Recruiting
Phase 2
Phase 3

PRX-102

Sponsor:

Chiesi Farmaceutici S.p.A.

Code:

NCT06328608

Conditions

Fabry Disease

Eligibility Criteria

Sex: All

Age: 2 - 17

Healthy Volunteers: Not accepted

Interventions

PRX-102 1 mg/kg every two weeks

Study Details

Brief summary:

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

Conditions

Fabry Disease

Study ID

NCT06328608

Start date

Jul 29, 2025

Status verified date

Mar, 2026

Completion date

Apr, 2031

Anticipated

Primary completion date

Oct, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2 - 17

Healthy Volunteers: Not accepted

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 Design

Enrollment

22 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Single Arm - Pegunigalsidase alfa (PRX-102)

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.

Interventions

PRX-102 1 mg/kg every two weeks

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

Primary outcome measure

  • 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 [ 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 [ Time Frame: Baseline and 12 Months ]
  • Change from baseline in LVMi as assessed by echocardiogram [ Time Frame: Baseline and 12 Months ]
  • Change from baseline in LVMi as assessed by echocardiogram [ Time Frame: Baseline and 12 Months ]
  • Change from baseline in LVMi as assessed by echocardiogram [ Time Frame: Baseline and 12 Months ]
  • Change from baseline in LVMi as assessed by echocardiogram [ 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 [ 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 [ Time Frame: 12 Months ]
  • Change from baseline of Mainz Severity Score Index (MSSI) scores [ 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 ]

Central Contacts and Locations

Central contacts

Chiesi Clinical Trial

+3905212791clinicaltrials_info@chiesi.com

Locations

Phoenix Children's

Recruiting

Phoenix, Arizona, United States, 85016

Contacts

Jasmine Knoll

602-933-4363jknoll@phoenixchildrens.com

Emory Genetics Clinical Trials Center

Recruiting

Atlanta, Georgia, United States, 30322

Contacts

William Wilcox

404-727-2931william.wilcox@emory.edu

University of Iowa

Recruiting

Iowa City, Iowa, United States, 52242

Contacts

John Bernat

319-356-2675john-bernat@uiowa.edu

Cincinnati Children's Hospital Medical Center

Recruiting

Cincinnati, Ohio, United States, 45229

Contacts

Robert Hopkin

513-636-4760rob.hopkin@cchmc.org

University of Utah

Recruiting

Salt Lake City, Utah, United States, 84108

Contacts

Julie M Porter

801-587-3605Julie.Porter@hsc.utah.edu

Lysosomal and Rare Disorders Research and Treatment Center Inc

Recruiting

Fairfax, Virginia, United States, 22030

Contacts

Ozlem Goker-Alpan

240-643-6003ogoker-alpan@ldrtc.org

More Information

Sponsor

Chiesi Farmaceutici S.p.A.

Last update posted

Aug 10, 2026

Last verified

Mar, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Chiesi Farmaceutici S.p.A. on 2026-08-10.