Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia (NCT06722586) | Clinical Trial Compass
RecruitingPhase 4
Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia
China84 participantsStarted 2025-01-07
Plain-language 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
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* 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).
Exclusion Criteria:
* 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
* hematocytopen…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Percentage of participants with overall response at Month 6