A Study of PRX-102 for Children and Adolescents with Fabry Disease
This study is looking at the safety and effects of PRX-102 in children and adolescents with Fabry disease. PRX-102 is an enzyme replacement therapy (ERT) given through an IV every two weeks. Researchers want to find the safest and most effective dose for different age groups (2-7, 8-12, and 13-17 years old) and see how it affects symptoms like kidney and heart function, pain, and stomach issues. About 20 to 22 boys and girls with a confirmed diagnosis of Fabry disease and certain characteristic features (like neuropathic pain or cornea verticillata) will participate. The main goal is to see how many side effects, infusion reactions, and injection site reactions occur over 12 months.
- Study design
- This is an interventional study with a planned enrollment of 22 participants. It is divided into three stages: a dose-finding stage, a confirmatory stage, and an optional extension stage.
- What's involved
- Participants will receive PRX-102 through an intravenous infusion every two weeks. The study will involve different stages, including a dose-finding stage and a confirmatory stage.
- Compensation
- Not stated in the trial record.
- Follow-up
- The primary endpoints, such as adverse events and reactions, will be measured at 12 months.
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NCT06328608
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
Recruiting
PHASE2Ages 2–17InterventionalTreatmentChiesi Farmaceutici S.p.A.
~22 participants
Updated 2026-08-10 on ClinicalTrials.gov
What's tested:PRX-102 1 mg/kg every two weeks
At a glance
Recruiting sites
11 of 12 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of Treatment Emergent Adverse Events (TEAEs)
Measured over 12 Months
+41 more outcomes measured
Conditions
Where it's being run
12 sites across 11 statesFrance2
Arizona1
Georgia1
Iowa1
Ohio1
Utah1
Virginia1
Austria1
Who to contact
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Do you actually qualify for this trial?
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Eligibility criteria
Inclusion
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
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.
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
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.
What this trial measures
- Incidence of Treatment Emergent Adverse Events (TEAEs)12 Months
- Incidence of Infusion Related Reactions (IRRs)12 Months
- Incidence of Injection site reactions (ISRs)12 Months
- Change in Tanner stageBaseline and 12 Months
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.
- Change from baseline of 12-lead ECG quantitative parameters: Mean Heart RateBaseline and 12 Months
- Change from baseline of 12-lead ECG quantitative parameters: PR IntervalBaseline and 12 Months
- Change from baseline of 12-lead ECG quantitative parameters: QRS DurationBaseline and 12 Months
- Change from baseline of 12-lead ECG quantitative parameters: QT IntervalBaseline and 12 Months
- Change from baseline of 12-lead ECG quantitative parameters: QTc IntervalBaseline and 12 Months
- Change from baseline of 12-lead ECG quantitative parameters: ST SegmentBaseline and 12 Months
- Incidence of treatment-emergent Anti-Drug Antibodies (ADAs)Baseline and 12 Months
- Incidence of premedication use at each visit and change of infusion premedications from baselineBaseline and 12 Months
- Pharmacokinetics: Time to maximum plasma concentration (tmax)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)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)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-∞)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)Baseline, week 2, week 4, week 12, week 26 and week 52]
- Pharmacokinetics: Area under the curve over a dosing interval (AUCτ)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)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)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)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 eGFRBaseline and 12 Months
- Change in annualized eGFR slopeBaseline and 12 Months
- Change in urine albumin levelsBaseline and 12 Months
- Change in urine protein levelsBaseline and 12 Months
- Change from baseline in LVMi as assessed by echocardiogramBaseline and 12 Months
Echocardiogram parameters include left ventricular mass index (LVMi)
- Change from baseline in LVMi as assessed by echocardiogramBaseline and 12 Months
Echocardiogram parameters include ejection fraction
- Change from baseline in LVMi as assessed by echocardiogramBaseline and 12 Months
Echocardiogram parameters include, fractional shortening
- Change from baseline in LVMi as assessed by echocardiogramBaseline and 12 Months
Echocardiogram parameters include left ventricular mass
- Change from baseline in LVMi as assessed by echocardiogramBaseline and 12 Months
Echocardiogram parameters include valve abnormalities and thickness.
- Incidence of any cardiac arrythmias as assessed by Holter ECGBaseline and 12 Months
- Change in plasma levels of cardiac biomarkersBaseline and 12 Months
High-sensitivity cardiac troponin T (hs-cTnT) and N- terminal pro brain natriuretic peptide (NT-Pro BNP) will be assessed.
- Change in plasma level of Gb3 concentration (nM)Baseline and 12 Months
- Change in plasma level of lyso-Gb3 (nM)Baseline and 12 Months
- Change in urine level of lyso-Gb3 (nM)Baseline and 12 Months
- Incidence of change from baseline in the number of different pain medicationsBaseline and 12 Months
- Incidence of Fabry Clinical Events12 Months
FCEs are classified into four categories: renal, cardiac, cerebrovascular and death due to non-cardiac reasons
- Change from baseline of Mainz Severity Score Index (MSSI) scoresBaseline and 12 Months
Domains (general, neurological, cardiovascular, renal dysfunction)
- Change from baseline of PedsQL-GI (or GSRS for subjects who reaches 18 yrs of age) scoresBaseline and 12 Months
- Change from baseline of FPHPQ scoresBaseline and 12 Months
- Change from baseline of PedsQL-PPQ (or BPI-SF for subjects who reaches 18 yrs of age) scoresBaseline and 12 Months
- Change from baseline of EQ-5D-Y (or EQ-5D-5L for subjects who reaches 18 yrs of age) scoresBaseline and 12 Months