🏛️ Verified NIH Record • Updated 2026-08-07

A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease (NCT06328608)

🟢 Recruiting Phase2 / Phase3 💊 Investigational Drug / Biologic Sponsor: Chiesi Farmaceutici S.p.A.

🩺 Plain-English Summary (8th-Grade Level)

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

Who can join: To join this study, participants generally need to meet the listed inclusion criteria (6 items) and avoid the listed exclusions (18 items).

Location: Phoenix Children's — Phoenix, Arizona

📋 "Do I Qualify?" — 1-Minute Self Screener

Instant Checklist

Check every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).

Usually NOT eligible if any of the following apply:

  • 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:

🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.

📍 All Participating Trial Locations (12 Sites)

  • Phoenix Children's — Phoenix, Arizona
  • Emory Genetics Clinical Trials Center — Atlanta, Georgia
  • University of Iowa — Iowa City, Iowa
  • Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio
  • University of Utah — Salt Lake City, Utah
  • Lysosomal and Rare Disorders Research and Treatment Center Inc — Fairfax, Virginia
  • UK für Kinder- und Jugendheilkunde der PMU Salzburg — Salzburg,
  • Centre Hospitalier Universitaire (CHU) de Bordeaux - Groupe Hospitalier Pellegrin — Bordeaux,
  • Hopital Arnaud de Villeneuve — Montpellier,
  • Haukeland Universitetssjukehus — Bergen,
  • Hospital Clinico Universitario De Santiago De Compostela — Santiago de Compostela,
  • Great Ormond Street Hospital for Children NHS Foundation Trust — London,

💰 Cost, Insurance & Patient Rights

  • 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
  • Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
  • Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
🏛️ View Official NIH Record ↗