The Combination of Atorvastatin, Acetylcysteine and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia
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|ClinicalTrials.gov Identifier: NCT03460808|
Recruitment Status : Not yet recruiting
First Posted : March 9, 2018
Last Update Posted : March 9, 2018
|Condition or disease||Intervention/treatment||Phase|
|Immune Thrombocytopenia||Drug: atorvastatin Drug: Acetylcysteine Drug: Danazol||Phase 1 Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||200 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||The Combination of Atorvastatin, Acetylcysteine and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia|
|Estimated Study Start Date :||March 10, 2018|
|Estimated Primary Completion Date :||January 1, 2020|
|Estimated Study Completion Date :||January 1, 2023|
Experimental: atorvastatin, acetylcysteine & danazol
atorvastatin 20mg qd po plus acetylcysteine 400mg tid po plus danazol 200mg bid po for 12 weeks
Atorvastatin was used in combination with acetylcysteine and danazol.
Acetylcysteine was used in combination with atorvastatin and danazol.
Danazol was used in combination with atorvastatin and acetylcysteine or as the monotherapy
- the sustained platelet response at the 6-month follow-up [ Time Frame: From the start of study treatment (Day 1) up to the end of Month 6 ]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.
- overall response [ Time Frame: From the start of study treatment (Day 1) up to the end of Month 6 ]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 Frame: From the start of study treatment (Day 1) up to the end of Year 2 ]Time to response was defined as the time from starting treatment to the time to achieve the response.
- duration of response [ Time Frame: From the start of study treatment (Day 1) up to the end of Year 2 ]Duration of response was measured from the achievement of response to the loss of response.
- incidence of treatment-emergent adverse events [ Time Frame: From the start of study treatment (Day 1) up to the end of Year 2 ]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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03460808
|Contact: Xiao-hui Zhang, Professor||+86 firstname.lastname@example.org|
|Peking University Insititute of Hematology, Peking University People's Hospital|
|Beijing, Beijing, China, 100044|
|Contact: Xiao-hui Zhang, Professor email@example.com|