Clinical trial NCT05471050: 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
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Immune Thrombocytopenia
Keywords: Immune Thrombocytopenia
tacrolimus
danazol
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
First posted: 2022-07-22
Enrollment: 120
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention 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.
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Sustained response
timeFrame: 6 months
Secondary outcomes:
measure: Complete response
timeFrame: 6 months
measure: Response
timeFrame: 6 months
measure: Time to response
timeFrame: 6 months
measure: Duration of response
timeFrame: 6 months
measure: Adverse events
timeFrame: 6 months
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking University Insititute of Hematology, Peking University People's Hospital
state: Beijing
Study officials:
affiliation: Peking University People's Hospital, Peking University Insititute of Hematology
name: Xiao-Hui Zhang, MD
role: PRINCIPAL_INVESTIGATOR
References: