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
This trial does not accept healthy volunteersQualifiers
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
Design model
Parallel
Treatments tested in this trial
Sirolimus
DrugSirolimus 1 mg (two pills) once daily during the 6-month double-blind phase; sirolimus 1.5 mg once daily during the optional 3-month open-label extension for eligible participants.
Placebo
DrugMatching placebo two pills once daily during the 6-month double-blind phase; sirolimus 1.5 mg once daily during the optional 3-month open-label extension for eligible participants.
Treatment groups
84 Participants
are divided into 2 treatment groupsGroup A: TreatmentExperimental treatment 1 intervention
Group B: ControlPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
Percentage of participants with overall response at Month 6
Overall response is defined as achieving either complete response or partial response. Complete response is defined as platelet count \>=100 x 10\^9/L. Partial response is defined as platelet count \<100 x 10\^9/L with at least a 2-fold increase from baseline. Participants with missing Month 6 assessment or meeting prespecified treatment failure criteria will be counted as non-responders.
Time frame
Month 6Secondary outcomes
1
Percentage of participants with overall response at Month 1
Overall response is defined as achieving either complete response or partial response. Complete response is defined as platelet count \>=100 x 10\^9/L. Partial response is defined as platelet count \<100 x 10\^9/L with at least a 2-fold increase from baseline. Participants with missing Month 1 assessment or meeting prespecified treatment failure criteria will be counted as non-responders.
Time frame
Month 12
Percentage of participants with overall response at Month 3
Overall response is defined as achieving either complete response or partial response. Complete response is defined as platelet count \>=100 x 10\^9/L. Partial response is defined as platelet count \<100 x 10\^9/L with at least a 2-fold increase from baseline. Participants with missing Month 3 assessment or meeting prespecified treatment failure criteria will be counted as non-responders.
Time frame
Month 33
Percentage of participants with complete response at Month 6
Complete response is defined as platelet count \>=100 x 10\^9/L.
Time frame
Month 64
Percentage of participants with partial response at Month 6
Partial response is defined as platelet count \<100 x 10\^9/L with at least a 2-fold increase from baseline.
Time frame
Month 6Other outcomes
1
Percentage of open-label extension participants with overall response at Month 9
Overall response is defined as achieving either complete response or partial response. Complete response is defined as platelet count \>=100 x 10\^9/L. Partial response is defined as platelet count \<100 x 10\^9/L with at least a 2-fold increase from baseline.
Time frame
Month 92
Number of participants with serious adverse events during the double-blind treatment phase
Serious adverse events occurring after initiation of study treatment through the end of the 6-month double-blind treatment phase.
Time frame
From first dose through Month 6Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Peking University First Hospital
Lead sponsor
North China Pharmaceutical Co.,Ltd
Collaborator
Source ClinicalTrials.gov