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

Recruiting now · Phase 2/Phase 3

Conditions studied: Fabry Disease

In brief

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

Key facts

Study ID

NCT06328608

Run by

Chiesi Farmaceutici S.p.A.

People needed

22

Starts

2025-07-29

Expected to finish

2031-04-01

Last updated by the study team

2026-03-19

Who can join

Age: 2 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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 <18 years (Cohort C).
• Confirmed diagnosis of Fabry disease
• Presence of at least one of the following characteristic features of Fabry disease: neuropathic pain, cornea verticillata, and/or clustered angiokeratoma.
• History of Fabry pain: Fabry crises OR chronic pain.
• Clinical condition that, in the investigator's opinion, requires ERT treatment.

You may not qualify if…

• All Subjects:
• Estimated glomerular filtration rate (eGFR) at screening < 80 mL/min/1.73 m2.
• History of type I hypersensitivity reactions (anaphylactic or anaphylactoid life-threatening reaction) to other ERT treatment for Fabry disease or any component of the study drug.
• Initiation of treatment with an angiotensin-converting enzyme inhibitor (ACEi) or angiotensin II receptor blocker (ARB) or a dose change in ongoing treatment in the four weeks before screening.
• Urine protein to creatinine ratio (UPCR) > 0.5 g/g (0.5 mg/mg or 500 mg/g) if not treated with an ACE inhibitor or ARB.
• Currently taking another investigational drug for any condition.
• History of acute kidney injury in the 12 months before screening, including specific kidney diseases (e.g., acute interstitial nephritis, acute glomerular and vasculitic renal diseases); non-specific conditions (e.g., ischaemia, toxic injury); or extrarenal pathology (e.g., prerenal azotaemia, acute postrenal obstructive nephropathy).
• History of renal dialysis or kidney transplantation.
• History of or current malignancy requiring treatment.
• Severe cardiomyopathy or significant unstable cardiac disease within six months before screening.
• A positive test for Severe Acute Respiratory Syndrome-Coronavirus 2 (SARS-CoV-2) within three months before screening.
• Presence of any medical, emotional, behavioural, or psychological condition that, in the Investigator's judgement, could interfere with the subject's compliance with the requirements of the study.
• Additional Exclusion Criteria for Subjects Enrolled in Stage I:
• Female
• Non-classic form of Fabry disease
• Receipt of treatment for Fabry disease within six months before screening
• Positive for anti-PRX-102 antibodies at screening
• Additional Exclusion Criteria for Subjects in Stage II (i.e., non-treatment naïve males or females):
• Unwilling to discontinue current ERT treatment for Fabry disease before baseline.
• Females: Pregnant or lactating, or of childbearing potential with a fertile male partner and unwilling to use a highly reliable method of contraception from the informed consent signature until 30 days after the last infusion.

Where it is running

• Phoenix Children's — Phoenix, Arizona, United States (enrolling)
• Emory Genetics Clinical Trials Center — Atlanta, Georgia, United States (enrolling)
• University of Iowa — Iowa City, Iowa, United States (enrolling)
• Hopital Arnaud de Villeneuve — Montpellier, France (enrolling)
• Haukeland Universitetssjukehus — Bergen, Norway (enrolling)
• Hospital Clinico Universitario De Santiago De Compostela — Santiago de Compostela, Spain (enrolling)
• University of Utah — Salt Lake City, Utah, United States (enrolling)
• UK für Kinder- und Jugendheilkunde der PMU Salzburg — Salzburg, Austria (enrolling)
• Centre Hospitalier Universitaire (CHU) de Bordeaux - Groupe Hospitalier Pellegrin — Bordeaux, France (enrolling)
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Great Ormond Street Hospital for Children NHS Foundation Trust — London, United Kingdom
• Lysosomal and Rare Disorders Research and Treatment Center Inc — Fairfax, Virginia, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.