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Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia
Sponsor: Peking University First Hospital
Summary
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
Official title: Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia: a Multicenter Randomized, Double Blind, Placebo Controlled, Clinical Trial
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
84
Start Date
2025-01-07
Completion Date
2027-12
Last Updated
2026-06-30
Healthy Volunteers
No
Interventions
Sirolimus
Sirolimus 1 mg (two pills) once daily during the 6-month double-blind phase; sirolimus 1.5 mg once daily during the optional 3-month open-label extension for eligible participants.
Placebo
Matching placebo two pills once daily during the 6-month double-blind phase; sirolimus 1.5 mg once daily during the optional 3-month open-label extension for eligible participants.
Locations (10)
Xiangya Hospital of Central South University
Changsha, Hunan, China
Qilu Hospital of Shandong University
Jinan, Shangdong, China
West China Hospital, Sichuan University
Chengdu, Sichuan, China
People's Hospital of Xinjiang Uygur Autonomous Region
Ürümqi, Xinjiang, China
The 1st Affiliated Hospital of Wenzhou Medical University
Wenzhou, Zhejiang, China
Beijing Chao-Yang Hospital
Beijing, China
Peking University First Hospital
Beijing, China
Peking University Third Hospital
Beijing, China
Beijing Shijitan Hospital
Beijing, China
Shanghai Renji Hospital
Shanghai, China