Assessment of Macrophage Activation syndromE in STill's Disease in Italy

NCT ID: NCT06992505

Last Updated: 2025-12-02

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 32 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2025-06-06
• Study Completion Date: 2025-11-27

Brief Summary

Assessment of macrophage activation syndrome in Still's disease:

retrospective chart analysis of patient history, symptom resolution and treatment characteristics in Italy

Detailed Description

This is an observational, retrospective cohort study on the treatment utilization and outcomes of Macrophage Activation Syndrome (MAS) refractory to glucocorticoids (GC) in patients with Still's disease. The study will be conducted entirely through medical chart abstraction; all data will be taken from the patient's medical record, with no additional assessments. The study will be conducted in Italy.

Conditions

Macrophage Activation Syndrome (MAS); Still Disease, Juvenile Onset; Still's Disease, Adult-Onset

Study Design

• Observational Model Type: OTHER
• Study Time Perspective: RETROSPECTIVE

Eligibility Criteria

Inclusion Criteria

• Age >6 months and ≤80 years at the beginning of the index MAS episode.
• Diagnosis of Still's disease (sJIA or AOSD diagnosis).
• Diagnosis of MAS according to treating physician in the medical record.
• Patients who have received at least 3 consecutive days of GC after diagnosis of MAS and/or are judged by the Investigator to be refractory to GC due to clinical worsening of patient's condition.
• The onset of the index MAS episode occurred between 01 January 2012 and 30 September 2022.
• According to local regulations, waivers of consent will be sought for study participants from the appropriate regulatory authorities and/or the independent ethics committee (IEC)/institutional review board (IRB). o For patients not covered by waivers of consent, signed, and dated informed consent provided by the patient, or the patient's legally authorized representative(s) for patients under the legal age (with patient assent, as applicable) or who have died, should be obtained before any study-related activities are undertaken.

Exclusion Criteria

• A diagnosis of primary HLH prior to the beginning of the index MAS episode.
• Confirmed malignancy prior to the beginning of the index MAS episode.
• Patient treated with any investigational product as a part of clinical trial during the index MAS episode.

• Minimum Eligible Age: 6 Months
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Swedish Orphan Biovitrum

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Physician

Role: STUDY_DIRECTOR

Swedish Orphan Biovitrum

Locations

Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

Milan, , Italy

Ospedale Pediatrico Bambin Gesù

Rome, , Italy

Countries

Italy

Other Identifiers

Sobi.EMAPALUMAB-106

Identifier Type: -

Identifier Source: org_study_id