Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia
Trial statusRecruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexAll
Age18+
SponsorPeking University First Hospital
About this trial
The goal of this clinical trial is to evaluate the efficacy and safety of sirolimus in patients with anti-phospholipid antibody-associated thrombocytopenia.
In the 6-month randomized, double-blind, placebo-controlled phase, participants will receive either sirolimus 1 mg once daily or matching placebo. Participants will be followed at 2 weeks, 1 month, 3 months, and 6 months after enrollment.
Participants who do not achieve the primary endpoint at 6 months may enter a 3-month open-label extension and receive sirolimus 1.5 mg once daily, with follow-up through month 9.
The main question is whether sirolimus improves the overall response rate at 6 months compared with placebo.
Primary outcome:
\- Overall response rate at 6 months
Secondary outcomes:
* Overall response rate at 1 month and 3 months * Complete response rate at 6 months * Partial response rate at 6 months * Change in anti-phospholipid antibody titers at 6 months * Change in oral glucocorticoid dosage at 6 months
Exploratory outcomes:
* Proportion and reasons for early discontinuation during the double-blind phase * Among participants who do not achieve the primary endpoint at 6 months and enter the open-label extension, overall response rate, complete response rate, partial response rate, and safety outcomes after 3 months of open-label treatment
Eligibility criteria
Qualifiers
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)
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
Disqualifiers
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.
Trial design
Treatments tested in this trial
- Sirolimus
- Placebo
Treatment groups
84 Participants
are divided into 2 treatment groupsSponsors and collaborators
Peking University First Hospital
Lead sponsor
North China Pharmaceutical Co.,Ltd
Collaborator
Source ClinicalTrials.gov