ClinicalTrials.gov record NCT06328608

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

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

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 2/3A study that tests whether a treatment works while continuing to check safety, in a larger group than a phase 2 study alone. It combines the aims of phase 2 and phase 3 in one protocol.
AgeSee eligibility The registry's age field and its own written criteria give different ages for this study. Check the full criteria below and ask the study team.
SexAll
LocationAustria, France, Norway, Spain, United Kingdom, United States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is an open-label, single-arm Phase 2/3 study of pegunigalsidase alfa (PRX-102) given by intravenous infusion every two weeks to children and adolescents aged 2 to less than 18 years with confirmed Fabry disease. About 20–22 boys and girls will be enrolled in three age cohorts (2–7, 8–12, 13–<18) across a dose-finding stage, a confirmatory stage, and an optional extension. The study aims to learn about safety, the appropriate dose, how the drug behaves in the body, and its effects on Fabry-related signs and symptoms.

What it is studying. The study is testing pegunigalsidase alfa (PRX-102) as an enzyme replacement given every two weeks to learn its safety, the best dose for children, how the body processes the drug (pharmacokinetics), and effects on Fabry disease signs and symptoms such as kidney and heart measures, pain, and gastrointestinal issues.

What the researchers hope to learn. To assess safety and tolerability of PRX-102 in children and adolescents; to identify appropriate pediatric dosing; to measure pharmacokinetics and immunogenicity; and to evaluate effects on kidney and heart measures, pain, gastrointestinal symptoms, and other Fabry-related outcomes.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

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

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Show the detailed description from the registry

This study aims to learn how safe pegunigalsidase alfa (PRX-102 for short) is and how it works at treating Fabry disease in children and adolescents. PRX-102 is an enzyme replacement therapy (ERT), meaning it acts like a natural enzyme. PRX-102 is given through a needle placed in a vein (intravenous infusion) every two weeks. The main questions this study aims to answer are:

  • Which is the safest and most effective dose to be given to children and adolescents.
  • Which effects PRX-102 has on signs and symptoms of Fabry disease (e.g. renal and cardiac function, pain, gastrointestinal symptoms)

20 to 22 boys and girls with Fabry disease between the ages of 2 and 17 will be part of this study. There will be three age cohorts, with children aged 2 to 7 years included (enrolled) in Cohort A, children aged 8 to 12 years in Cohort B, and adolescents aged 13 to less than 18 years in Cohort C. The study is divided into three parts, or "stages":

  • A dose-finding stage (Stage I). In this stage, researchers will determine the dose for children.
  • A confirmatory stage (Stage II). In this part, researchers will learn about the safety and efficacy of PRX-102.
  • and an optional extension stage (Stage III) will continue until the study drug becomes commercially available or the Sponsor chooses to end this study.

PRX-102 will be given at the study visits, which will occur at least every two weeks. Tests for verifying the study drug's safety and efficacy and determining the dose will also be conducted at different time points throughout the study (not all tests will be done at all visits). These tests may include a review of any health problems and medications the participants have had or taken since the last visit; a physical examination; ECG; ultrasound of the heart; questionnaires that evaluate the nature and severity of Fabry disease symptoms, quality of life and pain; a collection of blood and urine samples for standard safety tests, to analyse the severity of Fabry disease and to see how the drug is behaving and how long it remains active in the body (this involves taking multiple blood samples over several days with the first sample taken just before the start of the PRX-102 infusion and the last one taken just before the start of the next PRX-102 at the next visit).

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants receive an intravenous infusion of the study drug PRX‑102 every two weeks and attend study visits at least every two weeks. At visits they will have physical exams, heart tests (ECG and echocardiogram), questionnaires about symptoms, pain and quality of life, and give blood and urine samples for safety, biomarkers and pharmacokinetics. Pharmacokinetic testing involves multiple blood samples over several days (the first before an infusion and the last just before the next infusion). There is an optional extension stage that can continue until the drug becomes commercially available or the Sponsor ends the study.

How the study is structured. Interventional, open-label, single-group Phase 2/3 trial. Participants receive intravenous PRX-102 every two weeks. The trial has a Stage I dose-finding part, a Stage II confirmatory part, and an optional Stage III extension; participants have regular clinic visits with physical exams, heart tests (ECG and echocardiogram), questionnaires, and blood and urine sampling including multiple blood samples for pharmacokinetics.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The listed sites are in Austria, France, Norway, Spain, United Kingdom, United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Fabry disease

Clinical trials47 trials listed for Fabry diseaseEvery trial the registry lists for Fabry disease, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Fabry disease researchLatest monthly update: September 2026. Includes: SGLT2 outcome link. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Jul 29, 2025
Primary completion
October 2028 (estimated)
Study completion
April 2031 (estimated)
Target enrollment
22 participants (estimated)
Last updated on registry
Aug 10, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Chiesi Farmaceutici S.p.A.
Collaborators
  • ICON plc

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (12)

Terms used on this page · explanations by MedicalUpdates.ai

Study Drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
Cohort
A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
Estimated glomerular filtration rate
A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
at screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
childbearing potential
Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
dose-finding
Groups of participants receive increasing doses of a treatment, one group after another, to find a dose that can be given safely.
Sponsor
The organisation or person that initiates the study and has authority over it. The sponsor may be a company, hospital, university, government agency or individual, and is not necessarily the organisation that funds the study.
ECG
A recording of the heart's electrical activity. QTc is one measurement from it; some drugs are avoided if it is prolonged.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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