RecruitingPhase 2/3

Sirolimus in Patients With Systemic Lupus Erythematosus-Associated Immune Thrombocytopenia

China164 participantsStarted 2026-06-07

Plain-language summary

The goal of this clinical trial is to learn if sirolimus, added to standard background therapy (corticosteroids and hydroxychloroquine), works to treat systemic lupus erythematosus-associated immune thrombocytopenia (SLE-ITP) in adults aged 18 to 65 years. The study also aims to evaluate the long-term effectiveness and safety of sirolimus over 48 weeks. The main questions it aims to answer are: 1. Does sirolimus increase the overall response rate (complete or partial remission of thrombocytopenia) at 24 weeks compared with placebo? 2. The safety issues when taking sirolimus over 24 weeks (Phase 1) and up to 48 weeks (Phase 2). 3. How long does the treatment effect last, and what is the relapse rate during open-label extension? In Phase 1 (first 24 weeks), researchers will compare sirolimus to a placebo (an identical-looking capsule containing no active drug) to see if sirolimus works to treat SLE-ITP when both groups also receive standardized prednisone and hydroxychloroquine. In Phase 2 (weeks 24 to 48), all participants will receive open-label sirolimus to assess long-term efficacy and safety. Participants will: 1. Phase 1: Take sirolimus (1.5 mg once daily) or a placebo for 24 weeks, plus standardized prednisone (tapered according to protocol) and hydroxychloroquine (0.4 g daily) 2. Phase 2: After completing Phase 1, take open-label sirolimus (1.5 mg once daily) for an additional 24 weeks, continuing stable or tapering doses of prednisone and hydroxychloroquine 3. Visit the clinic at screening, baseline, weeks 4, 12, 24 (end of Phase 1), and then at weeks 28, 36, and 48 (end of Phase 2) for checkups, blood tests, and disease activity assessments 4. Receive telephone follow-ups at weeks 8, 16, 20, 32, 40, and 44 to report platelet counts, medication adherence, and adverse events

Who can participate

Age range

18 Years – 65 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

. Meet 2012 SLICC or 2019 EULAR/ACR criteria for systemic lupus erythematosus
. Meet ITP diagnostic criteria: platelet count ≤ 30×10\^9/L, or ≤ 50×10\^9/L with bleeding tendency
. At least two CBC tests showing thrombocytopenia
. Bone marrow smear showing megakaryocyte maturation disorder
. Willing to sign informed consent form

Exclusion criteria

. Other causes of thrombocytopenia

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.

1This trial is testing sirolimus specifically for ITP that's caused by lupus — how do my platelet levels and lupus activity right now compare to the kinds of patients this study is designed for, and is my situation a close enough match to make this worth discussing?
2Since this is a Phase 2/3 trial, there's still a lot being learned about how well sirolimus works for lupus-related ITP — what do you already know about sirolimus's safety profile from its use in other conditions, and what risks would be new territory in this specific setting?
3The trial measures 'overall response' at Week 24, which means committing to at least six months — given everything else I'm managing with my lupus, do you think I could realistically handle the monitoring schedule and any side effects that might come up over that time?
4Are there standard treatments for lupus-associated ITP — like corticosteroids, IVIG, or other immunosuppressants — that you'd recommend trying first before considering an experimental option like this trial?
5If I enrolled and didn't respond to sirolimus by Week 24, what would my options look like at that point, and would participating in this trial limit or delay any other treatments I might want to pursue?

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

Overall response rate (complete response + partial response) of ITP at Week 24

Timeframe: Week 24

Trial details

NCT IDNCT07657793

SponsorChinese SLE Treatment And Research Group

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2027-09-30

Contact for this trial

Liying Peng

86 010 69159958 liying_peng@qq.com

View on ClinicalTrials.gov More Systemic Lupus Erythematosus trials