Macrophage Activation Syndrome Clinical Trial
— AMETHYSTOfficial title:
Assessment of Macrophage Activation syndromE in STill's Disease: Retrospective Chart Analysis of Patient HistorY, Symptom Resolution and Treatment Characteristics
Assessment of Macrophage activation syndrome in STill's disease: retrospective chart analysis of patient History, Symptom resolution and Treatment characteristics
Status | Recruiting |
Enrollment | 80 |
Est. completion date | September 19, 2024 |
Est. primary completion date | September 19, 2024 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 6 Months to 80 Years |
Eligibility | Inclusion Criteria: - Age >6 months and =80 years at the beginning of the index MAS episode. - Diagnosis of Still's disease (sJIA (systemic Juvenile Idiopathic Arthritis) or AOSD (Adult Onset Stills Disease) 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. Exclusion Criteria: - A diagnosis of primary Hemophagocytic Lymphohistiocytosis (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. |
Country | Name | City | State |
---|---|---|---|
Canada | Swedish Orphan Biovitrum Research Site | Calgary | |
France | Swedish Orphan Biovitrum Research Site | Paris | |
Germany | Swedish Orphan Biovitrum Research Site | Heidelberg | |
Italy | Swedish Orphan Biovitrum Research Site | Milan | |
Italy | Swedish Orphan Biovitrum Research Site | Rome | |
Netherlands | Swedish Orphan Biovitrum Research Site | Utrecht | |
Spain | Swedish Orphan Biovitrum Research Site | Barcelona | |
United States | Swedish Orphan Biovitrum Research Site | Atlanta | Georgia |
United States | Swedish Orphan Biovitrum Research Site | Cincinnati | Ohio |
United States | Swedish Orphan Biovitrum Research Site | Los Angeles | California |
United States | Swedish Orphan Biovitrum Research Site | Philadelphia | Pennsylvania |
Lead Sponsor | Collaborator |
---|---|
Swedish Orphan Biovitrum |
United States, Canada, France, Germany, Italy, Netherlands, Spain,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Overall survival | The proportion of patients alive after 1-year of follow-up | 1 year | |
Primary | Time to laboratory value normalization | Time to normalization for the key laboratory values | From Index date up to 26 weeks | |
Primary | Time to Macrophage Activation Syndrome (MAS) laboratory remission | Time to normalization for all key laboratory values | From Index date up to 26 weeks | |
Primary | Time to partial MAS laboratory remission | Time to normalization for at least 3 key laboratory values | From Index date up to 26 weeks | |
Primary | Time to tapering of Glucocorticoids (GCs) | From index date to the last of 7 consecutive days receiving =1 mg/kg/day of prednisone (PDN). | from index date to the last of 7 consecutive days | |
Primary | Number of recurrent MAS episodes | Occurring any time after the end of the data collection period for the index MAS episode | through study completion | |
Primary | Administration of organ support care | Proportion of patients receiving hemodialysis, assisted ventilation, cardiac support, or inotropic drugs | from the index date until either the end of hospitalization or 26 weeks, whichever occurs later | |
Primary | Characteristics of MAS treatment | description of treatment patterns of MAS-related treatments for both index and recurrent MAS episodes. | for MAS episodes occurring during the data collection period | |
Primary | Clinical signs | No of MAS symptoms (Fever, Skin rash, Hemorrhagic manifestations, Evidence of CNS involvement) present | At index date and 8 weeks after index date or at the earliest time this data becomes available between 8 and 12 weeks | |
Secondary | Time to hospital discharge | Defined as time from the index date to discharge from the hospital | Up to 26 weeks | |
Secondary | Time to intensive care unit (ICU) discharge | For patients who are admitted to the ICU during the index MAS episode, this is defined as time from index date or ICU admission (whichever comes later) to ICU discharge | Up to 26 weeks | |
Secondary | Duration of Clinical Response | defined as the time (in days) from the date of first documented MAS laboratory remission until the date of first documented occurrence of a new MAS episode as per investigator's assessment. | Until week 26 |
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