NCT06722586

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

• Status: Recruiting
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 84 (estimated)
• Sponsor: Peking University First Hospital · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Complete response: the platelet count is more that 100×10\^9/L Partial response: If the platelet count is less than 100×10\^9/L, it should be more than 2 times of the baseline count Overall response: both complete and partial response

Conditions

• Antiphospholipid (aPL)-Positive
• Thrombocytopaenia

Interventions

Type	Name	Description
DRUG	Sirolimus	Sirolimus two pills (1mg) per day
DRUG	Placebo	Placebo two pills per day

Timeline

• Start date: 2025-01-07
• Primary completion: 2027-12-01
• Completion: 2027-12-01
• First posted: 2024-12-09
• Last updated: 2025-07-04

Locations

10 sites across 1 country: China