The Safety and Efficiency of Sirolimus in Primary Antiphospholipid Syndrome: A Randomized Control Study
- Conditions
- Primary Antiphospholipid Syndrome
- Interventions
- Drug: Placebo
- Registration Number
- NCT06504420
- Lead Sponsor
- Peking University People's Hospital
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 70
- Understand and sign the informed consent form
- Male or Female
- aged 18-70 at the time of screening visit
- Met 2006 Sapporo classification criteria of APS or 2023 ACR/EULAR classification criteria of APS
- With the stable combination therapy
- history of serious adverse events or contraindication to Sirolimus
- Catastrophic APS within 90 days
- Acute thrombosis within 30 days
- ≥4/11 American College of Rheumatology Classification Criteria for SLE or other systemic autoimmune diseases
- Historically positive HIV test or test positive at screening for HIV
- currently on any suppressive therapy for a chronic infection (such as tuberculosis, hepatitis B infection, hepatitis C infection, CMV, EBV, Syphilis, etc)
- Surgery treatment within one month
- History of malignant neoplasm within the last 5 years
- White blood cell counts<3×10*9/L
- Abnormal Liver function tests: ALT or AST ≥ 1.5 times the upper limit of the normal value, and total bilirubin and blood lipids ≥ 2 times the upper limit of the normal value
- Pregnant or pregnancy preparation or breastfeed
- Any circumstances that may cause the subjects to be unable to complete the study or pose significant risks to the subjects
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description placeobo Placebo Sirolimus placebo 1.5mg po. QD Sirolimus Sirolimus Sirolimus 1.5mg po. QD
- Primary Outcome Measures
Name Time Method Partial response (PR) rate at week 24 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).
Complete response(CR) rate at week 24 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）.
- Secondary Outcome Measures
Name Time Method Complete response(CR) rate at week 48 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）.
Partial response (PR) rate at week 48 week 48 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).
Rate of Participants with adverse effects and serious adverse effects during treatment baseline to week 48 Adverse effects include fever, rash, abnormal liver function, rate of new-onset infections and any abnormal measures after recivede study drug.