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

NCT06328608 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: CLI-06657AA1-012022-503128-29
• Study Type: interventional
• Target: 22
• Locations: 6 countries
• Sites: 12

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

Outcome Measures

Primary Outcomes (42)

• 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

  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.

  Baseline and 12 Months

• 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

  Echocardiogram parameters include left ventricular mass index (LVMi)

  Baseline and 12 Months

• Change from baseline in LVMi as assessed by echocardiogram

  Echocardiogram parameters include ejection fraction

  Baseline and 12 Months

• Change from baseline in LVMi as assessed by echocardiogram

  Echocardiogram parameters include, fractional shortening

  Baseline and 12 Months

• Change from baseline in LVMi as assessed by echocardiogram

  Echocardiogram parameters include left ventricular mass

  Baseline and 12 Months

• Change from baseline in LVMi as assessed by echocardiogram

  Echocardiogram parameters include valve abnormalities and thickness.

  Baseline and 12 Months

• Incidence of any cardiac arrythmias as assessed by Holter ECG

  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.

  Baseline and 12 Months

• 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

  FCEs are classified into four categories: renal, cardiac, cerebrovascular and death due to non-cardiac reasons

  12 Months

• Change from baseline of Mainz Severity Score Index (MSSI) scores

  Domains (general, neurological, cardiovascular, renal dysfunction)

  Baseline and 12 Months

• 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

Study Arms (1)

Single Arm - Pegunigalsidase alfa (PRX-102)

EXPERIMENTAL

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

Interventions

PRX-102 1 mg/kg every two weeks DRUG

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

Also known as: pegunigalsidase alfa, Recombinant human alpha galactosidase-A

Single Arm - Pegunigalsidase alfa (PRX-102)

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• Female
• Non-classic form of Fabry disease
• Receipt of treatment for Fabry disease within six months before screening

Sponsors & Collaborators

• Chiesi Farmaceutici S.p.A.: lead
• ICON plc: collaborator

Study Sites (12)

Phoenix Children's

Phoenix, Arizona, 85016, United States

RECRUITING

Emory Genetics Clinical Trials Center

Atlanta, Georgia, 30322, United States

RECRUITING

University of Iowa

Iowa City, Iowa, 52242, United States

RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

NOT YET RECRUITING

University of Utah

Salt Lake City, Utah, 84108, United States

RECRUITING

Lysosomal and Rare Disorders Research and Treatment Center Inc

Fairfax, Virginia, 22030, United States

NOT YET RECRUITING

UK für Kinder- und Jugendheilkunde der PMU Salzburg

Salzburg, Austria

RECRUITING

Centre Hospitalier Universitaire (CHU) de Bordeaux - Groupe Hospitalier Pellegrin

Bordeaux, 33076, France

RECRUITING

Hopital Arnaud de Villeneuve

Montpellier, France

RECRUITING

Haukeland Universitetssjukehus

Bergen, 5021, 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

MeSH Terms

Conditions

Fabry Disease

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Central Study Contacts

Chiesi Clinical Trial

CONTACT

+3905212791; clinicaltrials_info@chiesi.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 10, 2023
• First Posted: March 25, 2024
• Study Start: July 29, 2025
• Primary Completion (Estimated): October 1, 2028
• Study Completion (Estimated): April 1, 2031
• Last Updated: March 19, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1); NO (1); US (6); FR (2); AU (1); ES (1)