Immune Thrombocytopenia
Conditions
Keywords
ITP, zanubrutinib
Brief summary
The goal of this clinical trial is to find the best dose of zanubrutinib when used together with high-dose dexamethasone for adults who are newly diagnosed with primary immune thrombocytopenia (ITP), and to learn how safe and effective this combination treatment is.
Interventions
DRUGZanubrutinib 80mg/d
Zanubrutinib: 80 mg taken orally once daily for 26 weeks
Dexamethasone: 40 mg taken orally once daily for 4 consecutive days at the beginning of treatment. A second identical 4-day course may be repeated after 2 weeks if an adequate initial platelet response is not achieved.
DRUGZanubrutinib 160mg/d
Zanubrutinib: 160 mg taken orally once daily for 26 weeks
DRUGZanubrutinib 240mg/d
Zanubrutinib: 240 mg taken orally once daily for 26 weeks
Sponsors
Peking University People's Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Outcomes Assessor)
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 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; 9. Patients who are deemed unsuitable for the study by the investigator.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Sustained response | 26 weeks | 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 26-week follow-up. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Loss of response | 26 weeks | 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) |
| Initial Response (R) | day 14 | 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. |
| Initial complete response (CR) | day 14 | Complete response (CR) was defined as platelet count more than 100,000 per cubic millimeter and absence of bleeding. |
| Health related quality of life | baseline and 26 weeks | Health-related quality of life using the Immune Thrombocytopenic Purpura-Patient Assessment Questionnaire (ITP-PAQ) |
| Duration of response (DOR) | 26 weeks | Duration of response at 26-week follow up |
| Bleeding events | 26 weeks | Number of patients with bleeding |
| Adverse events | 26 weeks | Number of patients with adverse events |
| Time to response | 6 month | The time from starting treatment to time of achievement of CR or R |
Contacts
Primary ContactXiaohui Zhang, MD
Backup ContactQiusha Huang, MD
Outcome results
None listed