Actively Recruiting
Gaucher Disease Outcome Survey (GOS)
Led by Shire · Updated on 2026-05-14
1257
Participants Needed
1
Research Sites
822 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Gaucher Disease Outcome Survey (GOS)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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 applicable for cognitively impaired patients
You will not qualify if you...
- Patients currently enrolled in ongoing blinded clinical trials (drugs or devices; includes all blinded trials)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Central Contact
Lexington, Massachusetts, United States, 02421
Actively Recruiting
Research Team
S
Shire Contact
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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