Effect of Belimumab on Antibody Titers in Primary APS Patients

A Single Center, Randomized Controlled, Open Label Trial to Explore the Regulatory Effect of Belimumab on Antibody Titers in Primary Antiphospholipid Syndrome Patients Carrying Medium to High Titers of Antiphospholipid Antibodies

The purpose of this study is to evaluate the regulatory effect of Belimumab on the antiphospholipid antibody (aPL) as well as to observe related past and new clinical events in primary antiphospholipid syndrome patients.

Study Overview

• Status: Recruiting
• Conditions: Antiphospholipid Syndrome (APS)
• Intervention / Treatment: Drug: SOC; Drug: Belimumab+SOC

Detailed Description

This is a single center, randomized controlled trial in Ruijin Hospital. The enrolled patients will be randomized in a 1: 1 ratio to receive either SOC+Belimumab or SOC treatment. Belimumab is administered intravenously at a dose of 10mg/kg once a month for 18 months.

• Study Type: Interventional
• Enrollment (Estimated): 20
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Hui Shi, Dr; Phone Number: 008613585515854; Email: sh12053@rjh.com.cn

Study Locations

• China
  • Shanghai
    • Shanghai, Shanghai, China, 200025
      • Recruiting
      • Ruijin Hospital
      • Contact: Chengde Yang, Dr; Phone Number: 0086-0021-64370045; Email: yangchengde@sina.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria:

• Age ≥ 18 years old.
• Positive for high-titer antiphospholipid antibodies according to the criteria of the "EULAR Guidelines for the Treatment of Antiphospholipid Syndrome in Adults" published in 2019.
• Stable APS regimen according to EULAR recommendations for antiphospholipid syndrome.
• Female patients who are not pregnant, breastfeeding, of childbearing potential, or not using contraception.

Exclusion Criteria:

• Patients with a history of malignant tumor in the past 5 years, except for basal cell carcinoma or squamous cell carcinoma of skin or cervical carcinoma in situ treated locally, and there is no evidence of metastasis within 3 years;
• Patients with a history of primary immunodeficiency;
• Serious lack of IgG (IgG level < 400 mg/dL);
• IgA deficiency (IgA level < 10 mg/dL);
• Patients with a current history of infection;
• Patients with a current history of drug or alcohol abuse or dependence, or have a history of drug or alcohol abuse or dependence within 365 days before day 0;
• HIV test is historically positive or HIV screening is positive;
• Hepatitis status;
• Patients with a history of allergic reaction caused by injection of contrast agent, human or mouse protein or monoclonal antibody;
• Patients with other abnormal laboratory values with clinical significance;
• If women with reproductive potential (WCBP) are included, please refer to the following special instructions;
• Patients with concurrent major medical or mental illnesss;
• Patients with diseases of liver, kidney, heart and other important organs,blood and Endocrine system;
• Patients who have been vaccinated with live vaccine in the last month;
• Patients who have participated in any clinical trial within 28 days before the initial screening and/or within 5 times of the half-life of the study compound (whichever is longer);
• Patients who use B-cell targeted therapy drugs within one year, such as rituximab or epratuzumab;
• Patients who use tumor necrosis factor inhibitor and interleukin receptor blocker within one year;
• Patients who use Intravenous gamma globulin (IVIG) and prednisone ≥100mg/d for more than 14 days within one year or plasma exchange;
• Patients with active infection (such as herpes zoster, HIV infection, active tuberculosis, etc.) during the screening period;
• Patients with depression or suicidal thoughts;
• Other conditions that the investigator considers would make the candidate unsuitable for the study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Experimental: SOC arm; SOC treatment includes aspirin and/or vitamin K antagonists (VKA) and/or low molecular weight heparin.	Drug: SOC; SOC treatment includes aspirin and/or vitamin K antagonists (VKA) and/or low molecular weight heparin.
Experimental: Experimental: SOC+Belimumab arm; Belimumab 10mg/kg once a month as an add-on treatment regimen. SOC treatment includes aspirin and/or vitamin K antagonists (VKA) and/or low molecular weight heparin.	Drug: SOC; SOC treatment includes aspirin and/or vitamin K antagonists (VKA) and/or low molecular weight heparin.; Drug: Belimumab+SOC; 10mg/kg once a month for 18 months

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
The level of antiphospholipid antibody titers	18 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
New thrombotic event	Any new thrombotic event during Belimumab treatment	18 months
Effects of Belimumab on the genotype, phenotype, and functional heterogeneity of T and B cells	Application of single-cell RNA sequencing technology (scRNAseq)	18 months

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
The incidence and severity of adverse events	Number and intensity of adverse events	18 months

Collaborators and Investigators

• Sponsor: Ruijin Hospital

Study record dates

Study Major Dates

• Study Start (Actual): April 1, 2024
• Primary Completion (Estimated): May 31, 2026
• Study Completion (Estimated): May 31, 2027

Study Registration Dates

• First Submitted: December 18, 2024
• First Submitted That Met QC Criteria: December 20, 2024
• First Posted (Actual): March 25, 2025

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: December 20, 2024
• Last Verified: July 1, 2024

More Information

Terms related to this study

• Keywords: Belimumab; Antiphospholipid Antibodies; Antiphospholipid Syndrome (APS)
• Additional Relevant MeSH Terms: Pathologic Processes; Autoimmune Diseases; Immune System Diseases; Disease; Syndrome; Antiphospholipid Syndrome; Immunosuppressive Agents; Immunologic Factors; Physiological Effects of Drugs; Belimumab
• Other Study ID Numbers: 2024136

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No