The Combination of Zanubrutinib and High-dose Dexamethasone as First-line Treatment in Adult Immune Thrombocytopenia
- Conditions
- Immune Thrombocytopenia
- Interventions
- Registration Number
- NCT05369364
- Lead Sponsor
- Peking University People's Hospital
- Brief Summary
Randomized, open-label, multicenter study to compare the efficacy and safety of zanubrutinib plus high-dose dexamethasone compared to high-dose dexamethasone monotherapy for the first-line treatment of adults with primary immune thrombocytopenia (ITP).
- Detailed Description
The investigators are undertaking a parallel group, multicenter, randomized controlled trial of patients with ITP in China. Patients were randomized to Zanubrutinib + high-dose dexamethasone and high-dose dexamethasone 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
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 150
- Confirmed newly-diagnosed, treatment-naive ITP;
- Platelet counts <30×10^9/L ;
- Platelet counts < 50×10^9/L and significant bleeding symptoms (WHO bleeding scale 2 or above);
- Willing and able to sign written informed consent.
- Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit;
- Received first-line and second-line ITP-specific treatments (eg, steriods, cyclophosphamide, 6-mercaptopurine, vincristine, vinblastine, etc) ;
- Current HIV infection or hepatitis B virus or hepatitis C virus infections;
- Active infection;
- Maligancy;
- Severe medical condition (lung, hepatic or renal disorder) other than chronic ITP. Unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure, uncontrolled hypertension or cardiac arrhythmia);
- Female patients who are nursing or pregnant, who may be pregnant, or who contemplate pregnancy during the study period; a history of clinically significant adverse reactions to previous corticosteroid therapy;
- Have a known diagnosis of other autoimmune diseases, established in the medical history and laboratory findings with positive results for the determination of antinuclear antibodies, anti-cardiolipin antibodies, lupus anticoagulant or direct Coombs test.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Zanubrutinib and HD-DXM Zanubrutinib Dexamethasone 40 mg per day, 4 consecutive days (the 4-day course of dexamethasone was repeated in the case of lack of response by day 10) and Zanubrutinib 80mg qd po, 6 consecutive weeks Zanubrutinib and HD-DXM Dexamethasone Dexamethasone 40 mg per day, 4 consecutive days (the 4-day course of dexamethasone was repeated in the case of lack of response by day 10) and Zanubrutinib 80mg qd po, 6 consecutive weeks HD-DXM Dexamethasone Dexamethasone 40 mg per day, 4 consecutive days (the 4-day course of dexamethasone was repeated in the case of lack of response by day 10)
- Primary Outcome Measures
Name Time Method Sustained response 12 months The maintenance of platelet count ≥ 30 x 10\^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 12-month follow-up
- Secondary Outcome Measures
Name Time Method Loss of response 12 month Platelet counts below 100 x 109/L or bleeding (from CR) or platelet counts below 30 x 109/L, less than 2-fold increase of baseline platelet count or bleeding (from R)
Number of patients with bleeding 12 months Number of patients with bleeding complication ( WHO bleeding score).
Time to response (TTR) 12 months The time from starting treatment to time of achievement of CR or R
Durable response 6 months Platelet count ≥30 X 10\^9/L and at least doubling of the baseline count at 6 mo
Initial complete response 1 month Initial complete response (CR) was defined as platelet count more than 100,000 per cubic millimeter and absence of bleeding.
Initial response 1 month Initial response (R) as platelet count more than 30,000 per cubic millimeter and at least 2-fold increase of the baseline count and absence of bleeding.
Number of patients with adverse events 12 months Number of patients with adverse events
Duration of response (DOR) 12 months Duration of response at 12-month follow up
Early response 1 week Platelet count ≥30 X 10\^9/L and at least doubling baseline at 1 wk
Remission 12 months Platelet count \>100 X 10\^9/L at 12 month