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The Combination of Tacrolimus and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia

Phase 2
Conditions
Immune Thrombocytopenia
Interventions
Drug: TAC
Registration Number
NCT05471050
Lead Sponsor
Peking University People's Hospital
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.

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
120
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.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
tacrolimus and DanazolDanazolTacrolimus 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.
tacrolimus and DanazolTACTacrolimus 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.
DanazolDanazolDanazol is given at 200mg bid for 12 weeks.
Primary Outcome Measures
NameTimeMethod
Sustained response6 months

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
NameTimeMethod
Response6 months

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.

Adverse events6 months

Adverse events were scaled according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.

Complete response6 months

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 to response6 months

Time to response was defined as the time from starting treatment to the time to achieve the response.

Duration of response6 months

Duration of response was measured from the achievement of response to the loss of response.

Trial Locations

Locations (1)

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

🇨🇳

Beijing, Beijing, China

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