Taking people in United StatesPhase 2/3
Tests PRX-102 in children and teens with Fabry disease
Tests PRX-102 in children and teens with Fabry disease.
Key facts
- Who runs it
- Chiesi Farmaceutici S.p.A.
- Where
- Atlanta, Cincinnati, Fairfax, Iowa City, Phoenix, and Salt Lake City, United StatesSee the 6 sitesAlso in 5 other countries, 6 sites
- Trial code
- Registry title
- A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
- Status
- Recruiting
- Phase
- Phase 2/3
- People taking part
- 22 people, planned
- Started
- 29 Jul 2025
- Expected to end
- Apr 2031
- Type of study
- Interventional
- Treatments tested
- PRX-102 1 mg/kg every two weeks
- Record updated
- 10 August 2026
Who can take part
AI summary
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for boys and girls aged 2 to under 18 with confirmed Fabry disease who need enzyme replacement therapy (ERT).
- Age
- 2 to under 18 years (Cohort A: 2 to 7, Cohort B: 8 to 12, Cohort C: 13 to under 18).
- Sex
- Boys and girls can join. Girls cannot join Stage I.
- Disease stage
- Confirmed Fabry disease with at least one of: nerve pain, cornea verticillata, or clustered angiokeratoma. A history of Fabry pain (Fabry crises or chronic pain). The investigator must judge that ERT treatment is needed. Stage I excludes the non-classic form.
- Earlier treatment
- Stage I: no treatment for Fabry disease in the six months before screening. Stage II: people already on ERT must be willing to stop it before baseline.
- Biomarker or test result
- Stage I: no anti-PRX-102 antibodies at screening.
Main reasons for not taking part
- Kidney function (eGFR) below 80 mL/min/1.73 m2 at screening
- Past severe allergic reaction to other ERT for Fabry disease or to the study drug
- History of dialysis or kidney transplant
- Current or past cancer needing treatment
- Severe heart disease
The registry criteria, word for word
Show all 24 criteriaShow less
Who can take part
- 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
Who cannot take part
- 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
- 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)
- 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
- 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):
- 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
- History of renal dialysis or kidney transplantation
- History of or current malignancy requiring treatment
- 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:
- Non-classic form of Fabry disease
- Unwilling to discontinue current ERT treatment for Fabry disease before baseline
- Estimated glomerular filtration rate (eGFR) at screening < 80 mL/min/1.73 m2
- 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
- All Subjects:
- Female
- 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
Check if you might fit
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 2 and 17 years old?
Where in United States
Also in 5 other countries, 6 sites
- Emory Genetics Clinical Trials CenterAtlanta, United StatesContact: William WilcoxTaking people
- Cincinnati Children's Hospital Medical CenterCincinnati, United StatesContact: Robert HopkinTaking people
- Lysosomal and Rare Disorders Research and Treatment Center, IncFairfax, United StatesContact: Ozlem Goker-AlpanTaking people
- University of IowaIowa City, United StatesContact: John BernatTaking people
- Phoenix Children'sPhoenix, United StatesContact: Jasmine KnollTaking people
- University of UtahSalt Lake City, United StatesContact: Julie M PorterTaking people
About the trial
What the trial wants to find out
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.
The group in the trial
- Single Arm - Pegunigalsidase alfa (PRX-102)Treatment being tested
For Cohort C, PXR-102 administered every two weeks at 1.0 mg/kg is believed to be the minimum effective dose. For Cohorts A and B, the starting dose will be 1.0 mg/kg every two weeks but it may be adjusted on the outcomes of Stage I, with the support of the Data Safety Monitoring Board.
Treatment: PRX-102 1 mg/kg every two weeks
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorData and sources
- Registry record
- Listed on ClinicalTrials.gov and on the EU's official list of trials (CTIS).
- Official title
- 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
- Plain-language title
- Tests PRX-102 in children and teens with Fabry diseaseGenerated from the registry record. The registry title is the official one.
- AI summary
- From the trial's eligibility criteria. Read by machine on 12 November 2026.
- Fit questions
- Built from NCT06328608, who can take part
- Record updated
- 10 August 2026