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

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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 Recruiting
• University of Utah
  Salt Lake City, Utah, United States Recruiting
• Lysosomal and Rare Disorders Research and Treatment Center Inc
  Fairfax, Virginia, United States Recruiting

Study contacts

• Study coordinator: Chiesi Clinical Trial
• Email: clinicaltrials_info@chiesi.com
• Phone: +3905212791

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Quality of life for people 65+ with Fabry disease, treated or untreated

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• Agalsidase alfa for heart inflammation in Fabry disease measured by PET‑CMR

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.