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
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are conducting a large, multi-country observational study to assess the safety and effectiveness of pegunigalsidase alfa (Elfabrio ) in patients with Fabry disease. This study includes patients who are currently treated with pegunigalsidase alfa in routine care and gathers data both retrospectively and prospectively. The study considers different patient groups such as those with Fabry-related heart disease, treatment-na ve patients, and those transitioning from an earlier open-label extension study. The treatment being observed is pegunigalsidase alfa, administered through intravenous infusion as part of routine clinical care. Participants are grouped into cohorts based on their disease status and treatment history, including a cardiac cohort with Fabry-related heart involvement, a na ve cohort who have not received prior Fabry treatment, and a long-term cohort of patients continuing from a previous clinical study. The study does not intervene in treatment but observes patients receiving this drug in real-world settings. Participants will be followed for up to four years, during which researchers will collect various clinical measurements such as kidney function (estimated glomerular filtration rate), plasma lysoGb3 levels, heart structure and function via left ventricular mass index and cardiac biomarkers, and overall safety assessments. Data will be collected through routine clinical visits, imaging studies, and patient-reported outcomes. The study aims to provide comprehensive long-term information on the effects and safety of pegunigalsidase alfa in everyday clinical practice.
CONDITIONS
Brief 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
- 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 requirements including completion of electronic patient reported outcomes (ePROs)
- Cardiac Cohort: evidence of Fabry-related heart disease including left ventricular hypertrophy (LVH), elevated left ventricular mass index (LVMI), posterior septum wall thickness ≥13mm not explained by other factors, or typical Fabry-like scar on cMRI
- Participants able to receive cMRI with gadolinium as part of standard care
- Estimated glomerular filtration rate (eGFR) >45 mL/min/1.73 m2 assessed within prior 6 months
- Na�ve Cohort: most recent eGFR >45 mL/min/1.73 m2 within prior 6 months
- Male participants in Na�ve Cohort should have abnormal elevation in plasma lysoGb3 within 6 months prior to enrolment
- Long-Term Cohort: previously enrolled in the open label study CLI-06657AA1-04 using pegunigalsidase alfa 1mg/kg every 2 weeks and initiating or planning to initiate commercial pegunigalsidase alfa
You will not qualify if you...
- Contraindication to MRI including known hypersensitivity to gadolinium contrast not managed by premedication
- Pregnant at time of enrolment
- Any medical, emotional, behavioural, or psychological condition interfering with study participation as judged by physician
- Active participation in any interventional Fabry disease study
- Treatment regimen different from approved 1mg/kg every two weeks at enrolment
- Prior participation in a pegunigalsidase alfa trial using 2 mg/kg every 4 weeks
- Cardiac Cohort: history of acute myocardial infarction, congestive heart failure with left ventricular ejection fraction <35%, cerebral vascular accident within prior 6 months, chronic liver cirrhosis, FD-unrelated heart disease, severe cardiac fibrosis (>3 segments with >50% fibrosis on cMRI)
- Na�ve Cohort: prior exposure to Fabry disease therapies (Replagal�, Fabrazyme�, Galafold�)
- Prior treatment with investigational Fabry drugs within 6 months or gene therapy at any time point
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 4 years
Participants who undergo routine intravenous pegunigalsidase alfa treatment as part of their clinical care are observed for safety and effectiveness of the therapy over time.
Visits in line with routine clinical care over 4 years
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
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
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