Who is eligible for clinical trial NCT03291223?

Participants must meet protocol criteria for Gaucher Disease. Confirm exact inclusion and exclusion requirements with the study coordinator.

Does clinical trial NCT03291223 provide travel or cost reimbursement?

Check with the site coordinator at the participating facility for travel and lodging stipend availability, and for which routine-care costs are covered.

🏛️ Verified NIH Record • Updated 2026-05-11

Gaucher Disease Outcome Survey (GOS) (NCT03291223)

🟢 Recruiting Interventional Protocol 📊 Observational Clinical Research Sponsor: Shire

🩺 Plain-English Summary (8th-Grade Level)

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.

Who can join: To join this study, participants generally need to meet the listed inclusion criteria (2 items) and avoid the listed exclusions (1 items).

Location: Central Contact — Lexington, Massachusetts

📋 "Do I Qualify?" — 1-Minute Self Screener

Instant Checklist

Check every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).

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.

Usually NOT eligible if any of the following apply:

Exclusion Criteria: \- Patients currently enrolled in ongoing blinded clinical trials (drugs or devices; includes all blinded trials)

🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.

📍 All Participating Trial Locations (1 Sites)

Central Contact — Lexington, Massachusetts

💰 Cost, Insurance & Patient Rights

100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

🏛️ View Official NIH Record ↗