Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia
Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia: a Multicenter Randomized, Double Blind, Placebo Controlled, Clinical Trial
Peking University First Hospital
84 participants
Jan 7, 2025
INTERVENTIONAL
Conditions
Summary
The goal of this clinical trial is to learn the efficacy and safety of sirolimus in the treatment of anti-phospholipid antibody associated thrombocytopenia. The patients would be followed at 2 weeks, 1 month, 3 months, and 6 months after the enrollment. The main questions it aims to answer are the differences between sirolimus and control group at below outcomes: Primary outcome: the overall response rate at 6 months Secondary outcome: the complete response rate at 6 months the partial response rate at 6 months the change of anti-phospholipid antibody titers the change of oral glucocorticoids dosage Other pre-defined outcome: the dropout rate within 6 months Participants will receive either sirolimus 1mg per day or placebo.
Eligibility
Inclusion Criteria7
- persistent positive of antiphospholipid antibody (either lupus anticoagulant, anti-cardiolipin antibody, or anti-b2GP1 antibody, at least two times with 12 weeks apart)
- persistent thrombocytopenia (30-100×10\^9/L, at least for 2 weeks)
- Eligible concomitant treatment:
- prednisone or equivalent dose less than 10mg per day is allowed, and dose should be stable for more than 2 weeks
- hydroxychloroquine less than 400mg per day is allowed, and dose should be stable for more than 1 month
- anti-platelet and/or anti-coagulant therapy is allowed, and strength should be the stable for 1 week
- these following therapies should be discontinued for more than 5 half-lives before the enrollment, including thrombopoietin or thrombopoietin receptor antagonist, intravenous immunoglobulin, immunosuppressants, B cell inhibitors (Belimumab or Talitacicept) and B cell depletion therapy (Rituximab or Obinutuzumab).
Exclusion Criteria10
- fulling the criteria of other connective tissue disease other than antiphospholipid syndrome
- received oral/intravenous antibiotics within 2 weeks before the enrollment.
- new onset of thrombosis within 4 weeks before the enrollment.
- apparent bleeding tendency.
- life or organ threatening manifestations, includes but not limit to catastrophic antiphospholipid syndrome and thrombotic microangiopathy.
- liver and renal dysfunction: ALT or AST more than three times of upper limit of normal range; eGFR<40mL/min/1.73m\^2
- hematocytopenia: WBC<3.0×10\^9/L, Hb<100g/L.
- uncontrollable hyperlipidemia: low density lipoprotein cholesterol>3.1 mmol/L, triglycerides>2.3 mmol/L after lipid lowering therapy.
- current active infection
- women in pregnancy and postpartum period
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Interventions
Sirolimus two pills (1mg) per day
Placebo two pills per day
Locations(10)
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NCT06722586