Fabry Outcome Survey (FOS)



Status:Recruiting
Conditions:Hematology, Metabolic
Therapuetic Areas:Hematology, Pharmacology / Toxicology
Healthy:No
Age Range:Any
Updated:11/21/2018
Start Date:April 1, 2001
End Date:April 1, 2030
Contact:Shire Contact
Email:ClinicalTransparency@shire.com
Phone:+1 866 842 5335

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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.


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.
We found this trial at
1
site
300 Shire Way
Lexington, Massachusetts 02421
Principal Investigator: Shire PI
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mi
from
Lexington, MA
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