New enzyme therapy shows promise for Long-Term fabry disease management

NCT ID NCT03566017

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

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study looked at the long-term safety of pegunigalsidase alfa, an enzyme replacement therapy, in 97 adults with Fabry disease. Participants received the drug intravenously every two weeks for up to several years. The main goal was to track side effects and see how well the drug was tolerated over time.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: pegunigalsidase alfa (a lab-made enzyme replacement therapy)
What this could lead to: If successful, this could confirm that pegunigalsidase alfa is a safe long-term treatment option for managing Fabry disease and slowing kidney damage.
What could go wrong: This is an open-label extension study with no placebo group, so results are less definitive. Side effects are possible, and the drug may not work for everyone.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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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

Academisch Medisch Centrum

Amsterdam, 1105 AZ, Netherlands
Addenbrooke's Hospital

Cambridge, CB2 0QQ, United Kingdom
Azienda Ospedaliera Universitaria "Federico II"

Naples, Via Pansini, 80131, Italy
Capital District Health Authority

Halifax, Nova Scotia, B3H 1V8, Canada
Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States
Emory University School of Medicine

Atlanta, Georgia, 30322, United States
General Hospital Slovenj Gradec

Slovenj Gradec, 2380, Slovenia
Haukeland University Hospital Klinisk Forskningspost

Bergen, 5021, Norway
Hospital Raymond-Poincaré

Garches, 92380, France
Hospital de Dia Quiron Zaragoza

Zaragoza, 50012, Spain
Infusion Associates

Grand Rapids, Michigan, 49525, United States
Institute of Metabolic Disease

Dallas, Texas, 75246, United States
Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States
O+O Alpan LLC

Fairfax, Virginia, 22030, United States
Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States
Renal Disease Research Institute, LLC - Dallas

Dallas, Texas, 75235, United States
Royal Melbourne Hospital

Parkville, Victoria, 3050, Australia
Salford Royal

Salford, Greater Manchester, M6 8HD, United Kingdom
Semmelweis Egyetem

Budapest, 1083, Hungary
The Royal Free Hospital

London, NW3 2QG, United Kingdom
Turku University Central Hospital

Turku, FI-20521, Finland
UAB Medicine

Birmingham, Alabama, 35233, United States
University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital

Edgbaston, Birmingham, B152TH, United Kingdom
University of California Irvine Center

Orange, California, 92868, United States
University of California San Diego

La Jolla, California, 92037, United States
University of Florida

Gainesville, Florida, 32610, United States
University of Iowa Hospitals and Clinics

Iowa City, Iowa, 52242, United States
University of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States
University of Utah Hospitals & Clinics

Salt Lake City, Utah, 84108, United States
Vseobecna fakultni nemocnice v Praze

Prague, Czech Republic, 12808, Czechia

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