Gaucher Disease Outcome Survey (GOS)
Recruiting now
Conditions studied: Gaucher Disease
In brief
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.
Key facts
- Study ID
- NCT03291223
- Run by
- Shire
- People needed
- 1257
- Starts
- 2010-12-29
- Expected to finish
- 2026-09-30
- Last updated by the study team
- 2026-05-14
Who can join
Age: any. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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.
You may not qualify if…
- Patients currently enrolled in ongoing blinded clinical trials (drugs or devices; includes all blinded trials)
Where it is running
- Central Contact — Lexington, Massachusetts, United States (enrolling)
Full record on ClinicalTrials.gov
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