Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia

NCT06722586 · tracked via the Priya Life Science China tracker

Sponsor

Peking University First Hospital

Phase

Phase 4

Started

2025-01-07

Last updated

2026-06-30

Condition(s) studied

Antiphospholipid (aPL)-PositiveThrombocytopaenia

Investigational drug(s) / intervention(s)

Sirolimus →Placebo

Sirolimus: Sirolimus 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: Matching 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.

Study summary

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

Sex

ALL

Min age

18 Years

Max age

—

Healthy volunteers

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

Primary outcome measure(s)

Percentage of participants with overall response at Month 6 — 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.

Trial sites (10)

Facility	City	Region	Status
Xiangya Hospital of Central South University	Changsha	Hunan	Recruiting
Qilu Hospital of Shandong University	Jinan	Shangdong	Recruiting
West China Hospital, Sichuan University	Chengdu	Sichuan	Recruiting
People's Hospital of Xinjiang Uygur Autonomous Region	Ürümqi	Xinjiang	Recruiting
The 1st Affiliated Hospital of Wenzhou Medical University	Wenzhou	Zhejiang	Recruiting
Beijing Chao-Yang Hospital	Beijing	China	Recruiting
Peking University First Hospital	Beijing	China	Recruiting
Peking University Third Hospital	Beijing	China	Recruiting
Beijing Shijitan Hospital	Beijing	China	Recruiting
Shanghai Renji Hospital	Shanghai	China	Recruiting

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06722586 on ClinicalTrials.gov ↗ ← All trials in China