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A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients

Observational study on Elfabrio® for Fabry patients' safety and effectiveness.

Recruiting
18 years and older
All
Phase N/A
**Study Overview**: This study looks at how safe and effective Elfabrio® is for people with Fabry disease. Fabry disease is a genetic condition that affects the body's ability to break down fats, leading to problems with the heart and kidneys. The study will observe participants who are already taking or planning to take a medicine called pegunigalsidase alfa (Elfabrio®). It involves both looking back at past treatments (retrospective) and observing future treatments (prospective). **Eligibility**: To join, you must be over 18, have a confirmed diagnosis of Fabry disease, and either be taking or planning to take Elfabrio®. You cannot join if you have certain health conditions or if you are participating in another interventional study. **Key Points**: - **Study Type**: Observational (no new treatments given, just monitoring). - **Participation Requirements**: Must have regular heart imaging tests and complete surveys. - **Exclusions**: Cannot have had recent heart attacks, severe heart disease, or be pregnant. For more details, participants need to sign a consent form to agree to the study's rules and requirements.
Study details
    Fabry Disease

NCT06663358

Chiesi Farmaceutici S.p.A.

7 March 2026

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