Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia
SMART
1 other identifier
interventional
84
1 country
10
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_4
Started Jan 2025
Typical duration for phase_4
10 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
December 4, 2024
CompletedFirst Posted
Study publicly available on registry
December 9, 2024
CompletedStudy Start
First participant enrolled
January 7, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 1, 2027
July 4, 2025
January 1, 2025
2.9 years
December 4, 2024
July 1, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Overall response
Both complete response and partial response
From enrollment to the end of treatment at 6 months
Secondary Outcomes (4)
Complete response
From enrollment to the end of treatment at 6 months
Partial response
From enrollment to the end of treatment at 6 months
The change of antiphospholipid antibodies titers
From enrollment to the end of treatment at 6 months
The change of oral glucocorticoids dosage
From enrollment to the end of treatment at 6 months
Other Outcomes (1)
Dropout rate
From enrollment to the end of treatment at 6 months
Study Arms (2)
Treatment
EXPERIMENTALSirolimus two pills (1mg) per day
Control
PLACEBO COMPARATORPlacebo two pills per day
Interventions
Eligibility Criteria
You may qualify if:
- 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)
- Eligible concomitant treatment:
- 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
- anti-platelet and/or anti-coagulant therapy is allowed, and strength should be the stable for 1 week
- these following therapies should be discontinued for more than 5 half-lives before the enrollment, including thrombopoietin or thrombopoietin receptor antagonist, intravenous immunoglobulin, immunosuppressants, B cell inhibitors (Belimumab or Talitacicept) and B cell depletion therapy (Rituximab or Obinutuzumab).
You may not qualify if:
- 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.
- life or organ threatening manifestations, includes but not limit to catastrophic antiphospholipid syndrome and thrombotic microangiopathy.
- liver and renal dysfunction: ALT or AST more than three times of upper limit of normal range; eGFR\<40mL/min/1.73m\^2
- hematocytopenia: WBC\<3.0×10\^9/L, Hb\<100g/L.
- uncontrollable hyperlipidemia: low density lipoprotein cholesterol\>3.1 mmol/L, triglycerides\>2.3 mmol/L after lipid lowering therapy.
- current active infection
- women in pregnancy and postpartum period
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (10)
Xiangya Hospital of Central South University
Changsha, Hunan, 410013, China
Qilu Hospital of Shandong University
Jinan, Shangdong, 250012, China
West China Hospital, Sichuan University
Chengdu, Sichuan, 610041, China
People's Hospital of Xinjiang Uygur Autonomous Region
Ürümqi, Xinjiang, 830001, China
The 1st Affiliated Hospital of Wenzhou Medical University
Wenzhou, Zhejiang, 325015, China
Beijing Chao-Yang Hospital
Beijing, 100020, China
Peking University First Hospital
Beijing, 100034, China
Peking University Third Hospital
Beijing, 100083, China
Beijing Shijitan Hospital
Beijing, China
Shanghai Renji Hospital
Shanghai, 200001, China
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 4
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- MD, PhD, Director of Rheumatology and Immunology Department
Study Record Dates
First Submitted
December 4, 2024
First Posted
December 9, 2024
Study Start
January 7, 2025
Primary Completion (Estimated)
December 1, 2027
Study Completion (Estimated)
December 1, 2027
Last Updated
July 4, 2025
Record last verified: 2025-01