NCT03291223

Gaucher Disease Outcome Survey (GOS)

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 1,257 (estimated)
• Sponsor: Shire · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

20 MAY 2020: The temporary enrollment stop of new patients into this study due to the COVID-10 pandemic has been lifted in one or more countries/sites, and the study is now again enrolling new patients. However, some countries/sites may still have paused the enrollment of new patients due to the pandemic. 24 APRIL 2020: Enrollment of new patients into this study has been paused due to the COVID-19 situation. The duration of this pause is dependent on the leveling and control of the COVID-19 pandemic.

Conditions

• Gaucher Disease

Timeline

• Start date: 2010-07-27
• Primary completion: 2026-09-30
• Completion: 2026-09-30
• First posted: 2017-09-25
• Last updated: 2025-10-09

Locations

1 site across 1 country: United States