Promising enzyme therapy for kids with rare fabry disease enters key trial

NCT ID NCT06328608

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

First seen Jun 27, 2026 · Last updated Aug 11, 2026 · Updated 2 times

Summary

This study tests a drug called PRX-102 (pegunigalsidase alfa) in 22 children and teens aged 2 to 18 with Fabry disease, a rare genetic disorder that causes pain and organ damage. The drug is an enzyme replacement therapy given by infusion every two weeks. Researchers will monitor safety, side effects, and how well it controls disease symptoms like pain and kidney function.

What this could mean

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

Active substance: PRX-102 (pegunigalsidase alfa), a lab-made enzyme that replaces the missing or faulty enzyme in Fabry disease
What this could lead to: If successful, this could provide a treatment option for children with Fabry disease to help manage symptoms and slow disease progression.
What could go wrong: This is an early-to-mid stage trial with only 22 participants, so results may not apply to all patients. Side effects like infusion reactions are possible.

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

As listed by the trial registrant

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

FABRY DISEASE

Contacts and locations

Locations

Centre Hospitalier Universitaire (CHU) de Bordeaux - Groupe Hospitalier Pellegrin
RECRUITING

Bordeaux, 33076, France
Cincinnati Children's Hospital Medical Center
RECRUITING

Cincinnati, Ohio, 45229, United States
Emory Genetics Clinical Trials Center
RECRUITING

Atlanta, Georgia, 30322, United States
Great Ormond Street Hospital for Children NHS Foundation Trust
NOT_YET_RECRUITING

London, United Kingdom
Haukeland Universitetssjukehus
RECRUITING

Bergen, 5021, Norway
Hopital Arnaud de Villeneuve
RECRUITING

Montpellier, France
Hospital Clinico Universitario De Santiago De Compostela
RECRUITING

Santiago de Compostela, Spain
Lysosomal and Rare Disorders Research and Treatment Center Inc
RECRUITING

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

Phoenix, Arizona, 85016, United States
UK für Kinder- und Jugendheilkunde der PMU Salzburg
RECRUITING

Salzburg, Austria
University of Iowa
RECRUITING

Iowa City, Iowa, 52242, United States
University of Utah
RECRUITING

Salt Lake City, Utah, 84108, United States

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