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Gaucher Disease Outcome Survey (GOS)

This study, called the Gaucher Outcomes Survey (GOS), is an ongoing, long-term registry for people with Gaucher disease. It's an observational study, meaning you would receive your usual care from your doctor, and no experimental treatments are involved. Researchers are collecting information to better understand Gaucher disease and the long-term safety and effectiveness of treatments like velaglucerase alfa. They are looking at things like side effects, infusion reactions, and changes in hemoglobin (a protein in red blood cells). Anyone with a confirmed diagnosis of Gaucher disease can join, regardless of age or gender. Enrollment was temporarily paused due to COVID-19 but has now resumed in some locations.

Study design
This is an observational study with a planned enrollment of 1257 participants. It is not a randomized trial.
What's involved
You would undergo clinical assessments and receive care as determined by your treating physician. The study aims to collect data for up to 20 years.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for up to 20 years, with primary endpoints measured from baseline to one year.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT03291223

Gaucher Disease Outcome Survey (GOS)

Recruiting
Not specifiedAll AgesObservational
Shire
~1,257 participants
Updated 2026-09-15 on ClinicalTrials.gov

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured over Baseline to one year for up to 20 years
+6 more outcomes measured

Conditions

Gaucher Disease

Explore related trials

Gaucher Disease

NCT03291223

Where you'd take part

This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Central Contact

    Lexington, Massachusettsstudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Shire Study Physician · STUDY_DIRECTOR · Shire

Who to contact

Shire Contact
[email protected]
+1 866 842 5335
Email the study team

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Eligibility criteria

Inclusion

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.

What this trial measures

  • Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)Baseline to one year for up to 20 years

    Treatment-emergent adverse events (TEAEs) are defined as adverse events (AEs) that either commenced or worsened following the first dose of VPRIV.

  • Number of Participants With Infusion-related Reactions (IRRs)Baseline to one year for up to 20 years

    An IRR is defined as an AE that has been assessed as at least possibly related to treatment with VPRIV and occurs during an infusion or up to 24 hours post-VPRIV infusion.

  • Increase of Hemoglobin ConcentrationBaseline to one year for up to 20 years

    Hemoglobin concentration will be assessed.

  • Increase of Platelet CountBaseline to one year for up to 20 years

    Platelet count will be assessed.

  • Decrease in Liver VolumeBaseline to one year for up to 20 years

    Liver volume will be assessed by abdominal imaging.

  • Decrease in Spleen VolumeBaseline to one year for up to 20 years

    Spleen volume will be assessed by abdominal imaging.

  • Increase in Bone Mineral Density (BMD)Baseline to one year for up to 20 years

    Bone mineral density will be assessed.