RecruitingPhase 2Phase 3NCT06328608

A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease

Multi-centre, Open-label Trial to Assess the saFety, Pharmacodynamics, Efficacy and Pharmacokinetics of pegunigaLsidase Alfa in Patients From 2 Years to Less Than 18 Years of Age With Confirmed FabrY Disease

Sponsor

Chiesi Farmaceutici S.p.A.

Enrollment

22 participants

Start Date

Jul 29, 2025

Study Type

INTERVENTIONAL

Conditions

Fabry Disease

Summary

A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.

Eligibility

Min Age: 2 YearsMax Age: 17 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called PRX-102 1 mg/kg every two weeks for people with fabry disease. The study is currently recruiting participants at 12 locations. People eligible for this study include aged 2 Years to 17 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGPRX-102 1 mg/kg every two weeks

Drug: PRX-102 1 mg/kg every two weeks

Locations(12)

Phoenix Children's

Phoenix, Arizona, United States

Emory Genetics Clinical Trials Center

Atlanta, Georgia, United States

University of Iowa

Iowa City, Iowa, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

University of Utah

Salt Lake City, Utah, United States

Lysosomal and Rare Disorders Research and Treatment Center Inc

Fairfax, Virginia, United States

UK für Kinder- und Jugendheilkunde der PMU Salzburg

Salzburg, Austria

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

Bordeaux, France

Hopital Arnaud de Villeneuve

Montpellier, France

Haukeland Universitetssjukehus

Bergen, Norway

Hospital Clinico Universitario De Santiago De Compostela

Santiago de Compostela, Spain

Great Ormond Street Hospital for Children NHS Foundation Trust

London, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT06328608

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