Immune Thrombocytopenia
Conditions
Keywords
BTK inhibitor, High-dose Dexamethasone
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.
Interventions
DRUGZanubrutinib
Zanubrutinib 80mg qd po, 6 consecutive weeks
DRUGDexamethasone
Dexamethasone, iv, 40 mg/d, for 4 days (The 4-day course of dexamethasone was repeated in the case of lack of response by day 10)
Sponsors
Beijing Hospital
Navy General Hospital, Beijing
Beijing Aerospace General Hospital
Qilu Hospital of Shandong University
Beijing Tongren Hospital
Peking University People's Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
1. Confirmed newly-diagnosed, treatment-naive ITP; 2. Platelet counts \<30×10\^9/L ; 3. Platelet counts \< 50×10\^9/L and significant bleeding symptoms (WHO bleeding scale 2 or above); 4. Willing and able to sign written informed consent.
Exclusion criteria
1. Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit; 2. Received first-line and second-line ITP-specific treatments (eg, steriods, cyclophosphamide, 6-mercaptopurine, vincristine, vinblastine, etc) ; 3. Current HIV infection or hepatitis B virus or hepatitis C virus infections; 4. Active infection; 5. Maligancy; 6. 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); 7. 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; 8. 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 bleeding | 12 months | Number of patients with bleeding complication ( WHO bleeding score). |
| Number of patients with adverse events | 12 months | Number of patients with adverse events |
| Time to response (TTR) | 12 months | The time from starting treatment to time of achievement of CR or R |
| 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. |
| 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) |
| Durable response | 6 months | Platelet count ≥30 X 10\^9/L and at least doubling of the baseline count at 6 mo |
| 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 |
| Duration of response (DOR) | 12 months | Duration of response at 12-month follow up |
Contacts
Primary ContactXiaohui Zhang, MD
Backup ContactQiusha Huang, MD
Outcome results
None listed