Gaucher Disease Outcome Survey (GOS)

Part of paid clinical trials in Lexington, Massachusetts.

Sponsor
Shire
Study ID
NCT03291223
Status
Recruiting
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Conditions

  • Gaucher Disease

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Study Details

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.

Key Dates

Start date
Dec 29, 2010
Status verified
May 2026
Primary completion
Sep 30, 2026
Completion
Sep 30, 2026

Study Design

Enrollment
1,257 participants (estimated)

Arms

  • Arm: GOS Participants
    GOS is a disease specific registry open to all Gaucher patients irrespective of treatment status or type of treatment

Primary Outcome Measure

Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Baseline to one year for up to 20 years ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Central ContactLexingtonMassachusetts02421
Shire Central Contact
[email protected]
Central Contact (PRINCIPAL_INVESTIGATOR)

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