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

NCT06328608 · tracked via the Priya Life Science Norway tracker

Sponsor

Chiesi Farmaceutici S.p.A

Phase

Phase 2/3

Started

2025-07-29

Last updated

2026-08-10

Condition(s) studied

Fabry Disease

Investigational drug(s) / intervention(s)

PRX-102 1 mg/kg every two weeks

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

Study summary

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

Eligibility

Sex

ALL

Min age

2 Years

Max age

17 Years

Healthy volunteers

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.

Primary outcome measure(s)

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 stage — Baseline 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 Rate — Baseline and 12 Months
Change from baseline of 12-lead ECG quantitative parameters: PR Interval — Baseline and 12 Months
Change from baseline of 12-lead ECG quantitative parameters: QRS Duration — Baseline and 12 Months
Change from baseline of 12-lead ECG quantitative parameters: QT Interval — Baseline and 12 Months
Change from baseline of 12-lead ECG quantitative parameters: QTc Interval — Baseline and 12 Months
Change from baseline of 12-lead ECG quantitative parameters: ST Segment — Baseline 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 baseline — Baseline 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 eGFR — Baseline and 12 Months
Change in annualized eGFR slope — Baseline and 12 Months
Change in urine albumin levels — Baseline and 12 Months
Change in urine protein levels — Baseline and 12 Months
Change from baseline in LVMi as assessed by echocardiogram — Baseline and 12 Months
Echocardiogram parameters include left ventricular mass index (LVMi)
Change from baseline in LVMi as assessed by echocardiogram — Baseline and 12 Months
Echocardiogram parameters include ejection fraction
Change from baseline in LVMi as assessed by echocardiogram — Baseline and 12 Months
Echocardiogram parameters include, fractional shortening
Change from baseline in LVMi as assessed by echocardiogram — Baseline and 12 Months
Echocardiogram parameters include left ventricular mass
Change from baseline in LVMi as assessed by echocardiogram — Baseline and 12 Months
Echocardiogram parameters include valve abnormalities and thickness.
Incidence of any cardiac arrythmias as assessed by Holter ECG — Baseline and 12 Months
Change in plasma levels of cardiac biomarkers — Baseline 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 medications — Baseline and 12 Months
Incidence of Fabry Clinical Events — 12 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) scores — Baseline 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) scores — Baseline and 12 Months
Change from baseline of FPHPQ scores — Baseline and 12 Months
Change from baseline of PedsQL-PPQ (or BPI-SF for subjects who reaches 18 yrs of age) scores — Baseline and 12 Months
Change from baseline of EQ-5D-Y (or EQ-5D-5L for subjects who reaches 18 yrs of age) scores — Baseline and 12 Months

Trial sites (12)

Facility	City	Region	Status
Phoenix Children's	Phoenix	Arizona	Recruiting
Emory Genetics Clinical Trials Center	Atlanta	Georgia	Recruiting
University of Iowa	Iowa City	Iowa	Recruiting
Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio	Recruiting
University of Utah	Salt Lake City	Utah	Recruiting
Lysosomal and Rare Disorders Research and Treatment Center Inc	Fairfax	Virginia	Recruiting
UK für Kinder- und Jugendheilkunde der PMU Salzburg	Salzburg	Austria	Recruiting
Centre Hospitalier Universitaire (CHU) de Bordeaux - Groupe Hospitalier Pellegrin	Bordeaux	France	Recruiting
Hopital Arnaud de Villeneuve	Montpellier	France	Recruiting
Haukeland Universitetssjukehus	Bergen	Norway	Recruiting
Hospital Clinico Universitario De Santiago De Compostela	Santiago de Compostela	Spain	Recruiting
Great Ormond Street Hospital for Children NHS Foundation Trust	London	United Kingdom	Not Yet Recruiting

Other trials for the same condition

A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease Active, not recruiting

A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease Active, not recruiting

Fabry Disease Registry & Pregnancy Sub-registry Recruiting

A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease Active, not recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06328608 on ClinicalTrials.gov ↗ ← All trials in Norway