Clinical trial NCT06992505: Assessment of Macrophage Activation syndromE in STill's Disease in Italy
Official title: Assessment of Macrophage Activation syndromE in STill's Disease: Retrospective Chart Analysis of Patient hIstory, Symptom Resolution and TreAtment Characteristics in Italy (AMETISTA)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Macrophage Activation Syndrome (MAS)
Still Disease, Juvenile Onset
Still's Disease, Adult-Onset
Lead sponsor: Swedish Orphan Biovitrum
Sponsor class: INDUSTRY
First posted: 2025-05-28
Enrollment: 35
Sex: ALL
Minimum age (years): 6
Maximum age (years): 80
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: RETROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: The study population will include patients with Still's disease who were diagnosed with MAS that was refractory to GC treatment.

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.

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.

Primary outcomes:
measure: Overall survival
timeFrame: from 2012 to 2022 (data abdstraction period)
measure: Time to laboratory value normalization
timeFrame: up to 24 weeks
measure: Time to MAS laboratory remission
timeFrame: up to 24 weeks
measure: Time to partial MAS laboratory remission
timeFrame: up to 24 weeks
measure: Time to tapering of GCs
timeFrame: from index date to the last of 7 consecutive days receiving ≤1 mg/kg/day of prednisone (PDN) equivalent dose
measure: Number of recurrent MAS episodes
timeFrame: from hospital discharge or 26-weeks from the index date, whichever occurs later, until the end of study data collection]
measure: Administration of organ support care
timeFrame: from the index date until either the hospital discharge or 26 weeks, whichever occurs later
measure: Characteristics of MAS treatment
timeFrame: up to 24 weeks
measure: Clinical signs
timeFrame: at index date and 8 weeks after index date (allowed time window 6 to 12 weeks)
Secondary outcomes:
measure: Time to hospital discharge
timeFrame: up to 24 weeks
measure: Time to intensive care unit (ICU) discharge
timeFrame: up to 24 weeks
measure: Normalization of key laboratory values
timeFrame: Week 8 after index date (allowed time window 6 to 12 weeks)
measure: MAS laboratory remission
timeFrame: at Week 8 after index date (allowed time window 6 to 12 weeks).
measure: Partial MAS laboratory remission
timeFrame: at Week 8 after index date (allowed time window 6 to 12 weeks)
measure: Duration of Clinical Response
timeFrame: up to 24 weeks
Other outcomes:
Locations:
city: Milan
country: Italy
facility: Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
city: Rome
country: Italy
facility: Ospedale Pediatrico Bambin Gesù
Study officials:
affiliation: Swedish Orphan Biovitrum
name: Study Physician
role: STUDY_DIRECTOR
References: