Actively Recruiting
Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia: A Multicenter Randomized, Double Blind, Placebo Controlled Clinical Trial
Led by Peking University First Hospital · Updated on 2025-07-04
84
Participants Needed
10
Research Sites
N/A
Total Duration
On this page
Sponsors
P
Peking University First Hospital
Lead Sponsor
N
North China Pharmaceutical Co.,Ltd
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the effectiveness and safety of sirolimus in treating thrombocytopenia associated with anti-phospholipid antibodies. This clinical trial aims to compare sirolimus with a placebo in patients who have persistent positive antiphospholipid antibodies and low platelet counts. The study primarily measures the overall response rate at 6 months, including complete and partial platelet count responses, changes in antibody levels, and steroid dosage adjustments. Participants receive either sirolimus at a daily dose of 1 mg (two pills) or a matching placebo. The study is randomized and double-blind, meaning neither the participants nor the researchers know which treatment is given. Assessments occur at 2 weeks, 1 month, 3 months, and 6 months after enrollment to monitor treatment effects and safety. During the study, participants will have regular blood tests to track platelet counts, antibody levels, and steroid use. Researchers will also record any dropouts and monitor for side effects or complications. The total participation lasts for 6 months, with visits scheduled to carefully observe responses to the study treatment and ensure participant safety.
CONDITIONS
Brief Title
Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Persistent positive antiphospholipid antibody (lupus anticoagulant, anti-cardiolipin antibody, or anti-b2GP1 antibody) confirmed at least twice, 12 weeks apart
- Persistent thrombocytopenia with platelet count between 30 and 100 x 10^9/L for at least 2 weeks
- Stable prednisone or equivalent dose less than 10 mg per day for more than 2 weeks allowed
- Stable hydroxychloroquine dose less than 400 mg per day for more than 1 month allowed
- Stable anti-platelet and/or anti-coagulant therapy for at least 1 week allowed
- Discontinuation of thrombopoietin or thrombopoietin receptor agonists, intravenous immunoglobulin, immunosuppressants, B cell inhibitors, and B cell depletion therapies for more than five half-lives before enrollment
You will not qualify if you...
- Diagnosis of other connective tissue diseases besides antiphospholipid syndrome
- Use of oral or intravenous antibiotics within 2 weeks before enrollment
- New onset thrombosis within 4 weeks before enrollment
- Apparent bleeding tendency
- Life- or organ-threatening conditions such as catastrophic antiphospholipid syndrome or thrombotic microangiopathy
- Liver dysfunction with ALT or AST more than three times the upper normal limit
- Renal dysfunction with eGFR less than 40 mL/min/1.73 m^2
- Hematocytopenia with WBC less than 3.0 x 10^9/L or hemoglobin less than 100 g/L
- Uncontrollable hyperlipidemia with LDL cholesterol over 3.1 mmol/L or triglycerides over 2.3 mmol/L after lipid-lowering therapy
- Current active infection
- Women who are pregnant or in the postpartum period
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 6 months
Participants receive sirolimus or placebo pills daily to treat antiphospholipid antibody related thrombocytopenia.
Visits scheduled throughout the 6-month treatment period
Trial Site Locations
Total: 10 locations
1
Xiangya Hospital of Central South University
Changsha, Hunan, China, 410013
Actively Recruiting
2
Qilu Hospital of Shandong University
Jinan, Shangdong, China, 250012
Actively Recruiting
3
West China Hospital, Sichuan University
Chengdu, Sichuan, China, 610041
Actively Recruiting
4
People's Hospital of Xinjiang Uygur Autonomous Region
Ürümqi, Xinjiang, China, 830001
Actively Recruiting
5
The 1st Affiliated Hospital of Wenzhou Medical University
Wenzhou, Zhejiang, China, 325015
Actively Recruiting
6
Beijing Chao-Yang Hospital
Beijing, China, 100020
Actively Recruiting
7
Peking University First Hospital
Beijing, China, 100034
Actively Recruiting
8
Peking University Third Hospital
Beijing, China, 100083
Actively Recruiting
9
Beijing Shijitan Hospital
Beijing, China
Actively Recruiting
10
Shanghai Renji Hospital
Shanghai, China, 200001
Actively Recruiting
Research Team
L
Lanlan Ji
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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