Sirolimus Treatment for Antiphospholipid Antibody-Related Thrombocytopenia Using a Randomized, Double-Blind, Placebo-Controlled Phase 4 Trial

What this Trial Is About

Researchers are evaluating the efficacy and safety of sirolimus in patients with antiphospholipid antibody-associated thrombocytopenia. This condition involves low platelet counts linked to persistent positive antiphospholipid antibodies. The study is a phase 4 randomized, double-blind, placebo-controlled trial designed to compare sirolimus with placebo regarding overall response rates at 6 months. Participants are randomly assigned to receive either sirolimus 1 mg once daily or a matching placebo for 6 months during the double-blind phase. Those who do not reach the primary endpoint at 6 months may join a 3-month open-label extension receiving sirolimus 1.5 mg once daily. Follow-up visits occur at 2 weeks, 1 month, 3 months, and 6 months after enrollment, with additional monitoring through month 9 for those in the extension. During the study, participants will have their platelet counts and antibody levels measured, along with assessments of glucocorticoid dosage changes. Researchers will evaluate the overall, complete, and partial response rates at various time points. Safety outcomes and reasons for early discontinuation will also be monitored. The total participation may last up to 9 months, depending on extension eligibility.

Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia