Gaucher Disease Outcome Survey (GOS)

Launched by SHIRE · Sep 19, 2017

Keywords

ClinConnect Summary

The Gaucher Outcomes Survey (GOS) is a research study focused on understanding Gaucher Disease, a genetic condition that affects how the body processes certain fats. This study does not involve any new treatments; instead, it collects information from patients who are receiving various treatments or none at all. The goal is to evaluate the safety and long-term effectiveness of a specific treatment called velaglucerase alfa, learn more about how Gaucher Disease affects patients over time, and create a valuable resource for doctors to manage the condition better in real-life situations.

Anyone diagnosed with Gaucher Disease, regardless of age or gender, can participate in this study, as long as they provide consent. For younger patients, their parents or guardians can give consent on their behalf. Participants will receive regular medical assessments and care based on their doctor’s recommendations. This study is currently recruiting participants, and it’s important to note that individuals who are already involved in other blinded clinical trials (where neither the patient nor the doctor knows which treatment is being given) cannot join this one.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Patients of any age or gender with confirmed diagnosis (biochemical and/or genetic) of Gaucher disease
• • Signed and dated written informed consent from the patient or, for patients aged \<18 years (\<16 years in the United Kingdom \[UK\]), their parent and/or legally authorized representatives (LAR), and assent of the minor where applicable. Legally authorized representatives are also applicable for cognitively impaired patients.
• Exclusion Criteria:
• • - Patients currently enrolled in ongoing blinded clinical trials (drugs or devices; includes all blinded trials)