Assessment of Macrophage Activation syndromE in STill's Disease

Macrophage Activation Syndrome Clinical Trial

— AMETHYST  

Official title:

Assessment of Macrophage Activation syndromE in STill's Disease: Retrospective Chart Analysis of Patient HistorY, Symptom Resolution and Treatment Characteristics

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06405152
• Other study ID #: Sobi.Emapalumab-105
• Status: Recruiting
• Start date: September 27, 2023
• Est. completion date: September 19, 2024

Study information

• Verified date: May 2024
• Source: Swedish Orphan Biovitrum
• Contact: Uwe Ullman, MD
• Phone: +46 8 6972000
• Email: uwe.ullman[@]sobi.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

Assessment of Macrophage activation syndrome in STill's disease: retrospective chart analysis of patient History, Symptom resolution and Treatment characteristics

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.

Recruitment information / eligibility

• 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.

Related Conditions & MeSH terms

• Arthritis, Juvenile
• Macrophage Activation Syndrome
• Still's Disease, Adult-Onset
• Syndrome

Intervention

Other:
• Chart review
Retrospective Chart Review

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Swedish Orphan Biovitrum

Countries where clinical trial is conducted

United States,  Canada,  France,  Germany,  Italy,  Netherlands,  Spain, 

Outcome

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