Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia
Trial statusRecruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexAll
Age18+
SponsorPeking University First Hospital
About this 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.
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
Eligibility criteria
Qualifiers
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 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
Disqualifiers
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.
Trial design
Treatments tested in this trial
- Sirolimus
- Placebo
Treatment groups
84 Participants
are divided into 2 treatment groupsLocations
10Xiangya Hospital of Central South University410013, ChangshaHunan, China
Qilu Hospital of Shandong University250012, JinanShangdong, China
West China Hospital, Sichuan University610041, ChengduSichuan, China
People's Hospital of Xinjiang Uygur Autonomous Region830001, ÜrümqiXinjiang, China
Sponsors and collaborators
Peking University First Hospital
Lead sponsor
North China Pharmaceutical Co.,Ltd
Collaborator
Source ClinicalTrials.gov