Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia (SMART)

July 1, 2025 updated by: Zhuoli ZHANG, Peking University First Hospital

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 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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Complete response: the platelet count is more that 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

Study Type

Interventional

Enrollment (Estimated)

84

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • China
      • Beijing, China, 100034
        • Recruiting
        • Peking University First Hospital
        • Contact:
      • Beijing, China
        • Recruiting
        • Beijing Shijitan Hospital
        • Contact:
      • Beijing, China, 100020
        • Recruiting
        • Beijing Chao-Yang Hospital
        • Contact:
      • Beijing, China, 100083
        • Recruiting
        • Peking University Third Hospital
        • Contact:
      • Shanghai, China, 200001
        • Recruiting
        • Shanghai Renji Hospital
        • Contact:
    • Hunan
      • Changsha, Hunan, China, 410013
        • Recruiting
        • Xiangya Hospital of Central South University
        • Contact:
    • Shangdong
      • Jinan, Shangdong, China, 250012
        • Recruiting
        • Qilu Hospital of Shandong University
        • Contact:
    • Sichuan
      • Chengdu, Sichuan, China, 610041
        • Recruiting
        • West China Hospital, Sichuan University
        • Contact:
    • Xinjiang
      • Urumqi, Xinjiang, China, 830001
        • Recruiting
        • People's Hospital of Xinjiang Uygur Autonomous Region
        • Contact:
    • Zhejiang
      • Wenzhou, Zhejiang, China, 325015
        • Recruiting
        • The 1st Affiliated hospital of Wenzhou Medical University
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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
  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Treatment
Sirolimus two pills (1mg) per day
Drug: Sirolimus
Sirolimus two pills (1mg) per day
Other Names:
  • Rapamycin
  • mTOR inhibitor
Placebo Comparator: Control
Placebo two pills per day
Drug: Placebo
Placebo two pills per day

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Overall response
Time Frame: From enrollment to the end of treatment at 6 months
Both complete response and partial response
From enrollment to the end of treatment at 6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Complete response
Time Frame: From enrollment to the end of treatment at 6 months
Platelet count more than 100×10^9/L
From enrollment to the end of treatment at 6 months
Partial response
Time Frame: From enrollment to the end of treatment at 6 months
If the platelet count is less than 100×10^9/L, it should be more than 2 times of the baseline platelet count
From enrollment to the end of treatment at 6 months
The change of antiphospholipid antibodies titers
Time Frame: From enrollment to the end of treatment at 6 months
The changes of titers of antiphospholipid antibodies comparing to that of baseline
From enrollment to the end of treatment at 6 months
The change of oral glucocorticoids dosage
Time Frame: From enrollment to the end of treatment at 6 months
The differences of oral glucocorticoids dosage change between two groups
From enrollment to the end of treatment at 6 months

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Dropout rate
Time Frame: From enrollment to the end of treatment at 6 months
The differences of dropout rates between two groups
From enrollment to the end of treatment at 6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Peking University First Hospital

Collaborators

North China Pharmaceutical Co.,Ltd

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 7, 2025

Primary Completion (Estimated)

December 1, 2027

Study Completion (Estimated)

December 1, 2027

Study Registration Dates

First Submitted

December 4, 2024

First Submitted That Met QC Criteria

December 4, 2024

First Posted (Actual)

December 9, 2024

Study Record Updates

Last Update Posted (Actual)

July 4, 2025

Last Update Submitted That Met QC Criteria

July 1, 2025

Last Verified

January 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SMART_V2.2

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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