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This study is an open label, randomized controlled, multicenter clinical trial aimed at comparing the efficacy and safety of the combination of atorvastatin and all trans retinoic acid with atorvastatin alone in the treatment of first-line ineffective or recurrent immune thrombocytopenia.
The study is divided into a screening period (2 weeks), a treatment period (0-24 weeks), a reduction period (25-36 weeks), and a follow-up period (37-52 weeks), with a total of one year from the start of treatment to the end of follow-up (52 weeks).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Avatrombopag+All trans retinoic acid | Experimental |
| |
| Avatrombopag | Active Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Avatrombopag+All trans retinoic acid | Drug | Avatrombopag, po, starting dose 20mg qd, later adjusted dose or frequency according to response; All trans retinoic acid, po, 10mg bid. |
| Measure | Description | Time Frame |
|---|---|---|
| Treatment Free Rate at week 52 | up to 52 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| At weeks 12 and 24 without rescue treatment, the proportion of subjects with platelet levels reaching CR, R, and ≥ 50 × 10^9/L, respectively | up to 12 and 24 weeks | |
| Treatment Free Rate at week 12 after discontinuation of treatment | up to 12 weeks after discontinuation of treatment |
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Inclusion Criteria:
Exclusion Criteria:
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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 |
|---|---|
| C533238 | avatrombopag |
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| Avatrombopag | Drug | Avatrombopag, po, starting dose 20mg qd, later adjusted dose or frequency according to response |
|
| The proportion of subjects whose platelet levels reached CR and R at 12 weeks after discontinuation of treatment | up to 12 weeks after discontinuation of treatment |
| The time from the start of treatment to the first platelet count ≥ 50 × 10^9/L | From date of randomization until the date of first documented platelet count ≥ 50 × 10^9/L, assessed up to 52 weeks | up to 52 weeks |
| The time required from the start of treatment to the first platelet count increasing by at least 2 times compared to the baseline platelet count | From date of randomization until the date of first documented platelet count increasing by at least 2 times compared to the baseline platelet count, assessed up to 52 weeks | up to 52 weeks |
| The number of times subjects received rescue therapy during treatment and follow-up, and the proportion of subjects who received at least one rescue therapy | up to 52 weeks |
| World Health Organization(WHO) bleeding scoring criteria | score range: 0-4; higher scores mean a worse outcome | up to 52 weeks |
| The Functional Assessment ot Chronic Illness Therapy-Fatigure(FACITF) score | score range: 0-52; higher scores mean a worse outcome | up to 52 weeks |
| Adverse event | up to 52 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 |