Registry for patients with Gaucher Disease
Gaucher Disease Outcome Survey (GOS)
Takeda · NCT03291223
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 |
|---|---|
| Enrollment | 1257 (estimated) |
| Sex | All |
| Sponsor | Takeda (industry) |
| Locations | 1 site (Lexington, Massachusetts) |
| Trial ID | NCT03291223 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates include patients of any age or gender with a confirmed diagnosis of Gaucher Disease.
Not a fit: Patients currently enrolled in ongoing blinded clinical trials may not benefit from this registry.
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 performed: Other observational studies have shown success in characterizing chronic conditions, making this approach promising for Gaucher Disease.
Eligibility criteria
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 this trial is running
Lexington, Massachusetts
- Central Contact — Lexington, Massachusetts, United States (RECRUITING)
Study contacts
- Study coordinator: Shire Contact
- Email: ClinicalTransparency@shire.com
- Phone: +1 866 842 5335
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Gaucher Disease