Clinical Assessment for Rheumatologic Disease - Research and Advancement in Safety and Efficacy

NCT07150000 | Recruiting

• 1 other identifier: 2025-169-BO
• Study Type: observational
• Target: 120
• Locations: 1 country
• Sites: 1

Brief Summary

The CARe RAiSE project represents a pioneering translational initiative aimed at advancing precision medicine in the treatment of autoimmune rheumatic diseases. The primary objective is the development and implementation of an innovative cell-based ex vivo assay that enables individualized prediction of therapeutic response to disease-modifying antirheumatic drugs (DMARDs). By identifying the most effective treatment option for each patient, this approach seeks to enhance therapeutic efficacy, reduce time to clinical response, and minimize healthcare costs. Despite the availability of numerous DMARDs, clinical decision-making remains largely empirical due to considerable interindividual variability in treatment response. This frequently results in a prolonged trial-and-error process, placing a significant burden on patients and the healthcare system. CARe RAiSE aims to overcome this limitation by providing a functional diagnostic tool that can predict a patient's immunological response to specific DMARDs prior to treatment initiation. The assay is based on peripheral blood mononuclear cells (PBMCs) obtained from individual patients, enabling a physiologically relevant assessment of immune responsiveness to targeted therapies. Combining high-content imaging with homogeneous well-based cytokine and inflammasome activity assays, the platform allows for a detailed single-cell analysis of inflammatory pathways. These data are used to generate predictive signatures of treatment response, thereby facilitating a mechanistically informed and personalized therapeutic strategy. Through this approach, CARe RAiSE introduces a scientifically grounded, efficient, and patient-specific method for DMARD selection, with the potential to substantially improve patient outcomes and reduce the socioeconomic impact of autoimmune rheumatic diseases.

Conditions

Rheumatic Diseases; Rheumatoid Arthritis (RA); Giant Cell Arteritis (GCA); Psoriatic Arthritis (PsA); Axial Spondylarthritis (axSpA); Polymyalgia Rheumatica (PMR); ANCA Associated Vasculitis (AAV); Connective Tissue Disease (CTD); Systemic Sclerosis (SSc); Systemic Lupus Erthematosus (SLE); Idiopathic Inflammatory Myopathy (IIM); Autoinflammatory Disease; Gout Arthritis

Outcome Measures

Primary Outcomes (1)

• Changes in Immune Cell Phenotype in the Ex-Vivo Assay

  Identification and quantification of immune cell subtypes, using the Ex-Vivo Essay. Unit of Measure: Changes in levels (percentages).

  Baseline. Follow-up after 6 weeks, 3 months, 6 months, 12 months and in case of relapse or therapy change. Cross sectional: Baseline. Healthy Controls: Baseline.

Study Arms (5)

Arthritis Group

Rheumatoid Arthritis, Psoriatic Arthritis, Axial Spondyloarthritis.

Diagnostic Test: Ex Vivo Assay

Vasculitis Group

Giant Cell Arteritis, Anca-associated Vasculitis.

Diagnostic Test: Ex Vivo Assay

Connective Tissue Disease Group

Systemic Lupus Erythematosus, Systemic Sclerosis, Mixed Connective Tissue Disease, Idiopathic Inflammatory Myopathies.

Diagnostic Test: Ex Vivo Assay

Autoinflammatory Disease Group

Familial Mediterranean Fever, Cryopyrin-associated Periodic Syndromes, TNF Receptor-Associated Periodic Syndrome, Adult-onset Still's disease, Gout.

Diagnostic Test: Ex Vivo Assay

Control Group

Age-/ gender matched healthy controls.

Diagnostic Test: Ex Vivo Assay

Interventions

Ex Vivo Assay DIAGNOSTIC_TEST

Ex vivo assay with patient's PBMCs. Cytokine quantification (e.g. TNFα, IL-1β). HCI with single-cell analysis (\>100 features/cell). Data preprocessing and normalization to transfer for machine learning.

Arthritis Group; Autoinflammatory Disease Group; Connective Tissue Disease Group; Control Group; Vasculitis Group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Participants will be recruited from the outpatient rheumatology clinic at the University Hospital Bonn. The study population includes adult patients (≥ 18 years) with a confirmed diagnosis of an autoimmune or autoinflammatory rheumatic disease, including: rheumatoid arthritis (RA), psoriatic arthritis (PsA), axial spondyloarthritis (axSpA), giant cell arteritis (GCA), connective tissue diseases \[systemic lupus erythematosus (SLE), systemic sclerosis (SSc), mixed connective tissue disease (MCTD), idiopathic inflammatory myopathies (IIM)\], ANCA-associated vasculitides \[microscopic polyangiitis (MPA), granulomatosis with polyangiitis (GPA), eosinophilic granulomatosis with polyangiitis (EGPA)\], and autoinflammatory disorders. In addition, age- and sex-matched healthy controls without known rheumatologic conditions will be included for comparative analysis. All participants must be able and willing to provide informed cons

You may qualify if:

• Participants aged ≥ 18 years
• Signed written informed consent to participate voluntarily in the study.
• Confirmed diagnosis (by the treating physician) of one of the following autoimmune or autoinflammatory rheumatic diseases:
• Rheumatoid arthritis (RA)
• Psoriatic arthritis (PsA)
• Axial spondyloarthritis (axSpA)
• Giant cell arteritis (GCA)
• Connective tissue diseases, including:
• Systemic lupus erythematosus (SLE)
• Systemic sclerosis (SSc)
• Mixed connective tissue disease (MCTD)
• Idiopathic inflammatory myopathies (IIM)
• ANCA-associated vasculitides (AAV), including:
• Microscopic polyangiitis (MPA)
• Granulomatosis with polyangiitis (GPA)

You may not qualify if:

• Refusal to participate in the study or inability to provide informed consent.
• Participants aged ≥ 18 years (capable of providing informed consent).
• Signed written informed consent to participate voluntarily in the study.
• \- Presence of a known or active rheumatologic disease.

Sponsors & Collaborators

• University of Bonn: lead

Study Sites (1)

University Hospital, Bonn

Bonn, North Rhine-Westphalia, 53127, Germany

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

Peripheral blood samples (EDTA, serum, PaxGene), biospecimens (e.g., synovial fluid, synovial tissue, skin biopsies).

MeSH Terms

Conditions

Rheumatic Diseases; Arthritis, Rheumatoid; Giant Cell Arteritis; Arthritis, Psoriatic; Polymyalgia Rheumatica; Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis; Connective Tissue Diseases; Scleroderma, Systemic; Myositis; Arthritis, Gouty

Condition Hierarchy (Ancestors)

Musculoskeletal Diseases; Skin and Connective Tissue Diseases; Arthritis; Joint Diseases; Autoimmune Diseases; Immune System Diseases; Vasculitis, Central Nervous System; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Vascular Diseases; Cardiovascular Diseases; Arteritis; Vasculitis; Skin Diseases, Vascular; Skin Diseases; Spondylarthropathies; Spondylarthritis; Spondylitis; Spinal Diseases; Bone Diseases; Psoriasis; Skin Diseases, Papulosquamous; Muscular Diseases; Systemic Vasculitis; Neuromuscular Diseases; Gout; Crystal Arthropathies; Purine-Pyrimidine Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Valentin S. Schäfer, MD

CONTACT

0049 228 287 17016; valentin.schaefer@ukbonn.de

Simon M. Petzinna, MD

CONTACT

0049 151 582 337 07; Simon_Micael.Petzinna@ukbonn.de

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Univ.-Prof. Dr. med. MUDr

Study Record Dates

• First Submitted: July 24, 2025
• First Posted: September 2, 2025
• Study Start: April 1, 2025
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2028
• Last Updated: September 2, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1)