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Fabry patients get extended look at enzyme therapy safety

NCT ID NCT03614234

First seen Jan 26, 2026 · Last updated Apr 30, 2026 · Updated 13 times

Summary

This study checks the long-term safety and effectiveness of a drug called pegunigalsidase alfa in adults with Fabry disease. All 29 participants had already completed a previous study of the same drug. They receive an infusion every 4 weeks for up to several years. The goal is to see if the treatment remains safe and helps control the disease over time.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • Addenbrooke's Hospital

    Cambridge, CB2 0QQ, United Kingdom

  • Azienda Ospedaliera Universitaria "Federico II"

    Naples, Via Pansini, 80131, Italy

  • Emory University School of Medicine

    Atlanta, Georgia, 30307, United States

  • Fakultní poliklinika Všeobecné fakultní nemocnice v Praze

    Prague, 128 08, Czechia

  • Helse Bergen HF Haukeland Universitetssykehus

    Bergen, 5021, Norway

  • Infusion Associates

    Grand Rapids, Michigan, 49525, United States

  • Medical Endocrinology PE 2132, Rigshospitalet

    Copenhagen, 2100, Denmark

  • O & O Alpan

    Fairfax, Virginia, 22030, United States

  • Renal Disease Research Institute, LLC

    Dallas, Texas, 75235, United States

  • The Royal Free Hospital

    London, United Kingdom

  • UAB Medicine

    Birmingham, Alabama, 35233, United States

  • UZ Antwerpen

    Edegem, 2650, Belgium

  • University of Iowa Hospitals and Clinica

    Iowa City, Iowa, 52242, United States

  • University of Utah Hospitals & Clinics

    Salt Lake City, Utah, 84112, United States

Conditions

Explore the condition pages connected to this study.