Actively Recruiting
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
Led by Chiesi Farmaceutici S.p.A. · Updated on 2026-03-19
22
Participants Needed
12
Research Sites
296 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
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.
CONDITIONS
Official 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...
- 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, including Fabry crises or chronic pain
- Clinical condition requiring enzyme replacement therapy as judged by the investigator
- Provision of informed consent from legal guardian
You will not qualify if you...
- Estimated glomerular filtration rate (eGFR) below 80 mL/min/1.73 m2 at screening
- History of life-threatening hypersensitivity reactions to other enzyme replacement therapies or any component of the study drug
- Starting or changing dose of ACE inhibitor or angiotensin II receptor blocker within four weeks before screening
- Urine protein to creatinine ratio (UPCR) over 0.5 g/g if not treated with ACE inhibitor or ARB
- Currently taking another investigational drug
- History of acute kidney injury within 12 months before screening
- History of kidney dialysis or transplantation
- History or current malignancy requiring treatment
- Severe or unstable heart disease within six months before screening
- Positive test for COVID-19 within three months before screening
- Any medical, emotional, behavioral, or psychological condition interfering with study compliance
- (Stage I only) Female participants
- (Stage I only) Non-classic form of Fabry disease
- (Stage I only) Treatment for Fabry disease within six months before screening
- (Stage I only) Positive for anti-PRX-102 antibodies at screening
- (Stage II only) Unwillingness to stop current enzyme replacement therapy before baseline
- (Stage II only) Females who are pregnant, breastfeeding, or of childbearing potential unwilling to use reliable contraception until 30 days after last infusion
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
CONTACT
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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