Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE4

Total Enrollment

84 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-01-07

Study Completion Date

2027-12-31

Brief Summary

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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.

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Detailed Description

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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

Conditions

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Antiphospholipid (aPL)-Positive Thrombocytopaenia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Treatment

Sirolimus two pills (1mg) per day

Group Type EXPERIMENTAL

Sirolimus

Intervention Type DRUG

Sirolimus two pills (1mg) per day

Control

Placebo two pills per day

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo two pills per day

Interventions

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Sirolimus

Sirolimus two pills (1mg) per day

Intervention Type DRUG

Placebo

Placebo two pills per day

Intervention Type DRUG

Other Intervention Names

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Rapamycin mTOR inhibitor

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No