ClinicalTrials.gov record NCT07109375
Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting
Observational Prospective Cohort Study on Long-term Effective and Safe Use of Pegunigalsidase Alfa in Adult Fabry Patients Under "Real-world" Setting
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This is a prospective observational study collecting real-world data on adult patients with genetically confirmed Fabry disease who are taking, or plan to start, pegunigalsidase alfa. The study follows participants with clinical tests and questionnaires over time to record safety and clinical outcomes. Treatment choices are made by patients and clinicians independently of study participation.
What it is studying. The study is testing how pegunigalsidase alfa is used in real clinical practice and collecting data on its safety and effectiveness, including heart and kidney measures, exercise capacity, blood and urine biomarkers, quality of life, adverse events, and anti-drug antibodies.
What the researchers hope to learn. To build real-world evidence on long-term safety and effectiveness of pegunigalsidase alfa in adults with Fabry disease. Specific goals include tracking changes in left ventricular structure and function, kidney function, exercise capacity, biomarkers (for example lyso-Gb3, NT‑pro‑BNP, troponin), quality-of-life scores, adverse events, anti-drug antibodies, and infusion-related reactions.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
PEGASO is an observational study designed to collect prospective data on the effectiveness and safety of pegunigalsidase alfa in adult patients with Fabry disease, being treated or planning to start treatment, under real-world setting.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- pegunigalsidase alfa
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Male and female adults (≥ 18 years).
- Patients with a clinical diagnosis of Fabry disease confirmed by α-Gal A assay and detection of mutation in α-Gal A gene.
- Patients either taking or planning to take pegunigalsidase alfa as treatment for Fabry disease. The treatment decision must be made independently from participation in this study.
- Written informed consent to participate in the study and for the processing of personal data.
Exclusion criteria
- History of hypersensitivity reaction to pegunigalsidase alfa.
- Presence of any medical, emotional, behavioural, or psychological condition that, in the judgment of the physician, could interfere with the ability to participate in the study.
- Female subjects who are pregnant or planning to become pregnant during the study.
Study description and what participation involves
The registry record has no detailed description beyond the summary above.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants will receive pegunigalsidase alfa as part of their usual care (intravenous infusion every two weeks as described) or start it per clinical practice while the study collects data. Scheduled assessments over up to 24 months include heart imaging (echocardiography and cardiac MRI), exercise testing (cardiopulmonary exercise testing), kidney tests including 24-hour urine collection, blood tests for biomarkers and anti-drug antibodies, slit‑lamp eye exams, and questionnaires about quality of life, pain, and gastrointestinal symptoms. The study will also record adverse events and any infusion-related reactions. Participation follows routine-care treatment decisions and requires signing informed consent.
How the study is structured. A prospective observational cohort: people who are treated with or plan to start pegunigalsidase alfa receive usual care while the study team collects medical data at baseline and during follow-up. Data include heart imaging (echocardiography, cardiac MRI), exercise testing, blood and urine tests, and patient questionnaires; no experimental treatment is assigned by the study.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
- The listed sites are in Italy. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on Fabry disease
Important dates
- Study start
- Jun 15, 2026
- Primary completion
- February 2029 (estimated)
- Study completion
- February 2029 (estimated)
- Target enrollment
- 75 participants (estimated)
- Last updated on registry
- Jul 8, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Chiesi Italia
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Chiesi Clinical Trial info 📞 +3905212791 ✉ clinicaltrials_info@chiesi.com
Participating sites (7)
- AUSL Toscana Sud-Est - Ospedale San DonatoNot Yet Recruiting
Arezzo · Italy
- Maurizio Pieroni, MD
- Maurizio Pieroni
- Azienda Ospedaliera Universitaria Consorziale Policlinico Di BariRecruiting
Bari · Italy
- Gesualdo
- Loreto Gesualdo
- Irccs Azienda Ospedaliero-Universitaria Di Bologna - Policlinico Di Sant'OrsolaRecruiting
Bologna · Italy
- La Manna
- Gaetano La Manna
- A.O. Sant'Anna e San SebastianoNot Yet Recruiting
Caserta · Italy
- Paolo Calabrò
- Paolo Calabrò
- U.O.C. Nefrologia ASST Monza - Ospedale San GerardoNot Yet Recruiting
Monza · Italy
- Federico Pieruzzi
- Federico Pieruzzi
- AOU Federico II, Dipartimento di NefrologiaNot Yet Recruiting
Naples · Italy
- Antonio Pisani, MD · +39 081 7464521
- A.O.U. Policlinico "Giaccone"Not Yet Recruiting
Palermo · Italy
- Antonino Tuttolomondo, MD
- Antonino Tuttolomondo
Terms used on this page · explanations by MedicalUpdates.ai
- Written informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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