Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia

Phase 4

Recruiting

• Conditions: Antiphospholipid (aPL)-Positive; Thrombocytopaenia
• Interventions: Drug: Sirolimus; Drug: Placebo

• Registration Number: NCT06722586
• Lead Sponsor: Peking University First Hospital

Brief Summary

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 Participants will receive either sirolimus 1mg per day or placebo.

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 Timeline

• Study First Submitted: 2024-12-04
• Study First Submitted QC: 2024-12-04
• Study First Posted: 2024-12-09
• Status Verified: 2025-01
• Study Start: 2025-01-07
• Last Update Submitted: 2025-04-29
• Last Update Posted: 2025-04-30
• Primary Completion: 2027-12
• Study Completion: 2027-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 84

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)
• prednisone or equivalent dose less than 10mg per day, and dose stable for more than 2 weeks
• hydroxychloroquine less than 400mg per day, and dose stable for more than 1 month
• strength of anti-platelet and/or anti-coagulant therapy is stable for 1 week

Exclusion Criteria

• fulling the criteria of other connective tissue disease other than antiphospholipid syndrome
• received thrombopoietin or thrombopoietin receptor antagonist within 2 weeks before the enrollment; intravenous immunoglobulin within 1 month before the enrollment; immunosuppressants within 3 months before the enrollment; B cell inhibitors (Belimumab or Talitacicept) within 3 months before the enrollment; B cell depletion therapy (Rituximab) within 6 months before the enrollment.
• 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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Treatment	Sirolimus	Sirolimus two pills (1mg) per day
Control	Placebo	Placebo two pills per day

Primary Outcome Measures

Name	Time	Method
Overall response	From enrollment to the end of treatment at 6 months	Both complete response and partial response

Secondary Outcome Measures

Name	Time	Method
Complete response	From enrollment to the end of treatment at 6 months	Platelet count more than 100×10\^9/L
Partial response	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
The change of antiphospholipid antibodies titers	From enrollment to the end of treatment at 6 months	The changes of titers of antiphospholipid antibodies comparing to that of baseline

Trial Locations

Locations (9)

Xiangya Hospital of Central South University; 🇨🇳 Changsha, Hunan, China

The 1st Affiliated Hospital of Wenzhou Medical University; 🇨🇳 Wenzhou, Zhejiang, China

Beijing Shijitan Hospital; 🇨🇳 Beijing, China

Beijing Chao-Yang Hospital; 🇨🇳 Beijing, China

West China Hospital, Sichuan University; 🇨🇳 Chengdu, Sichuan, China

Peking University First Hospital; 🇨🇳 Beijing, China

Peking University Third Hospital; 🇨🇳 Beijing, China

Qilu Hospital of Shandong University; 🇨🇳 Jinan, Shangdong, China

People's Hospital of Xinjiang Uygur Autonomous Region; 🇨🇳 Urumqi, Xinjiang, China