AUTOANTIBODIES AND SYSTEMIC SCLEROSIS (AUTO)

Systemic Sclerosis: the Role of Autoantibodies and Their Blockade in the Disease Pathogenesis. In Vitro and in Vivo Experimental Models

Established in vitro assays, combined with state-of-the-art multi-omics approaches and innovative in vitro experimental models, will gain insights into the mechanistic bases underlying Systemic Sclerosis (SS) pathogenesis and will dissect the consequences of treatment with Efgartigimod on functional and phenotypic cell behaviors. Healthy microvascular endothelial cells, fibroblasts and monocytes will be challenged with serum and autoantibodies prior to or upon treatment with Efgartigimod in order to assess both the prevention and the recovery capacity of the FcR blocker. The putative modulatory effect of Efgartigimod on vascular remodeling, endothelial-to-mesenchymal transition, fibroblast-to-myofibroblasts transition and monocyte behavior will be evaluated in in-vitro consolidated assays. Integrated Omics analyses will support and complement the in vitro findings by unveiling potential prognostic signatures and by identifying specific treatment-related profiles that might guide the modulation of the drug usage in order to maximize its beneficial effects.

Study Overview

• Status: Not yet recruiting
• Conditions: Understand Pathogenicity of SSc-specific Antibodies in SSc and the Effect of Antibody Blockade in Vitro
• Intervention / Treatment: Biological: Fcr blocker of autoantibodies

• Study Type: Observational
• Enrollment (Estimated): 6

Contacts and Locations

• Study Contact: Name: mARCO matucci cerinic, MD PHD; Phone Number: +393386249182; Email: matuccicerinic.marco@hsr.it

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

systemic sclerosis patients characterised by the autoantibody positivity

Description

Inclusion Criteria:

• positivity of autoantibodies

Exclusion Criteria:

• pregnant

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Verify if Fcblocker can block the pathogenic activity of autoantibodies	To assess both the prevention and the recovery capacity of the FcR blocker in different kind of cells submitted to the specific autoantibody. microvascular endothelial cells and fibroblasts	24 months

Collaborators and Investigators

• Sponsor: IRCCS Ospedale San Raffaele

Study record dates

Study Major Dates

• Study Start (Estimated): September 1, 2024
• Primary Completion (Estimated): December 31, 2025
• Study Completion (Estimated): December 31, 2025

Study Registration Dates

• First Submitted: July 9, 2024
• First Submitted That Met QC Criteria: July 9, 2024
• First Posted (Actual): July 16, 2024

Study Record Updates

• Last Update Posted (Actual): July 16, 2024
• Last Update Submitted That Met QC Criteria: July 9, 2024
• Last Verified: July 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Connective Tissue Diseases; Scleroderma, Systemic; Scleroderma, Diffuse; Physiological Effects of Drugs; Immunologic Factors; Autoantibodies
• Other Study ID Numbers: AUTO

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No