Gaucher Disease Outcome Survey (GOS)

Phase

N/A

Condition

Gaucher Disease

Treatment

N/A

Clinical Study ID

NCT03291223

GOS

All Genders

Study 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.

Eligibility Criteria

Inclusion

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 <18years (<16 years in the United Kingdom [UK]), their parent and/or legally authorizedrepresentatives (LAR), and assent of the minor where applicable. Legally authorizedrepresentatives are also applicable for cognitively impaired patients.

Exclusion

Exclusion Criteria:

Patients currently enrolled in ongoing blinded clinical trials (drugs or devices; includes all blinded trials)

Study Design

December 29, 2010

September 30, 2026

Study 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.

Connect with a study center

Central Contact
Lexington, Massachusetts 02421
United States
Active - Recruiting
Central Contact
Lexington 4941935, Massachusetts 6254926 02421
United States
Site Not Available

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