Registry for patients with Gaucher Disease
Official title: Gaucher Disease Outcome Survey (GOS)
Run by Takeda · NCT03291223 · Data updated 2026-11-12
This study is collecting information from people with Gaucher Disease to see how well different treatments work and to learn more about the disease over time.
Quick facts
| Study type | Observational |
|---|---|
| Sex | All |
| Enrollment | 1257 (estimated) |
| Sponsor | Takeda Industry-sponsored |
| Locations | 1 site (Lexington, Massachusetts) |
| Status | Recruiting |
| Started | December 29, 2010 |
| Main results expected | September 30, 2028 |
| Study ends | September 30, 2028 |
| Registry record updated | September 15, 2026 |
| Trial ID | NCT03291223 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates include patients of any age or gender with a confirmed diagnosis of Gaucher Disease.
✕Probably not a fit
Patients currently enrolled in ongoing blinded clinical trials may not benefit from this registry.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
The Gaucher Outcomes Survey (GOS) is an ongoing observational registry that collects data from patients with Gaucher Disease, regardless of their treatment status. This international, multi-center initiative aims to evaluate the long-term safety and effectiveness of treatments like velaglucerase alfa, while also characterizing the patient population and understanding the natural history of the disease. Patients receive care as determined by their treating physician, and the registry serves as a valuable database for evidence-based management of Gaucher Disease over time.
Why it matters
Potential benefit: If successful, this registry could enhance the understanding and management of Gaucher Disease, leading to improved patient outcomes.
How similar studies have gone: Other observational studies have shown success in characterizing chronic conditions, making this approach promising for Gaucher Disease.
Who can join: the full rules
Show full inclusion / exclusion 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)
Where it's running
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Central Contact
Lexington, Massachusetts, United States Recruiting
Study contacts
- Study coordinator: Shire Contact
- Email: ClinicalTransparency@shire.com
- Phone: +1 866 842 5335
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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Gaucher Disease: Following Patients on Standard Care to See How It Progresses
This study will observe people with Gaucher disease who are receiving standard care to see how their condition changes over time and to explore the biology behind persistent or worsening disease.
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This study is creating a detailed registry to collect information on rare diseases like amyloidosis and sarcoidosis to help improve how they are diagnosed and treated.
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Prevalence of Acid Sphingomyelinase Deficiency and Gaucher Disease in Patients with Monoclonal Gammopathies
This study is trying to see how common Acid Sphingomyelinase Deficiency and Gaucher Disease are in patients with monoclonal gammopathies, especially those with an enlarged spleen or who have had their spleen removed.
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Exploring extracellular vesicles in Gaucher Disease
This study is trying to see how certain tiny particles in the body behave differently in people with Gaucher Disease compared to healthy individuals to understand their role in communication between cells and possible links to brain inflammation.
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Ambroxol for people with Gaucher disease or GBA-related Parkinson's disease
This project tests whether ambroxol is safe and may help people with Gaucher disease or Parkinson's disease caused by GBA gene changes.
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Long-term observation of rare genetic brain disorders
This study is tracking how rare genetic brain disorders progress over time to better understand their effects on patients.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.