Long-term registry following people with Gaucher disease in routine care
Records health and treatment over time in people with Gaucher disease, without a study drug.
Plain-language title generated from the registry record
Registry title: Gaucher Disease Outcome Survey (GOS)
Key facts
- Where
- Lexington, United StatesSee the site
Where in United States
- LexingtonLexington, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Shire
- Trial code
- NCT03291223
- Record updated
- 15 September 2026
- Original description
The Gaucher Outcomes Survey (GOS) is an ongoing observational, international, multi-center, long-term Registry of Patients with Gaucher Disease irrespective of their treatment status or type of treatment received. No experimental intervention is involved. Patients undergo clinical assessments and receive care as determined by the patients' treating physician.Read full original descriptionShow less
Gaucher Disease Outcome Survey (GOS)
The Gaucher Outcomes Survey (GOS) is an ongoing observational, international, multi-center, long-term Registry of Patients with Gaucher Disease irrespective of their treatment status or type of treatment received. No experimental intervention is involved. Patients undergo clinical assessments and receive care as determined by the patients' treating physician.
The objectives of the registry include to evaluate the safety and long-term effectiveness of velaglucerase alfa, to characterize patients receiving velaglucerase alfa or other Gaucher Disease-specific treatments, to gain a better understanding of the natural history of GD and to serve as a database for evidence-based management of Gaucher Disease over time in real-life clinical practice.
Inclusion: 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: Patients currently enrolled in ongoing blinded clinical trials (drugs or devices; includes all blinded trials).