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A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients
NCT06663358 · View on ClinicalTrials.gov ↗
Study Summary
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 Studied
Interventions
- DRUG Pegunigalsidase-alfa
Study Locations (10)
Other
- General Hospital Slovenj Gradec — Slovenj Gradec
- The Royal Free Hospital — London
Alabama
- University of Alabama at Birmingham — Birmingham
Georgia
- Emory University School of Medicine — Atlanta
Illinois
- Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago
Iowa
- University of Iowa Hospitals and Clinics — Iowa City
Michigan
- Infusion Associates — Grand Rapids
Virginia
- Lysosomal & Rare Disorder Research & Treatment Center (LRDRTC) — Fairfax
Birmingham
- University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital — Edgbaston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 100 participants |
| Start Date | 2024-11-06 |
| Est. Completion | 2029-11 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06663358
The ClinicalTrials.gov registry entry for NCT06663358 describes a study currently listed as recruiting. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 100 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Chiesi Farmaceutici S.p.A., which has 7 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Fabry Disease appearing as the primary indexed condition, and to 1 intervention — of which Pegunigalsidase-alfa is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT06663358 reports 10 study locations spanning 9 distinct geographic areas — top geographies include Other, Alabama, Georgia. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT06663358 about?
NCT06663358 is a clinical study titled "A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients". 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.
What is the current status of trial NCT06663358?
This trial is currently recruiting. The enrollment target is 100 participants. The study started on 2024-11-06. Estimated completion is 2029-11.
What conditions does trial NCT06663358 study?
This clinical trial studies the following conditions: Fabry Disease. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT06663358?
The interventions under investigation include: Pegunigalsidase-alfa (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT06663358?
This trial is sponsored by Chiesi Farmaceutici S.p.A., which has 7 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT06663358 being conducted?
This trial has 10 study locations across Alabama, Georgia, Illinois, Iowa, Michigan. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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