Promising enzyme therapy shows Long-Term safety for rare fabry disease

NCT ID NCT03614234

Completed Disease control Sponsor: Chiesi Farmaceutici S.p.A. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study looked at the long-term safety and effectiveness of a drug called pegunigalsidase alfa for adults with Fabry disease, a rare genetic condition. Participants received an infusion every four weeks for up to several years. The goal was to see if the drug is safe over time and helps control the disease.

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Conditions

The condition(s) this trial relates to.

Fabry disease proteinuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

FABRY DISEASE

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

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