Actively Recruiting
A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients
Led by Chiesi Farmaceutici S.p.A. · Updated on 2025-11-14
100
Participants Needed
10
Research Sites
260 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
A multi-centre, multi-country, observational, non-interventional, retrospective and prospective (hybrid) study among Fabry disease participants treated with pegunigalsidase alfa (Elfabrio®) in routine clinical care.
CONDITIONS
Official Title
A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female aged > 18 years at the time of consent
- Genetically confirmed diagnosis of Fabry disease
- Currently taking or planning to take pegunigalsidase alfa as treatment for Fabry disease
- No contraindications for cardiac magnetic resonance imaging (cMRI)
- Signed informed consent form agreeing to study participation and electronic patient-reported outcomes
- Cardiac Cohort: evidence of Fabry disease-related heart disease, including elevated left ventricular mass index, posterior septum wall thickness ≥13mm not explained by other causes, low native T1 mapping on cMRI, or typical Fabry-like scar on cMRI
- Ability to receive cMRI with gadolinium enhancement as part of standard care
- Estimated glomerular filtration rate (eGFR) >45 mL/min/1.73 m2 within prior 6 months
- Naïve Cohort: most recent eGFR >45 mL/min/1.73 m2 within prior 6 months
- Male participants in Naïve Cohort must have elevated plasma lysoGb3 within 6 months prior to enrollment
- Long-Term Cohort: previously enrolled in open-label study CLI-06657AA1-04 using pegunigalsidase alfa at 1mg/kg every 2 weeks and initiated or planning to initiate commercial pegunigalsidase alfa
You will not qualify if you...
- Contraindications to MRI including hypersensitivity to gadolinium contrast not manageable by premedication
- Pregnant at time of enrollment
- Any medical, emotional, behavioral, or psychological condition that may interfere with study participation
- Active participation in any interventional Fabry disease study
- Treatment regimen at enrollment different from 1mg/kg every two weeks (participants can remain if regimen changes later)
- Prior participation in a pegunigalsidase alfa trial using 2 mg/kg every 4 weeks
- Cardiac Cohort: history of acute myocardial infarction or heart failure with left ventricular ejection fraction <35%
- Cerebral vascular accident within prior 6 months
- Chronic liver cirrhosis
- Fabry-unrelated heart disease such as scarring from myocardial infarction or symptomatic coronary artery disease
- Treatment with any investigational Fabry drug within 6 months or any investigational gene therapy for Fabry disease ever
- Severe cardiac fibrosis defined as >3 segments with >50% fibrosis on late gadolinium enhancement cMRI
- Naïve Cohort: prior exposure to Fabry disease therapies (Replagal®, Fabrazyme®, Galafold®)
- Severe cardiac fibrosis as defined above on any prior cMRI
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 10 locations
1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35294
Actively Recruiting
2
Emory University School of Medicine
Atlanta, Georgia, United States, 30322
Actively Recruiting
3
Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
4
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States, 52242
Actively Recruiting
5
Infusion Associates
Grand Rapids, Michigan, United States, 49525
Actively Recruiting
6
Lysosomal & Rare Disorder Research & Treatment Center (LRDRTC)
Fairfax, Virginia, United States, 22030
Actively Recruiting
7
General Hospital Slovenj Gradec
Slovenj Gradec, Slovenia, 2380
Actively Recruiting
8
University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital
Edgbaston, Birmingham, United Kingdom, B152TH
Actively Recruiting
9
Salford Royal
Salford, Greater Manchester, United Kingdom, M6 8HD
Actively Recruiting
10
The Royal Free Hospital
London, United Kingdom, NW3 2QG
Actively Recruiting
Research Team
M
Medical Information Chiesi
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
3
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here