The Combination of Atorvastatin, Acetylcysteine and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia

NCT ID: NCT03460808

Last Updated: 2018-03-09

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 200 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-03-10
• Study Completion Date: 2023-01-01

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.

Conditions

Immune Thrombocytopenia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

atorvastatin, acetylcysteine & danazol

atorvastatin 20mg qd po plus acetylcysteine 400mg tid po plus danazol 200mg bid po for 12 weeks

Group Type: EXPERIMENTAL

atorvastatin

Intervention Type: DRUG

Atorvastatin was used in combination with acetylcysteine and danazol.

Acetylcysteine

Intervention Type: DRUG

Acetylcysteine was used in combination with atorvastatin and danazol.

Danazol

Intervention Type: DRUG

Danazol was used in combination with atorvastatin and acetylcysteine or as the monotherapy

Interventions

atorvastatin

Atorvastatin was used in combination with acetylcysteine and danazol.

Intervention Type: DRUG

Acetylcysteine

Acetylcysteine was used in combination with atorvastatin and danazol.

Intervention Type: DRUG

Danazol

Danazol was used in combination with atorvastatin and acetylcysteine or as the monotherapy

Intervention Type: DRUG

Other Intervention Names

Danocrine; Cleregil; Danol

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Peking University People's Hospital

OTHER

Sponsor Role: lead

Responsible Party

Xiao-hui Zhang

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Peking University Insititute of Hematology, Peking University People's Hospital

Beijing, Beijing Municipality, China

Countries

China

Central Contacts

Xiao-hui Zhang, Professor

Role: CONTACT

zhangxh100@sina.com

+86 010-88324516

Facility Contacts

Xiao-hui Zhang, Professor

Role: primary

zhangxh100@sina.com

Other Identifiers

Z171100001017084

Identifier Type: -

Identifier Source: org_study_id