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This study is an Investigator initiated, randomized, multicenter, double-blind, placebo-control study. The aim of this study is to evaluate the safety and efficiency of Sirolimus for primaty antiphospholipid syndrome patients at week 24 and week 48.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Sirolimus | Experimental | Sirolimus 1.5mg po. QD |
|
| placeobo | Placebo Comparator | Sirolimus placebo 1.5mg po. QD |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Sirolimus | Drug | Subjects will receive Sirolimus 1.5mg/d for 48 weeks in addition to their ongoing APS treatment regimen |
|
| Measure | Description | Time Frame |
|---|---|---|
| Complete response(CR) rate at week 24 | CR will be defined as follows: 1.No thrombosis events; 2.PLT ≥100 and with nobleeding; 3. No persistent autoimmnune haemolytic anaemia; 4. No skin ulcer; 5.No renal thrombotic microangiopathy; 5.Normal cognitive function (MoCA score ≥26). | week 24 |
| Partial response (PR) rate at week 24 | PR will be defined as one of follows: 1.superficial thrombotic events;2. PLT ≥30 or increased at least 2 times of baseline and with no bleeding;3.haemoglobulin concentration normal or increased 20g/L compared to baseline; 4.50% improvement; 5. a serum creatinine level 15% abov baseline, RBCs per high-power field 50% above baseline with no casts, 50% improvement in the urinary prt:cr; 6. milde cognitive diysfunction (MoCA 9~25). | week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Complete response(CR) rate at week 48 | CR will be defined as follows: 1.No thrombosis events; 2.PLT ≥100 and with nobleeding; 3. No persistent autoimmnune haemolytic anaemia; 4. No skin ulcer; 5.No renal thrombotic microangiopathy; 5.Normal cognitive function (MoCA score ≥26). | week 48 |
| Partial response (PR) rate at week 48 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Liling Xu, Ph.D | Contact | +86 010 88324173 | xuliling1079@163.com |
| Name | Affiliation | Role |
|---|---|---|
| Zhanguo Li | Peking university peoples hospital | Study Director |
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| ID | Term |
|---|---|
| D020123 | Sirolimus |
| ID | Term |
|---|---|
| D018942 | Macrolides |
| D007783 | Lactones |
| D009930 | Organic Chemicals |
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| Placebo | Drug | Subjects will receive Placebo 1.5mg/d for first 24 weeks and Sirolimus 1.5mg/d for next 24 weeks in addition to their ongoing APS treatment regimen |
|
PR will be defined as one of follows: 1.superficial thrombotic events;2. PLT ≥30 or increased at least 2 times of baseline and with no bleeding;3.haemoglobulin concentration normal or increased 20g/L compared to baseline; 4.50% improvement; 5. a serum creatinine level 15% abov baseline, RBCs per high-power field 50% above baseline with no casts, 50% improvement in the urinary prt:cr; 6. milde cognitive diysfunction (MoCA 9~25). |
| week 48 |
| Rate of Participants with adverse effects and serious adverse effects during treatment | Adverse effects include fever, rash, abnormal liver function, rate of new-onset infections and any abnormal measures after recivede study drug. | baseline to week 48 |