NCT03291223Recruiting
Gaucher Disease Outcome Survey (GOS)
Source: ClinicalTrials.gov · Last updated 15 Sept 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 1257 (estimated)
- First submitted
- 8 Aug 2017
- Start date
- 29 Dec 2010
- Primary completion date
- 30 Sept 2028
- Lead sponsor
- Shire
- Diseases
- Gaucher disease
- NCT number
- NCT03291223
What you commit to
- Study type: Observational
- Planned participants: 1257
- From 29 Dec 2010 to the primary completion date, 30 Sept 2028
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- \- Patients currently enrolled in ongoing blinded clinical trials (drugs or devices; includes all blinded trials)
Who the study is looking for
- 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.
Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Central Contact Coordinator of this site? Confirm its status | Lexington | United States | Recruiting |
Related publications
- Natural-History Mapping of Lysosomal Storage Disorders (LSDs): Gaucher Disease as a Model for Precision Care.J Inherit Metab Dis · 2026 · PMID 41527340
This page is information, not medical advice. Discuss any trial with your doctor.