Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia

Launched by PEKING UNIVERSITY FIRST HOSPITAL · Dec 4, 2024

Keywords

ClinConnect Summary

This clinical trial is studying the use of a medication called sirolimus to treat patients with a condition known as antiphospholipid antibody-related thrombocytopenia, which means they have low platelet counts due to specific antibodies in their blood. The trial aims to find out how effective and safe sirolimus is compared to a placebo (a dummy treatment) over six months. Participants will be monitored at different points during the study to see how their condition improves.

To join the trial, participants should be between the ages of 65 and 74, have been diagnosed with certain antiphospholipid antibodies, and have had low platelet counts for at least two weeks. They should also be on stable doses of specific medications for a while. Throughout the study, participants will take sirolimus or a placebo daily and attend follow-up visits to track their health. This trial is currently recruiting participants, and it's important for potential volunteers to discuss any other medical conditions or treatments with their healthcare provider to see if they qualify.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 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 Criteria:
• • 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