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This prospective, open-label, single-center, one-arm clinical trial aims at evaluating the efficacy and safety of avatrombopag in Chinese adult Immune Thrombocytopenia (ITP) patients with autoantibodies fail (due to intolerance or resistance) to eltrombopag or herombopag treatment.
This prospective, open-label, single-center, one-arm clinical trial aims at evaluating the efficacy and safety of avatrombopag in Chinese adult ITP patients with autoantibodies fail (due to intolerance or resistance) to eltrombopag or herombopag treatment.
The subjects include ITP secondary to connective tissue diseases (including but not limited to systemic lupus erythematosus, Sjogren's syndrome and rheumatoid arthritis), primary ITP with positive antinuclear antibody but not up to the diagnostic criteria of connective tissue diseases, primary Evans syndrome, Evans syndrome secondary to connective tissue diseases, and primary ITP with positive Coomb's test but not up to the diagnostic criteria of Evans syndrome.
Fifty-two eligible subjects will be enrolled in this study. The dose will be adjusted according to the platelet count during the period from week 1 to week 12.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment group | Experimental | Fifty-two subjects will be enrolled with the indicated treatment dose of avatrombopag |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Avatrombopag | Drug | The subjects will receive avatrombopag treatment with an initial dose of 40mg once a day. Platelet counts will be obtained weekly during the first 4 weeks and then every 2 weeks until week 12 after treatment. The dose adjustment range is 20 mg per week to 40 mg per day to maintain the platelet level between 30×10^9/L and 200×10^9/L. If the platelet count does not reach to 30×10^9/L after taking avatrombopag 40mg once a day for 4 consecutive weeks, the treatment will be stopped. |
| Measure | Description | Time Frame |
|---|---|---|
| Platelet response | Percentage of participants achieving a platelet count >=30×10^9/L and at least doubling of the baseline count within 12 weeks of treatment. | From the start of study treatment (Day 1) up to the end of week 12. |
| Measure | Description | Time Frame |
|---|---|---|
| Platelet response | Proportion of subjects who achieve response (R) within 1, 2, 4 and 8 weeks of treatment. | From the start of study treatment (Day 1) up to the end of week 12. |
| Platelet response |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Toxicity | The proportion of subjects with specific pre-defined toxicity, including headache, fever, nausea and abdominal pain, and unpredictable toxicity. | From the start of study treatment (Day 1) up to the end of week 12. |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Rongfeng Fu, M.D. | Contact | +862223909009 | furongfeng@ihcams.ac.cn | |
| Lei Zhang, M.D. | Contact | +862223909240 | zhanglei1@ihcams.ac.cn |
| Name | Affiliation | Role |
|---|---|---|
| Lei Zhang, M.D. | Institute of Hematology & Blood Diseases Hospital, China | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Institute of Hematology & Blood Diseases Hospital | Recruiting | Tianjin | Tianjin Municipality | 300020 | China |
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| ID | Term |
|---|---|
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| C536380 | Evans Syndrome |
| D003240 | Connective Tissue Diseases |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| C533238 | avatrombopag |
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|
Proportion of subjects who achieve complete response (CR) within 4, 8 and 12 weeks of treatment.
| From the start of study treatment (Day 1) up to the end of week 12. |
| Duration of platelet response | Proportion of subjects with a platelet count >=30×10^9/L for at least 4 consecutive weeks during the 12 week treatment period without remedial treatment. | From the start of study treatment (Day 1) up to the end of week 12. |
| Platelet response | Percentage of participants achieving a platelet count >=50×10^9/L within 12 weeks of treatment. | From the start of study treatment (Day 1) up to the end of week 12. |
| Time to platelet response | Time to response is defined as time from the start of treatment to the first time of achieving a platelet count >= 30×10^9/L and at least doubling of the baseline count during the whole 12 weeks. | From the start of study treatment (Day 1) up to the end of week 12. |
| Duration of platelet response | Total duration of time a participant with a response of R. | From the start of study treatment (Day 1) up to the end of week 12. |
| Proportion of patients receiving remedial treatment. | Proportion of patients receiving remedial treatment. | From the start of study treatment (Day 1) up to the end of week 12. |
| Bleeding score | The incidence and grade of bleeding symptoms according to the World Health Organization Bleeding Scale. | From the start of study treatment (Day 1) up to the end of week 12. |
| Changes of disease activity index in patients with systemic lupus erythematosus | The proportion of subjects with improvement of disease activity index in patients with systemic lupus erythematosus according to the SLEDAI standard. | From the start of study treatment (Day 1) up to the end of week 12. |
| The improvement of symptoms | The proportion of subjects with improvement of symptoms including skin symptom, joint pain, dry mouth and dry eyes. | From the start of study treatment (Day 1) up to the end of week 12. |
| Improvement in immune indexes | The proportion of subjects with improvement immune indexes including antinuclear antibody, extractable nuclear antigens spectrum and Coomb's test. | From the start of study treatment (Day 1) up to the end of week 12. |
| Discontinuation rate of glucocorticoids | The proportion of subjects with discontinuation use of glucocorticoids. | From the start of study treatment (Day 1) up to the end of week 12. |
| Functional assessment of chronic illness therapy-fatigue | In all participants, functional assessment of chronic illness therapy-fatigue questionnaire will be used to assess the health related quality of life before and after treatment. | From the start of study treatment (Day 1) up to the end of week 12. |
| ITP-Patient Assessment Questionnaire | In all participants, ITP-Patient Assessment Questionnaire will be used to assess the health related quality of life before and after treatment. | From the start of study treatment (Day 1) up to the end of week 12. |
| 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 |
| D017437 | Skin and Connective Tissue Diseases |