Fabry Outcome Survey (FOS)

Shire

Status: Completed

Conditions: Fabry Disease

Study type: Observational

Funder types: Industry

Identifiers

NCT03289065

FOS

Details and patient eligibility

About

The purpose of this study is to collect data that will increase understanding of Fabry disease history and progression, in treated and untreated patients with Fabry disease. The data from FOS may provide guidance to healthcare professionals about disease treatment options.

Enrollment: 4,000 patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Participants must have a documented diagnosis of Fabry disease

   • This may include a genetic mutation analysis. The collection of the genetic mutation analysis result is optional and dependent on the participant providing their consent for this data to be used in the FOS registry.
   • Participants can be untreated, currently or previously treated with Replagal, or any other approved treatment for Fabry disease.

2. Signed and dated written informed consent from the participant

   • For participants aged less than (<) 18 years (or as per local regulation), parent and/or participant's legally authorized representative (LAR), and assent of the minor, where applicable, is necessary.
   • If a participant is unable to read or if a legally acceptable representative is unable to read, an impartial witness should be present during the informed consent discussion and should sign and personally date the informed consent.
   • Informed consent must be obtained from LARs for cognitively impaired participants when applicable.

Exclusion criteria

1. Participants currently enrolled in ongoing blinded clinical trials (drugs or devices; includes all blinded trials) will be excluded from the Registry.

Trial design

4,000 participants in 1 patient group

FOS Participant

Description:

FOS is a disease registry open to all Fabry participants irrespective of treatment status or type of treatment.