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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Zanubrutinib 80mg/d + Dexamethasone | Experimental | Participants in this arm will receive a combination therapy consisting of: 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. |
|
| Zanubrutinib 160mg/d + Dexamethasone | Experimental | Participants in this arm will receive a combination therapy consisting of: Zanubrutinib: 160 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. |
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| Zanubrutinib 240mg/d + Dexamethasone | Experimental | Participants in this arm will receive a combination therapy consisting of: Zanubrutinib: 240 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. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Zanubrutinib 80mg/d | Drug | Zanubrutinib: 80 mg taken orally once daily for 26 weeks |
|
| Measure | Description | Time Frame |
|---|---|---|
| Sustained response | 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. | 26 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Initial Response (R) | 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. | day 14 |
| Initial complete response (CR) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Xiaohui Zhang, MD | Contact | 8688324577 | zhangxh100@sina.com | |
| Qiusha Huang, MD | Contact | 8688324577 | huangfuqs@163.com |
| Name | Affiliation | Role |
|---|---|---|
| Xiaohui Zhang | Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Collaborative Innovation Center of Hematology | Principal Investigator |
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| ID | Term |
|---|---|
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| C000629551 | zanubrutinib |
| D003907 | Dexamethasone |
| ID | Term |
|---|---|
| D011246 | Pregnadienetriols |
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D013256 | Steroids |
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| High dose dexamethasone | Drug | 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. |
|
| Zanubrutinib 160mg/d | Drug | Zanubrutinib: 160 mg taken orally once daily for 26 weeks |
|
| Zanubrutinib 240mg/d | Drug | Zanubrutinib: 240 mg taken orally once daily for 26 weeks |
|
Complete response (CR) was defined as platelet count more than 100,000 per cubic millimeter and absence of bleeding.
| day 14 |
| Time to response | The time from starting treatment to time of achievement of CR or R | 6 month |
| Duration of response (DOR) | Duration of response at 26-week follow up | 26 weeks |
| Bleeding events | Number of patients with bleeding | 26 weeks |
| Adverse events | Number of patients with adverse events | 26 weeks |
| Loss of response | 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) | 26 weeks |
| Health related quality of life | Health-related quality of life using the Immune Thrombocytopenic Purpura-Patient Assessment Questionnaire (ITP-PAQ) | baseline and 26 weeks |
| D006425 |
| Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |
| D000072473 |
| Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
| D013259 | Steroids, Fluorinated |