Actively Recruiting
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
Led by Chiesi Farmaceutici S.p.A. · Updated on 2026-03-19
22
Participants Needed
12
Research Sites
130 weeks
Total Duration
On this page
Sponsors
C
Chiesi Farmaceutici S.p.A.
Lead Sponsor
I
ICON plc
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effects of pegunigalsidase alfa (PRX-102), an enzyme replacement therapy, in children and adolescents with Fabry disease. This study includes boys and girls aged 2 to less than 18 years who have confirmed Fabry disease and symptoms such as neuropathic pain, cornea verticillata, or clustered angiokeratoma. The aim is to find the safest and most effective dose and understand how the drug affects symptoms like kidney and heart function, pain, and gastrointestinal problems. PRX-102 is given by intravenous infusion every two weeks. The study is divided into three parts: Stage I to find the correct dose, Stage II to confirm safety and effectiveness, and an optional Stage III extension until the drug becomes commercially available or the study ends. The drug dosing starts at 1.0 mg/kg every two weeks, with adjustments possible for younger children. Participants are grouped by age into three cohorts: 2 to 7 years, 8 to 12 years, and 13 to less than 18 years. Participants will have visits at least every two weeks for drug administration and various tests. These include physical exams, heart monitoring with ECG and ultrasound, symptom and quality of life questionnaires, and blood and urine tests to check safety, disease severity, and drug levels. Blood samples are collected multiple times over dosing intervals to study how the drug behaves in the body. Researchers will measure outcomes like adverse reactions, heart and kidney function, pain medication use, and disease severity over 12 months.
CONDITIONS
Brief Title
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants with the provision of informed consent from their legal guardians
- Boys and girls aged 2 to 7 years (Cohort A), 8 to 12 years (Cohort B), or 13 to less than 18 years (Cohort C)
- Confirmed diagnosis of Fabry disease
- Presence of at least one characteristic feature of Fabry disease: neuropathic pain, cornea verticillata, or clustered angiokeratoma
- History of Fabry pain: Fabry crises or chronic pain
- Clinical condition requiring enzyme replacement therapy as judged by the investigator
You will not qualify if you...
- Estimated glomerular filtration rate (eGFR) less than 80 mL/min/1.73 m2 at screening
- History of life-threatening allergic reactions to other enzyme replacement therapies or study drug components
- Starting or changing dose of ACE inhibitors or angiotensin II receptor blockers within 4 weeks before screening
- Urine protein to creatinine ratio greater than 0.5 g/g if not treated with ACE inhibitors or ARBs
- Currently taking another investigational drug
- History of acute kidney injury within 12 months before screening
- History of kidney dialysis or transplantation
- History of or current cancer requiring treatment
- Severe or unstable heart disease within 6 months before screening
- Positive test for SARS-CoV-2 within 3 months before screening
- Any medical, emotional, behavioral, or psychological condition interfering with study compliance
- Female subjects in Stage I
- Non-classic form of Fabry disease in Stage I
- Treatment for Fabry disease within 6 months before screening in Stage I
- Positive for anti-PRX-102 antibodies at screening in Stage I
- Unwillingness to stop current enzyme replacement therapy before baseline in Stage II
- Females who are pregnant, breastfeeding, or of childbearing potential without reliable contraception from consent until 30 days after last infusion in Stage II
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to several months depending on study stage
Participants receive the study drug PRX-102 through an intravenous infusion every two weeks to assess safety and effectiveness in treating Fabry disease.
Biweekly visits for infusions and tests
Duration - Variable, until drug availability or study end
Participants may continue receiving PRX-102 until the drug becomes commercially available or the study ends.
Ongoing visits every two weeks for infusions and assessments
Trial Site Locations
Total: 12 locations
1
Phoenix Children's
Phoenix, Arizona, United States, 85016
Actively Recruiting
2
Emory Genetics Clinical Trials Center
Atlanta, Georgia, United States, 30322
Actively Recruiting
3
University of Iowa
Iowa City, Iowa, United States, 52242
Actively Recruiting
4
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Not Yet Recruiting
5
University of Utah
Salt Lake City, Utah, United States, 84108
Actively Recruiting
6
Lysosomal and Rare Disorders Research and Treatment Center Inc
Fairfax, Virginia, United States, 22030
Not Yet Recruiting
7
UK für Kinder- und Jugendheilkunde der PMU Salzburg
Salzburg, Austria
Actively Recruiting
8
Centre Hospitalier Universitaire (CHU) de Bordeaux - Groupe Hospitalier Pellegrin
Bordeaux, France, 33076
Actively Recruiting
9
Hopital Arnaud de Villeneuve
Montpellier, France
Actively Recruiting
10
Haukeland Universitetssjukehus
Bergen, Norway, 5021
Actively Recruiting
11
Hospital Clinico Universitario De Santiago De Compostela
Santiago de Compostela, Spain
Actively Recruiting
12
Great Ormond Street Hospital for Children NHS Foundation Trust
London, United Kingdom
Not Yet Recruiting
Research Team
C
Chiesi Clinical Trial
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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