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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT06405152
Other study ID # Sobi.Emapalumab-105
Secondary ID
Status Recruiting
Phase
First received
Last updated
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
Health authority
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.

Study Design


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
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