A Phase 2/3 interventional study of PRX-102 1 mg/kg every two weeks in Fabry Disease, sponsored by Chiesi Farmaceutici S.p.A.. Recruiting at 12 sites in 6 countries. Open to participants aged 2 Years to 17 Years. Per ClinicalTrials.gov, last updated 2026-08-10.
Sponsored by Chiesi Farmaceutici S.p.A. · Phase 2/3, Interventional, and Treatment
Among 10 studies testing pegunigalsidase alfa in Fabry Disease — 2 open to participants now, 2 under way, 2 of them phase 3. pegunigalsidase alfa · Fabry Disease.
Among the 10 studies testing pegunigalsidase alfa in Fabry Disease, this one is the 5th largest by planned enrollment, is the only one that started in 2025, is one of 2 in phase 2/3, and was the 10th registered.
244 studies on the registry are indexed under Fabry Disease; 55 are open to participants now.
This study's planned enrollment of 22 is close to the median of 22 across 108 interventional studies indexed under Fabry Disease.
Browse Fabry Disease studies →2 of the 10 studies testing pegunigalsidase alfa in Fabry Disease are open now, in 7 countries. Each count lists the studies.
pegunigalsidase alfa: first FDA approval 2023 (ELFABRIO), first EMA approval 2023. What it is approved for, and how studies give it, is on its page →
Of the 45 Fabry Disease studies recruiting now, 32 (71%) set no upper age limit and 11 (24%) stop at 75 or younger; 20 take children under 18; 7 take healthy volunteers.
This study: 2 Years to 17 Years · all sexes · no healthy volunteers.
The full table on the hub →10 studies; the count open now beside each. This study's row is marked.
First in 2012; this study’s year is marked. 1 started in 2025.
Median enrollment 22 across the 10 interventional studies. Of the 7 that completed or were terminated, 6 (86%) have results posted.
10 of 10 have an industry lead sponsor. The 2 most frequent:
Chiesi Farmaceutici S.p.A. is the lead sponsor of 185 studies on the registry; 22 are open to participants now.
Of its 18 completed or terminated interventional studies of FDA-regulated products, 11 (61%) have results posted.
Chiesi Farmaceutici S.p.A. has 8 studies filed under Fabry Disease — fewer than ten, so there is no comparison to draw.
1 of the studies testing pegunigalsidase alfa in Fabry Disease that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05710692Phase 3Chiesi Farmaceutici S.p.A.Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease | 2027-10 |
Among the 10 studies testing pegunigalsidase alfa in Fabry Disease, from their registry updates:
Nothing recorded among them since 2026-09-25.
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.
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 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":
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).
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
All Subjects:
Additional Exclusion Criteria for Subjects Enrolled in Stage I:
Additional Exclusion Criteria for Subjects in Stage II (i.e., non-treatment naïve males or females):
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Drug: PRX-102 1 mg/kg every two weeks
Drug page: Pegunigalsidase alfa
Drug: PRX-102 1 mg/kg every two weeks
Also known as: pegunigalsidase alfa, Recombinant human alpha galactosidase-A
Incidence of Treatment Emergent Adverse Events (TEAEs)
Time frame: 12 Months
Incidence of Infusion Related Reactions (IRRs)
Time frame: 12 Months
Incidence of Injection site reactions (ISRs)
Time frame: 12 Months
Change in Tanner stage
Tanner Staging of Sexual Development will be used to assess sexual development (i.e. breast development (B1 to B5) and pubic hair development (Ph-1 to Ph-5) in females and pubic hair and genetical development (G1-G5) in males.
Time frame: Baseline and 12 Months
Change from baseline of 12-lead ECG quantitative parameters: Mean Heart Rate
Time frame: Baseline and 12 Months
Change from baseline of 12-lead ECG quantitative parameters: PR Interval
Time frame: Baseline and 12 Months
Change from baseline of 12-lead ECG quantitative parameters: QRS Duration
Time frame: Baseline and 12 Months
Change from baseline of 12-lead ECG quantitative parameters: QT Interval
Time frame: Baseline and 12 Months
Change from baseline of 12-lead ECG quantitative parameters: QTc Interval
Time frame: Baseline and 12 Months
Change from baseline of 12-lead ECG quantitative parameters: ST Segment
Time frame: Baseline and 12 Months
Incidence of treatment-emergent Anti-Drug Antibodies (ADAs)
Time frame: Baseline and 12 Months
Incidence of premedication use at each visit and change of infusion premedications from baseline
Time frame: Baseline and 12 Months
Pharmacokinetics: Time to maximum plasma concentration (tmax)
Time frame: Stage I - Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Pharmacokinetic : Area under the plasma concentration-time curve from time 0 to time t (AUC0 t)
Time frame: Stage I - Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Pharmacokinetics: Area under the curve from time 0 to 2 weeks (AUC0-2wk)
Time frame: Stage I - Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Pharmacokinetics: Area under the curve from time 0 to infinity (AUC0-∞)
Time frame: Stage I - Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Pharmacokinetics: Terminal half-life (t1/2)
Time frame: Baseline, week 2, week 4, week 12, week 26 and week 52]
Pharmacokinetics: Area under the curve over a dosing interval (AUCτ)
Time frame: Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Pharmacokinetics: Observed drug concentration at the end of the dosing interval (Cτ)
Time frame: Stage I - Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Pharmacokinetics: Clearance (Cl)
Time frame: Stage I - Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Pharmacokinetics: Volume of distribution (Vz)
Time frame: Stage I - Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]
Change in eGFR
Time frame: Baseline and 12 Months
Change in annualized eGFR slope
Time frame: Baseline and 12 Months
Change in urine albumin levels
Time frame: Baseline and 12 Months
Change in urine protein levels
Time frame: Baseline and 12 Months
Change from baseline in LVMi as assessed by echocardiogram
Echocardiogram parameters include left ventricular mass index (LVMi)
Time frame: Baseline and 12 Months
Change from baseline in LVMi as assessed by echocardiogram
Echocardiogram parameters include ejection fraction
Time frame: Baseline and 12 Months
Change from baseline in LVMi as assessed by echocardiogram
Echocardiogram parameters include, fractional shortening
Time frame: Baseline and 12 Months
Change from baseline in LVMi as assessed by echocardiogram
Echocardiogram parameters include left ventricular mass
Time frame: Baseline and 12 Months
Change from baseline in LVMi as assessed by echocardiogram
Echocardiogram parameters include valve abnormalities and thickness.
Time frame: Baseline and 12 Months
Incidence of any cardiac arrythmias as assessed by Holter ECG
Time frame: Baseline and 12 Months
Change in plasma levels of cardiac biomarkers
High-sensitivity cardiac troponin T (hs-cTnT) and N- terminal pro brain natriuretic peptide (NT-Pro BNP) will be assessed.
Time frame: Baseline and 12 Months
Change in plasma level of Gb3 concentration (nM)
Time frame: Baseline and 12 Months
Change in plasma level of lyso-Gb3 (nM)
Time frame: Baseline and 12 Months
Change in urine level of lyso-Gb3 (nM)
Time frame: Baseline and 12 Months
Incidence of change from baseline in the number of different pain medications
Time frame: Baseline and 12 Months
Incidence of Fabry Clinical Events
FCEs are classified into four categories: renal, cardiac, cerebrovascular and death due to non-cardiac reasons
Time frame: 12 Months
Change from baseline of Mainz Severity Score Index (MSSI) scores
Domains (general, neurological, cardiovascular, renal dysfunction)
Time frame: Baseline and 12 Months
Change from baseline of PedsQL-GI (or GSRS for subjects who reaches 18 yrs of age) scores
Time frame: Baseline and 12 Months
Change from baseline of FPHPQ scores
Time frame: Baseline and 12 Months
Change from baseline of PedsQL-PPQ (or BPI-SF for subjects who reaches 18 yrs of age) scores
Time frame: Baseline and 12 Months
Change from baseline of EQ-5D-Y (or EQ-5D-5L for subjects who reaches 18 yrs of age) scores
Time frame: Baseline and 12 Months
Plan to share: No
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Studies of the drug tested here: Pegunigalsidase alfa