A Randomized, Multicenter, Double-blind, Placeobo-control Study of Sirolimus for Primary Antiphospholipid Syndrome Patients
Trial Snapshot
- Phase
- Phase 2
- Status
- Not yet recruiting
- Enrollment
- 70
- Primary Endpoint
- Partial response (PR) rate at week 24
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Triple (Participant, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 18 Years to 70 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •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
Exclusion Criteria
- •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
Arms & Interventions
Sirolimus
Sirolimus 1.5mg po. QD
Intervention: Sirolimus (Drug)
placeobo
Sirolimus placebo 1.5mg po. QD
Intervention: Placebo (Drug)
Outcomes
Primary Outcomes
Partial response (PR) rate at week 24
Time Frame: 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
Time Frame: 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 Outcomes
- Complete response(CR) rate at week 48(week 48)
- Partial response (PR) rate at week 48(week 48)
- Rate of Participants with adverse effects and serious adverse effects during treatment(baseline to week 48)
Investigators
Zhanguo Li
Department of Rheumatology and immunology
Peking University People's Hospital