Clinical trial NCT06923124: A Study to Describe a Group of Spanish Patients With FMF and to Identify Suboptimally Treated Patients With Minimal Disease Activity and Its Impact on Quality of Life
Official title: A Retrospective, Non-interventional, Single-center Study to Describe a Spanish Cohort of Patients With FMF and to Identify Suboptimally Treated Patients With Minimal Disease Activity and Its Impact on HRQoL (Health-Related Quality of Life)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Familial Mediterranean Fever
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2025-04-11
Enrollment: 44
Sex: ALL
Maximum age (years): 28
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: This was a retrospective, non-interventional cohort study.

Brief summary: A retrospective, non-interventional, single-center study conducted in Spain, based on the collection of data from the medical histories of patients with Familial Mediterranean fever (FMF) diagnosed during childhood from January 2005 to December 2021.

Data from the patients' medical records was collected retrospectively, from the time of diagnosis to the current moment of the patient's enrollment in the study. Patients had a minimum follow-up of 3 months after diagnosis.


Eligibility criteria: Inclusion criteria:

1. Patients with FMF clinically and/or genetically diagnosed during childhood according to the site's clinical practice.
2. Patients with at least 3 months of follow-up from the date of diagnosis, at the time of their enrollment in the study.

Exclusion criteria:

1. Patients with autoinflammatory disease excluding FMF.
2. Patients with recurrent fever of another etiology (infectious, tumor or other).

Primary outcomes:
measure: Number of Patients per Clinical Characteristic Category
timeFrame: Baseline
measure: Clinical Characteristic: Time From FMF Diagnosis to Study Inclusion
timeFrame: Baseline
measure: Clinical Characteristic: Time From the Start of Symptomatology to Study Inclusion
timeFrame: Baseline
measure: Clinical Characteristic: Time From the Start of Symptomatology to FMF Diagnosis
timeFrame: Baseline
measure: Clinical Characteristic: Age at the Start of Symptomatology
timeFrame: Baseline
measure: Number of Patients by Symptomatology
timeFrame: Baseline, Month 3, 6, 9, 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, and 132
measure: Number of Patients by Characteristics of Relapses (Flares)
timeFrame: Baseline, Month 3, 6, 9, 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, and 132
measure: Flare and Non-flare Related Inflammatory Marker Levels
timeFrame: Baseline, Month 3, 6, 9, 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, and 132
measure: Flare and Non-flare Related Erythrocyte Sedimentation Rate (ESR)
timeFrame: Baseline, Month 3, 6, 9, 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, and 132
measure: Number of Flares per Month Since Last Visit
timeFrame: Baseline, Month 3, 6, 9, 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, and 132
measure: Flare Duration Since Last Visit
timeFrame: Baseline, Month 3, 6, 9, 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, and 132
measure: Number of Patients With Recurrency of Flares at Diagnosis
timeFrame: Baseline
measure: Number of Patients by Comorbidity
timeFrame: Baseline, Month 3, 6, 9, 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, and 132
measure: Number of Patients by Treatment Received Before FMF Diagnosis
timeFrame: Baseline
measure: Number of Patients by Treatment Received At Least Once During the Study
timeFrame: Up to 11 years
measure: Number of Patients by Treatment and Treatment Combinations Used at the end of the Study
timeFrame: Up to 11 years
measure: Number of Patients Using Colchicine With Another Treatment at the end of the Study
timeFrame: Up to 11 years
measure: Number of Patients by First Prescribed Colchicine Dose and Age Group
timeFrame: Up to 11 years
measure: Number of Patients by Colchicine Maximum Global Dose and Age Group
timeFrame: Up to 11 years
measure: Number of Patients With a Change in Colchicine Dose
timeFrame: Up to 11 years
measure: Number of Patients by Reason for Stopping Colchicine Treatment and Age Group
timeFrame: Up to 11 years
measure: Number of Patients by Anti-IL-1 Treatment Used at the end of the Study
timeFrame: Up to 11 years
measure: Number of Patients by Anti-IL-1 Dose at the end of the Study
timeFrame: Up to 11 years
measure: Number of Patients With a Change in Anti-IL-1 Dose
timeFrame: Up to 11 years
measure: Number of Patients With a Change in Anti-IL-1 Treatment
timeFrame: Up to 11 years
measure: Number of Patients by Reason for Stopping Anti-IL-1 Treatment
timeFrame: Up to 11 years
Secondary outcomes:
measure: Number of Patients by Last Reported Response to Colchicine Treatment
timeFrame: Up to 11 years
measure: Time to Achieve Response to Colchicine Treatment
timeFrame: Up to 11 years
measure: Number of Patients With Intolerance to Colchicine Treatment
timeFrame: Up to 11 years
measure: Autoinflammatory Disease Activity Index (AIDAI) Score
timeFrame: Up to 11 years
measure: Autoinflammatory Disease Damage Index (ADDI) Score
timeFrame: Up to 11 years
measure: Familial Mediterranean Fever 50 (FMF50) Score
timeFrame: Up to 11 years
measure: Number of Patients by Last Response Reported to Anti-IL-1 Treatment During the Study
timeFrame: Up to 11 years
measure: Time to Achieve Response to Anti-IL-1 Treatment
timeFrame: Up to 11 years
measure: Number of Patients With MEFV Mutation by Treatment Response
timeFrame: Up to 11 years
measure: Number of Patients Without MEFV Mutation by Treatment Response
timeFrame: Up to 11 years
measure: Number of Patients With Mutation in Other Loci by Treatment Response
timeFrame: Up to 11 years
measure: Number of Medical Visits of Patients With Scholar Absenteeism During Flares by Response to Anti-IL-1 Treatment
timeFrame: Up to 11 years
measure: Number of Medical Visits of Patients With Physical Activity Limitations by Response to Anti-IL-1 Treatment
timeFrame: Up to 11 years
measure: Number of Medical Visits of Patients Requiring Psychological Care by Response to Anti-IL-1 Treatment
timeFrame: Up to 11 years
measure: Number of Medical Visits of Patients With Presence of Pain by Response to Anti-IL-1 Treatment
timeFrame: Up to 11 years
measure: Number of Medical Visits of Patients With Presence of Fatigue by Response to Anti-IL-1 Treatment
timeFrame: Up to 11 years
measure: Visual Analogue Scale (VAS) Score From Clinician Point of View
timeFrame: Up to 11 years
measure: Visual Analogue Scale (VAS) Score From Patient Point of View
timeFrame: Up to 11 years
Other outcomes:
Locations:
city: East Hanover
country: United States
facility: Novartis
state: New Jersey
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: