Clinical Autoinflammatory Disease Cohort

NCT07126145 | Not Yet Recruiting

• 1 other identifier: LY2025-170-A
• Study Type: observational
• Target: 1,000
• Locations: 0 countries
• Sites: N/A

Brief Summary

Autoinflammatory diseases (AIDs) are a group of rare disorders caused by dysregulation of the innate immune system, characterized by recurrent fever, systemic inflammation, and involvement of specific organs. Diagnosis relies on a combination of clinical features, laboratory tests, genetic findings, and response to treatment. Still's disease is a representative type of AID, marked by high spiking fevers, polyarthritis, evanescent rash, and markedly elevated inflammatory markers. Among its complications, macrophage activation syndrome (MAS) is the most life-threatening, affecting approximately 10-15% of patients. MAS involves uncontrolled immune activation and systemic inflammation, and if left untreated, may result in a mortality rate exceeding 50%. This study aims to develop a standardized clinical dataset and diagnostic-treatment framework for AIDs based on their disease characteristics. After establishing a data collection template, eligible patients aged 18-75 years with AIDs will be enrolled. Clinical data will be collected to build a prospective follow-up cohort, focusing particularly on adult-onset Still's disease (AOSD), to explore the clinical features and pathogenesis of AIDs.

Conditions

Still Disease; Autoinflammatory Disease

Outcome Measures

Primary Outcomes (1)

• Sustained Event-Free Remission

  Proportion of participants who maintain clinical remission without experiencing disease-related complications or treatment-related adverse events, including glucocorticoid-associated toxicity.

  From the date of remission until the first documented disease progression, occurrence of treatment-related severe adverse events, or death from any cause, whichever occurred first, assessed up to 60 months.

Secondary Outcomes (6)

• Glucocorticoid discontinuation rate

  3 months

• Time to biologic DMARD initiation

  From enrollment to 36 months

• Time to first MAS event

  From enrollment to 3 months

• Time to Onset of Glucocorticoid-Associated Complications

  From initiation of glucocorticoid therapy to the first documented complication, with follow-up of up to 60 months.

• Incidence of Treatment-Related Adverse Events

  From treatment initiation to 36 months

Study Arms (1)

AIDs patients

Patients with Still's disease must meet the 1992 Yamaguchi diagnostic criteria; patients with monogenic autoinflammatory diseases must meet the confirmed diagnostic criteria for the respective conditions; all patients must provide written informed consent.；Age range: 18-75 years old patients with autoinflammatory diseases.

Other: Not applicable- observational study

Interventions

Not applicable- observational study OTHER

no interventions were involved

AIDs patients

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Adult patients (aged 18-75 years) diagnosed with autoinflammatory diseases.

You may qualify if:

• Diagnosis of adult-onset Still's disease (AOSD) according to the Yamaguchi criteria (as described above) or other established autoinflammatory disease diagnostic criteria.
• Age between 18 and 75 years, inclusive.
• Willingness and ability to comply with scheduled follow-up visits, examinations, and treatments.
• Voluntary provision of written informed consent.

You may not qualify if:

• Refusal or inability to provide informed consent; age under 18 or over 75 years; inability to provide at least 80% of the required core data for Still's disease or other autoinflammatory diseases.
• Presence of other connective tissue diseases.
• Positive detection of autoantibodies.
• Current acute infection or history of active tuberculosis.
• History of allergic constitution or multiple drug allergies.
• Psychiatric disorders or other conditions preventing compliance with examinations, follow-up, or treatment.
• Women who are currently pregnant or planning to become pregnant.
• Presence of malignant tumors.
• Participation in other clinical trials currently or within a specified washout period.

Sponsors & Collaborators

• RenJi Hospital: lead

MeSH Terms

Conditions

Arthritis, Juvenile

Condition Hierarchy (Ancestors)

Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Target Duration: 12 Months
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director

Study Record Dates

• First Submitted: August 2, 2025
• First Posted: August 17, 2025
• Study Start: August 1, 2025
• Primary Completion (Estimated): August 1, 2030
• Study Completion (Estimated): August 1, 2035
• Last Updated: August 17, 2025

Record last verified: 2025-08