Clinical trial NCT05494307: The Combination of Terbutaline and Danazol as the Treatment of Corticosteroid-resistant/Relapse Immune Thrombocytopenia
Official title: A Multicenter Randomized Trial of Second Line Treatment for Corticosteroid-Resistant or Relapsed Immune Thrombocytopenia: Combined Terbutaline and Danazol Versus Danazol Monotherapy
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Immune Thrombocytopenia
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
First posted: 2022-08-09
Enrollment: 228
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: A randomized, open-label, multicenter study to compare the efficacy and safety of terbutaline plus danazol compared to danazol monotherapy for the second-line treatment of adults with corticosteroid-resistant or relapsed primary immune thrombocytopenia (ITP).

Detailed description: The investigators are undertaking a parallel group, multicenter, randomized controlled trial of 228 adults with ITP in China. Patients were randomized to terbutaline plus danazol compared to 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: Patients \>18 years old with corticosteroid-resistant or relapsed ITP who had either a platelet count of \<30×10\^9/L or a platelet count of \<50×10\^9/L and clinically significant bleeding.

1. Not achieving a sustained response to therapy with full-dose corticosteroids for a duration of at least 4 weeks or relapsed during the process of corticosteroid tapering or discontinuation;
2. Platelet counts \<30×10\^9/L or platelet counts \< 50×10\^9/L and significant bleeding symptoms (WHO bleeding scale 2 or above);
3. Willing and able to sign written informed consent.

Exclusion Criteria:

1. Pregnant or lactating women;
2. Secondary ITP (have a known diagnosis of connective tissue diseases, malignancy, active infection, HIV infections or hepatitis B virus or hepatitis C virus infections);
3. Received drugs affecting the platelet counts within 6 months before the screening visit (e.g., chemotherapy, anticoagulants, etc);
4. Severe medical condition (lung, heart, hepatic or renal disorder);
5. Patients who are deemed unsuitable for the study by the investigator.
6. Patients who had hypertension, diabetes mellitus, hyperthyroidism or coronary heart disease.

Primary outcomes:
measure: Overall response
timeFrame: From date of randomization until 1 years or the end of follow-up
Secondary outcomes:
measure: Sustained response
timeFrame: From date of randomization until 1 years or the end of follow-up
measure: Complete response
timeFrame: From date of randomization until 1 years or the end of follow-up
measure: Remission
timeFrame: at 12-month follow-up
measure: Time to response
timeFrame: From date of randomization until 1 years or the end of follow-up
measure: Duration of response
timeFrame: From date of randomization until 1 years or the end of follow-up
measure: Rescue therapy
timeFrame: From date of randomization until 1 years or the end of follow-up
measure: Associated factors of treatment failure, OR, SR and remission
timeFrame: From date of randomization until 1 years or the end of follow-up
measure: Number of patients with bleeding
timeFrame: From date of randomization until 1 years or the end of follow-up
measure: Number of patients with side effects
timeFrame: From date of randomization until 1 years or the end of follow-up
measure: Relapse
timeFrame: From date of randomization until 1 years or the end of follow-up
measure: Relapse-free survival
timeFrame: From date of randomization until 1 years or the end of follow-up
Other outcomes:
Locations:
Study officials:
affiliation: Peking University People's Hospital
name: Xiao-Hui Zhang, MD
role: PRINCIPAL_INVESTIGATOR
References: