Antiphospholipid (aPL)-PositivePhase 4SMARTNCT06722586
Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia
A Phase 4 study testing Sirolimus in people with Antiphospholipid (aPL)-Positive and Thrombocytopaenia, run by Peking University First Hospital at 10 sites in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Antiphospholipid (aPL)-Positive, Thrombocytopaenia
- What is being tested?
- Sirolimus
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 84 participants
- Where
- 10 sitesChina
- Study dates
- 7 Jan 2025to Dec 2027
- Phase
- Phase 4
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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).
Who cannot join
- 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
Show all 10Show fewer
- 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
Trial locations and contacts
10 sites in 1 country. Contact the study team or a site near you to ask about taking part.
10 sites in 1 country
China10 sites, 10 recruiting
- Recruiting
Beijing Chao-Yang Hospital
Beijing, 100020
Juan Meng, +86-010-85231777, mserena@163.com
- Recruiting
Peking University First Hospital
Beijing, 100034
Zhuoli Zhang, +86-010-83575130, zhuoli.zhang@126.com
- Recruiting
Peking University Third Hospital
Beijing, 100083
Rong Mu, +86-010-82264567, murongster@163.com
- Recruiting
Beijing Shijitan Hospital
Beijing
Yuhua Wang, +86-010-63925588, wangyh@bjsjth.cn
- Recruiting
Xiangya Hospital of Central South University
Changsha, Hunan, 410013
Hui Luo, +86-0731-89753999, luohui@csu.edu.cn
- Recruiting
West China Hospital, Sichuan University
Chengdu, Sichuan, 610041
Qibing Xie, +86-028-85422114, xieqibing1971@163.com
- Recruiting
Qilu Hospital of Shandong University
Jinan, Shangdong, 250012
Qiang Shu, +86-531-8216-9114, shuqiang@sdu.edu.cn
- Recruiting
Shanghai Renji Hospital
Shanghai, 200001
Ting Li, +86-021-5388 2125, leeting007@163.com
- Recruiting
People's Hospital of Xinjiang Uygur Autonomous Region
Ürümqi, Xinjiang, 830001
Lijun Wu, +86-991-856-5302, wwlj330@126.com
- Recruiting
The 1st Affiliated Hospital of Wenzhou Medical University
Wenzhou, Zhejiang, 325015
Li Sun, +86-0577-55578037, grassandsun@126.com
About this study
Official title: Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia: a Multicenter Randomized, Double Blind, Placebo Controlled, Clinical Trial
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.
Read more
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
Read the detailed description
Complete response: the platelet count is more than or equal to 100×10^9/L Partial response: If the platelet count is less than 100×10^9/L, it should be more than 2 times of the baseline count Overall response: both complete and partial response
- Conditions
- Antiphospholipid (aPL)-Positive, Thrombocytopaenia
- Being tested
- Sirolimus, Placebo
- Keywords
- antiphospholipid, thrombocytopaenia, sirolimus, rapamycin, mTOR inhibitor
- Medical subject headings
- Thrombocytopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SirolimusDrug
Also called Rapamycin, mTOR inhibitor
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.
PlaceboDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | Participants receive sirolimus 1 mg (two pills) once daily for 6 months during the randomized, double-blind phase. Participants who do not achieve the primary endpoint at Month 6 may enter a 3-month open-label extension and receive sirolimus 1.5 mg once daily. Receives: Drug: Sirolimus |
| Control | Placebo comparator | Participants receive matching placebo (two pills) once daily for 6 months during the randomized, double-blind phase. Participants who do not achieve the primary endpoint at Month 6 may enter a 3-month open-label extension and receive sirolimus 1.5 mg once daily. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of participants with overall response at Month 6
Overall response is defined as achieving either complete response or partial response. Complete response is defined as platelet count >=100 x 10^9/L. Partial response is defined as platelet count <100 x 10^9/L with at least a 2-fold increase from baseline. Participants with missing Month 6 assessment or meeting prespecified treatment failure criteria will be counted as non-responders.
Measured: Month 6
Secondary outcomes (6)
Percentage of participants with overall response at Month 1
Overall response is defined as achieving either complete response or partial response. Complete response is defined as platelet count >=100 x 10^9/L. Partial response is defined as platelet count <100 x 10^9/L with at least a 2-fold increase from baseline. Participants with missing Month 1 assessment or meeting prespecified treatment failure criteria will be counted as non-responders.
Measured: Month 1
Percentage of participants with overall response at Month 3
Overall response is defined as achieving either complete response or partial response. Complete response is defined as platelet count >=100 x 10^9/L. Partial response is defined as platelet count <100 x 10^9/L with at least a 2-fold increase from baseline. Participants with missing Month 3 assessment or meeting prespecified treatment failure criteria will be counted as non-responders.
Measured: Month 3
Percentage of participants with complete response at Month 6
Complete response is defined as platelet count >=100 x 10^9/L.
Measured: Month 6
Percentage of participants with partial response at Month 6
Partial response is defined as platelet count <100 x 10^9/L with at least a 2-fold increase from baseline.
Measured: Month 6
Change from baseline in Global Antiphospholipid Syndrome Score (GAPSS) at Month 6
GAPSS will be calculated at baseline and Month 6 according to the prespecified scoring algorithm based on antiphospholipid antibody profile and cardiovascular risk factors. The outcome will be reported as the change in GAPSS from baseline to Month 6, calculated as Month 6 GAPSS minus baseline GAPSS. A negative value indicates a decrease in GAPSS.
Measured: Baseline and Month 6
Change from baseline in oral glucocorticoid dose at Month 6
Oral glucocorticoid dose will be converted to prednisone-equivalent dose and summarized as the change from baseline to Month 6.
Measured: Baseline and Month 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2024
- Study start7 Jan 2025
- Primary completionDec 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Antiphospholipid (aPL)-PositiveStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Participants are randomized 1:1 to receive sirolimus 1.0 mg or matching placebo during a 6-month double-blind, parallel-group phase. Participants who do not achieve the primary endpoint at Month 6 may enter a 3-month open-label extension and receive sirolimus 1.5 mg.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 84 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University First Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- North China Pharmaceutical Co.,Ltd
- Responsible party
- Principal investigator, Zhuoli ZHANG, Peking University First Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06722586
- Sponsor's protocol ID
- SMART_V3.0
- Other IDs
- 2024HQ06
- Acronym
- SMART
- Status last verified
- Jun 2026
- First submitted
- 4 Dec 2024
- Last updated
- 30 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: The de-identified participant-level STAR data underlying this study are not publicly available because they contain sensitive clinical information and public release is not covered by the applicable participant consent and ethics approval. Access may be requested from Zhuoli Zhang (zhuoli.zhang@126.com). Requests will be evaluated on the basis of scientific merit, participant privacy, ethical and institutional requirements, and the availability of a suitable data-use agreement.