A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients
NCT ID: NCT06663358
Last Updated: 2025-11-14
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
100 participants
OBSERVATIONAL
2024-11-06
2029-11-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
OTHER
Study Groups
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Cardiac Cohort
Patients with Fabry-related cardiac disease
Pegunigalsidase-alfa
Administered via intravenous (IV) infusion under conditions of routine clinical care
Naïve Cohort
Patients naïve to prior Fabry disease treatment
Pegunigalsidase-alfa
Administered via intravenous (IV) infusion under conditions of routine clinical care
Long-Term Cohort
Patients previously participating in the pegunigalsidase alfa open label extension study and transitioning to routine care
Pegunigalsidase-alfa
Administered via intravenous (IV) infusion under conditions of routine clinical care
Interventions
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Pegunigalsidase-alfa
Administered via intravenous (IV) infusion under conditions of routine clinical care
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Genetically confirmed diagnosis of Fabry disease.
* Either taking or planning to take pegunigalsidase alfa as treatment for Fabry disease.
* No contraindications for cardiac magnetic resonance imaging (cMRI)
* Informed consent form (ICF) signed and dated indicating the individual has been informed of and agreed to all pertinent aspects of the study and is willing to comply with all study requirements, including completion of electronic patient reported outcomes (ePROs).
* Cardiac Cohort:
* Evidence of Fabry disease (FD)-related heart disease including one or more of the following:
* Left ventricular hypertrophy (LVH) measured by left ventricular mass index (LVMI) (g/m2) elevation above age/sex specific reference ranges.
* Posterior septum wall thickness (e.g., \>=13mm) not explained by other factors (e.g., hypertension)
* Low native T1 mapping on cMRI.
* Typical Fabry-like scar on cMRI
* Participants can receive cardiac magnetic resonance imaging (cMRI) with gadolinium enhancement as part of their SoC.
* Estimated glomerular filtration rate (eGFR) \>45 mL/min/1.73 m2, assessed within the prior 6 months.
* Naïve Cohort:
* Most recent eGFR\>45 mL/min/1.73 m2, assessed within prior 6 months.
* Male participants should have abnormal elevation in plasma lysoGb3 as assessed within 6 months prior to enrolment.
* Long-Term Cohort:
* Participants previously enrolled in the open label study CLI-06657AA1-04 (Previously PB-102-F60) (using pegunigalsidase alfa at a dose of 1mg/kg every 2 weeks) who have initiated or plan to initiate commercial pegunigalsidase alfa (Elfabrio®).
Exclusion Criteria
* Pregnant at the time of enrolment.
* Presence of any medical, emotional, behavioural, or psychological condition that, in the judgment of the physician, could interfere with the ability to participate in the study.
* Active participation in any interventional study for Fabry disease
* Treatment regimen at the time of enrolment in the study is different from the approved 1mg/kg every two weeks (note if regimen subsequently changes during the prospective part of the study, the participants can remain in the study)
* Prior participation in a pegunigalsidase alfa trial using a dose of 2 mg/kg administered every 4 weeks.
* Cardiac Cohort:
* History of acute myocardial infarction or congestive heart failure with reduced left ventricular (LV) ejection fraction of less than 35%.
* Cerebral vascular accident (CVA) in the prior 6 months.
* Chronic liver cirrhosis.
* FD-unrelated heart disease (e.g., scarring due to myocardial infarction, symptomatic occlusive coronary artery disease, moderate valvular heart disease not thought to be Fabry related).
* The participant is or has been treated with any investigational drug for Fabry disease within 6 months of study start or investigational gene therapy for Fabry disease at any time point in the past.
* Severe cardiac fibrosis defined as more than 3 segments that each have \>50% fibrosis upon late gadolinium enhancement cMRI at any prior cMRI.
* Naïve Cohort:
* Prior exposure to a FD therapy (Replagal®, Fabrazyme®, and Galafold®) at any time point.
* Severe cardiac fibrosis defined as more than 3 segments that each have \>50% fibrosis upon late gadolinium enhancement cMRI on any prior cMRI
18 Years
ALL
No
Sponsors
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Chiesi Farmaceutici S.p.A.
INDUSTRY
Responsible Party
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Locations
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University of Alabama at Birmingham
Birmingham, Alabama, United States
Emory University School of Medicine
Atlanta, Georgia, United States
Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States
Infusion Associates
Grand Rapids, Michigan, United States
Lysosomal & Rare Disorder Research & Treatment Center (LRDRTC)
Fairfax, Virginia, United States
General Hospital Slovenj Gradec
Slovenj Gradec, , Slovenia
University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital
Edgbaston, Birmingham, United Kingdom
Salford Royal
Salford, Greater Manchester, United Kingdom
The Royal Free Hospital
London, , United Kingdom
Countries
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Central Contacts
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Facility Contacts
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John Bernat
Role: primary
Michael Mawby, MD
Role: primary
Ozlem Goker-Alpan
Role: primary
Other Identifiers
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CLI-06657AA1-10
Identifier Type: -
Identifier Source: org_study_id
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