of response to the loss of response.
Time Frame
From the start of study treatment (Day 1) up to the end of Year 2
Title
incidence of treatment-emergent adverse events
Description
All patients were assessed for treatment-emergent adverse events every week during the first 8 weeks of treatment, and at 2-week intervals thereafter. Adverse events were scaled according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0
Time Frame
From the start of study treatment (Day 1) up to the end of Year 2
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
ITP confirmed by excluding other supervened causes of thrombocytopenia;
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;
ECOG<2.
EPCs in bone marrow less than 0.02%
Exclusion Criteria:
Secondary immune thrombocytopenia (e.g., patients with HIV, HCV, Helicobacter pylori infection or patients with systemic lupus erythematosus)
congestive heart failure
severe arrhythmia
nursing or pregnant women
aspartate aminotransferase and alanine transaminase levels ≥ 3× the upper limit of the normal threshold criteria
creatinine or serum bilirubin levels each 1.5 times or more than the normal range
active or previous malignancy
Unable to do blood routine test for the sake of time, distance, economic issues or other reasons.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Xiao-hui Zhang, Professor
Phone
+86 010-88324516
Email
zhangxh100@sina.com
Facility Information:
Facility Name
Peking University Insititute of Hematology, Peking University People's Hospital
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100044
Country
China
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Xiao-hui Zhang, Professor
Email
zhangxh100@sina.com
12. IPD Sharing Statement
Learn more about this trial
The Combination of Atorvastatin, Acetylcysteine and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia
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