Clinical trial NCT06722586: Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia
Official title: Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia: a Multicenter Randomized, Double Blind, Placebo Controlled, Clinical Trial
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: Antiphospholipid (aPL)-Positive
Thrombocytopaenia
Keywords: antiphospholipid
thrombocytopaenia
sirolimus
rapamycin
Lead sponsor: Peking University First Hospital
Sponsor class: OTHER
Collaborators: name: North China Pharmaceutical Co.,Ltd
First posted: 2024-12-09
Enrollment: 84
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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

Eligibility criteria: 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

Primary outcomes:
measure: Overall response
timeFrame: From enrollment to the end of treatment at 6 months
Secondary outcomes:
measure: Complete response
timeFrame: From enrollment to the end of treatment at 6 months
measure: Partial response
timeFrame: From enrollment to the end of treatment at 6 months
measure: The change of antiphospholipid antibodies titers
timeFrame: From enrollment to the end of treatment at 6 months
Other outcomes:
Locations:
city: Changsha
country: China
facility: Xiangya Hospital of Central South University
state: Hunan
city: Jinan
country: China
facility: Qilu Hospital of Shandong University
state: Shangdong
city: Chengdu
country: China
facility: West China Hospital, Sichuan University
state: Sichuan
city: Urumqi
country: China
facility: People's Hospital of Xinjiang Uygur Autonomous Region
state: Xinjiang
city: Wenzhou
country: China
facility: The 1st Affiliated Hospital of Wenzhou Medical University
state: Zhejiang
city: Beijing
country: China
facility: Beijing Chao-Yang Hospital
city: Beijing
country: China
facility: Peking University First Hospital
city: Beijing
country: China
facility: Peking University Third Hospital
city: Beijing
country: China
facility: Beijing Shijitan Hospital
city: Shanghai
country: China
facility: Shanghai Renji Hospital
Study officials:
References: