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The goal of this clinical trial is to learn if sirolimus, added to standard background therapy (corticosteroids and hydroxychloroquine), works to treat systemic lupus erythematosus-associated immune thrombocytopenia (SLE-ITP) in adults aged 18 to 65 years. The study also aims to evaluate the long-term effectiveness and safety of sirolimus over 48 weeks.
The main questions it aims to answer are:
Participants will:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Sirolimus | Experimental |
| |
| Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Sirolimus | Drug | Sirolimus 1.5mg/day + prednisone + hydroxychloroquine |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Overall response rate (complete response + partial response) of ITP at Week 24 | Complete response: platelet count ≥ 100×10^9/L with no overt bleeding Partial response: platelet count ≥ 30×10^9/L and doubled from baseline, with no overt bleeding | Week 24 |
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Inclusion Criteria:
Age ≥ 18 and < 65 years
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Liying Peng | Contact | 86 010 69159958 | liying_peng@qq.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Peking Union Medical College Hospital | Recruiting | Beijing | Beijing Municipality | 100730 | China |
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| ID | Term |
|---|---|
| D008180 | Lupus Erythematosus, Systemic |
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| ID | Term |
|---|---|
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
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| ID | Term |
|---|---|
| D020123 | Sirolimus |
| ID | Term |
|---|---|
| D018942 | Macrolides |
| D007783 | Lactones |
| D009930 | Organic Chemicals |
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| Placebo |
| Drug |
Placebo + prednisone + hydroxychloroquine |
|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |