A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients (NCT06663358)

🧪 Interventions in This Study

• Pegunigalsidase-alfa (DRUG)

Full eligibility criteria are available on the official NIH protocol. View the criteria ↗

📍 All Participating Trial Locations (10 Sites)

• University of Alabama at Birmingham — Birmingham, Alabama
• Emory University School of Medicine — Atlanta, Georgia
• Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois
• University of Iowa Hospitals and Clinics — Iowa City, Iowa
• Infusion Associates — Grand Rapids, Michigan
• Lysosomal & Rare Disorder Research & Treatment Center (LRDRTC) — Fairfax, Virginia
• General Hospital Slovenj Gradec — Slovenj Gradec,
• University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital — Edgbaston, Birmingham
• Salford Royal — Salford, Greater Manchester
• The Royal Free Hospital — London,

📞 Contact the Study Coordinator

Phone: (888) 661-9260

Email: us.medical@chiesi.com

Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.

💰 Cost, Insurance & Patient Rights

• 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
• Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
• Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

More studies in Fabry Disease

• A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease — Birmingham, Alabama
• A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease — Birmingham, Alabama
• Long-Term Follow-up of Subjects Who Were Treated With ST-920 — Irvine, California
• An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease — La Jolla, California
• A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease — Birmingham, Alabama
• A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease — Atlanta, Georgia

Explore by condition: All Fabry Disease clinical trials

💡 How to Participate

1. Check the checklist above to see if you may qualify.
2. Contact the study coordinator using the phone/email above.
3. Ask about the visit schedule, what is covered at no cost, and any travel support.
4. Confirm with your own doctor before making a decision.