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Global Burden and Treatment Trajectiories in Italian Patients With Fabry Disease

NCT ID: NCT04916977

Last Updated: 2023-11-28

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

200 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-06-23

Study Completion Date

2022-06-30

Brief Summary

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GROUND study is an Italian, multicenter retrospective longitudinal cohort study with a cross-sectional phase with the aim to quantify the severe clinical burden in terms of severe and fatal outcomes and extension of clinical impairment in the Italian Fabry Disease patients' population

Detailed Description

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Conditions

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Fabry Disease

Study Design

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Observational Model Type

COHORT

Study Time Perspective

RETROSPECTIVE

Study Groups

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FD patients

FD patients

Intervention Type OTHER

Patients with Fabry disease diagnosis

Interventions

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FD patients

Patients with Fabry disease diagnosis

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

1. Adult patients living or deceased of either sex (age ≥18 years) with a documented FD diagnosis in the last 10 years from the beginning of the study.
2. Patients with at least 3 years of follow-up or early occurrence of severe/fatal outcomes. Also, adult patients who were lost by their care provider or physician and cannot be traced, but for which it is possible to retrieve data for at least 3 years before the last available follow-up visit or from diagnosis to the occurrence of severe/fatal outcomes if earlier, can be included.
3. Written informed consent to undergo in the cross-sectional protocol clinical visit and to retrospectively collect genetic/clinical data. In case of deceased or untraceable patients, the written informed consent is not required to collect retrospective clinical data (excluding genetic data).

Exclusion Criteria

* Not applicable
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Chiesi Italia

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Antonio Pisani, MD

Role: PRINCIPAL_INVESTIGATOR

Università degli Studi di Napoli "Federico II" NAPOLI

Federico Pieruzzi, MD

Role: PRINCIPAL_INVESTIGATOR

Ospedale San Gerardo MONZA

Renzo Mignani, MD

Role: PRINCIPAL_INVESTIGATOR

AUSL della Romagna - Ospedale Infermi RIMINI

Elena Verrecchia, MD

Role: PRINCIPAL_INVESTIGATOR

Fondazione Policlinico Universitario "Gemelli" IRCCS ROMA

Davide Noto, MD

Role: PRINCIPAL_INVESTIGATOR

A.O.U. Policlinico "Giaccone" PALERMO

Antonino Tuttolomondo, MD

Role: PRINCIPAL_INVESTIGATOR

A.O.U. Policlinico "Giaccone" PALERMO

Maurizio Pieroni, MD

Role: PRINCIPAL_INVESTIGATOR

AUSL Toscana Sud-Est - Ospedale San Donato AREZZO

Iacopo Olivotto, MD

Role: PRINCIPAL_INVESTIGATOR

A.O.U. Careggi FIRENZE

Locations

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AUSL Toscana Sud-Est - Ospedale San Donato

Arezzo, , Italy

Site Status

A.O.U. Careggi

Florence, , Italy

Site Status

U.O.C. Nefrologia ASST Monza - Ospedale San Gerardo

Monza, , Italy

Site Status

U.O. di Nefrologia - Dipartimento di Sanità Pubblica Università degli Studi di Napoli "Federico II" NAPOLI

Napoli, , Italy

Site Status

A.O.U. Policlinico "Giaccone"

Palermo, , Italy

Site Status

AUSL della Romagna - Ospedale Infermi

Rimini, , Italy

Site Status

Fondazione Policlinico Universitario "Gemelli" IRCCS

Roma, , Italy

Site Status

Countries

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Italy

Related Links

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https://www.chiesi.com/en/chiesi-clinical-study-register/

CSR Synopsis published on the Chiesi Group web-site

Other Identifiers

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DFIDM-2001

Identifier Type: -

Identifier Source: org_study_id