Brief Summary

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

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at P50-P75 for all trials

Timeline

Completed

Started Sep 2023

Shorter than P25 for all trials

Geographic Reach

7 countries

11 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

1 year study duration

Study Start

First participant enrolled

September 27, 2023

Completed

7 months until next milestone

First Submitted

Initial submission to the registry

April 9, 2024

Completed

29 days until next milestone

First Posted

Study publicly available on registry

May 8, 2024

Completed

5 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 26, 2024

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 26, 2024

Completed

Last Updated

October 21, 2024

Status Verified

October 1, 2024

Enrollment Period

1 year

First QC Date

April 9, 2024

Last Update Submit

October 18, 2024

Conditions

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

Outcome Measures

Primary Outcomes (9)

Overall survival
The proportion of patients alive after 1-year of follow-up
1 year
Time to laboratory value normalization
Time to normalization for the key laboratory values
From Index date up to 26 weeks
Time to Macrophage Activation Syndrome (MAS) laboratory remission
Time to normalization for all key laboratory values
From Index date up to 26 weeks
Time to partial MAS laboratory remission
Time to normalization for at least 3 key laboratory values
From Index date up to 26 weeks
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
Number of recurrent MAS episodes
Occurring any time after the end of the data collection period for the index MAS episode
through study completion
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
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
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 Outcomes (3)

Time to hospital discharge
Up to 26 weeks
Time to intensive care unit (ICU) discharge
Up to 26 weeks
Duration of Clinical Response
Until week 26

Interventions

Chart reviewOTHER

Retrospective Chart Review

Eligibility Criteria

Age6 Months - 80 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

Sampling MethodProbability Sample

Study Population

The study population will include patients with Still's disease who were diagnosed with MAS that was refractory to GC treatment.

You may qualify if:

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.

You may not qualify if:

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.

Contact the study team to confirm eligibility.