A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients

Part of paid clinical trials in Birmingham, Alabama.

Sponsor
Chiesi Farmaceutici S.p.A.
Study ID
NCT06663358
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Pegunigalsidase-alfa — DRUG
    Administered via intravenous (IV) infusion under conditions of routine clinical care

Study Details

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.

Key Dates

First listed
Oct 29, 2024
Start date
Nov 6, 2024
Status verified
Nov 2024
Primary completion
Nov 30, 2029
Completion
Nov 30, 2029

Study Design

Enrollment
100 participants (estimated)

Arms

  • Arm: Cardiac Cohort
    Patients with Fabry-related cardiac disease
  • Arm: Naïve Cohort
    Patients naïve to prior Fabry disease treatment
  • Arm: Long-Term Cohort
    Patients previously participating in the pegunigalsidase alfa open label extension study and transitioning to routine care

Primary Outcome Measure

Estimated Glomerular Filtration Rate (eGFR) [ Time Frame: 4 years ]

Central Contacts

Locations (6)

FacilityCityStateZIPSite coordinators
University of Alabama at BirminghamBirminghamAlabama35294
Eric Wallace, MD
[email protected]
+1 205-975-2935
Emory University School of MedicineAtlantaGeorgia30322
William Wilcox, MD
[email protected]
404-778-8518
Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinois60611
Carlos Prada, MD
[email protected]
+1 312-227-3724
University of Iowa Hospitals and ClinicsIowa CityIowa52242
John Bernat
319-356-2007
Infusion AssociatesGrand RapidsMichigan49525
Michael Mawby, MD
Lysosomal & Rare Disorder Research & Treatment Center (LRDRTC)FairfaxVirginia22030
Ozlem Goker-Alpan

Find similar trials in Birmingham, AL

By research site
University of Alabama at Birmingham· Birmingham, ALEmory University School of Medicine· Atlanta, GANorthwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago· Chicago, ILUniversity of Iowa Hospitals and Clinics· Iowa City, IAInfusion Associates· Grand Rapids, MILysosomal & Rare Disorder Research & Treatment Center (LRDRTC)· Fairfax, VA

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