Evaluating the safety and effects of PRX-102 in children and adolescents with Fabry disease

Multi-centre, Open-label Trial to Assess the saFety, Pharmacodynamics, Efficacy and Pharmacokinetics of pegunigaLsidase Alfa in Patients From 2 Years to Less Than 18 Years of Age With Confirmed FabrY Disease

This study is testing a new treatment called PRX-102 to see if it can safely help children and teenagers with Fabry disease feel better and improve their symptoms.

Quick facts

• Phase: Phase2; Phase3
• Study type: Interventional
• Enrollment: 22 (estimated)
• Ages: 2 Years to 17 Years
• Sex: All
• Sponsor: Chiesi Farmaceutici S.p.A. (Industry-sponsored)
• Locations: 12 sites (Phoenix, Arizona and 11 other locations)
• Trial ID: NCT06328608 on ClinicalTrials.gov

What this trial studies

This study investigates the safety and efficacy of the enzyme replacement therapy PRX-102 in treating Fabry disease in children and adolescents aged 2 to 17. Participants will receive intravenous infusions of PRX-102 every two weeks, with the study structured into three stages: a dose-finding stage, a confirmatory stage, and an optional extension stage. The study aims to determine the optimal dose and assess the drug's impact on various symptoms associated with Fabry disease, including renal and cardiac function, pain, and gastrointestinal symptoms.

Who should consider this trial

Good fit: Ideal candidates include boys and girls aged 2 to 17 with a confirmed diagnosis of Fabry disease and specific symptoms related to the condition.

Not a fit: Patients with severe renal impairment or a history of severe allergic reactions to enzyme replacement therapies may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new effective treatment option for managing Fabry disease in pediatric patients.

How similar studies have performed: Other studies on enzyme replacement therapies for Fabry disease have shown promise, indicating that this approach is not entirely novel but continues to be explored for improved outcomes.

Eligibility criteria

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.

Where this trial is running

Phoenix, Arizona and 11 other locations

• Phoenix Children's — Phoenix, Arizona, United States (Recruiting)
• Emory Genetics Clinical Trials Center — Atlanta, Georgia, United States (Recruiting)
• University of Iowa — Iowa City, Iowa, United States (Recruiting)
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (Not_yet_recruiting)
• University of Utah — Salt Lake City, Utah, United States (Recruiting)
• Lysosomal and Rare Disorders Research and Treatment Center Inc — Fairfax, Virginia, 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, 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)

Study contacts

• Study coordinator: Chiesi Clinical Trial

Conditions Fabry Disease