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Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients

Sponsored by Amicus Therapeutics

About this trial

Last updated 2 years ago

Study ID

AT-NIS-00002

Status

Recruiting

Type

Observational

Placebo

No

Accepting

18-75 Years
18+ Years
All
All

Not accepting

Not accepting
Healthy Volunteers

Trial Timing

Ended 2 years ago

What is this trial about?

The objective of this study is to increase the understanding surrounding the choices presented to patients and families impacted by Fabry disease.

What are the participation requirements?

Yes

Inclusion Criteria

- Participant must be a person with Fabry disease who is 18 years or older or the parent/legal guardian of a living person with Fabry disease who is under the age of 18 years or who are 18 years and older who are unable to answer for themselves.

- Confirmed diagnosis of Fabry disease with written proof of disease provided

- Must have a genetic mutation that is amenable to oral therapy

- Resident of Germany, the U.K or the U.S.

- Able to read, write and communicate in German, or English.

- Able to grant informed consent

- Willing to participate in a 50 to 60-minute telephone interview, including follow up questions (if necessary) and information regarding adverse events (if necessary).

No

Exclusion Criteria

- Inability to meet any of the inclusion criteria

Locations

Location

Status

Recruiting