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NCT Number: NCT03291223

Gaucher Disease Outcome Survey (GOS)

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.

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Key information

Conditions

Gaucher Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Sphingolipidoses

Sex eligibility

All sexes

Study type

Observational

Primary location

Central Contact

Lexington, Massachusetts, 02421, United States

Location status: Recruiting

Location contact

Central Contact

PRINCIPAL_INVESTIGATOR

Shire Central Contact

CONTACT

[email protected]

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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 <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.

Exclusion criteria

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

Treatment and study plan

Primary outcomes

  1. 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

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

  2. Number of Participants With Infusion-related Reactions (IRRs)

    Time frame: 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.

  3. Increase of Hemoglobin Concentration

    Time frame: Baseline to one year for up to 20 years

    Hemoglobin concentration will be assessed.

  4. Increase of Platelet Count

    Time frame: Baseline to one year for up to 20 years

    Platelet count will be assessed.

  5. Decrease in Liver Volume

    Time frame: Baseline to one year for up to 20 years

    Liver volume will be assessed by abdominal imaging.

  6. Decrease in Spleen Volume

    Time frame: Baseline to one year for up to 20 years

    Spleen volume will be assessed by abdominal imaging.

  7. Increase in Bone Mineral Density (BMD)

    Time frame: Baseline to one year for up to 20 years

    Bone mineral density will be assessed.

Study contacts

Contact information is provided by the study sponsor or research team.

Shire Contact

CONTACT

[email protected]

+1 866 842 5335

Sponsors and collaborators

Lead sponsor

Shire

Industry

Registry information

Important dates

Study start
2010
Primary completion
2026
Study completion
2026
First posted
Sep 25, 2017
Registry last updated
May 14, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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