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Gaucher Disease Outcome Survey (GOS)

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ClinicalTrials.gov Identifier: NCT03291223
Recruitment Status : Recruiting
First Posted : September 25, 2017
Last Update Posted : December 28, 2021
Information provided by (Responsible Party):
Takeda ( Shire )

Brief 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.

Condition or disease
Gaucher Disease

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.

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Study Type : Observational
Estimated Enrollment : 1257 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Gaucher Disease Outcome Survey (GOS)
Actual Study Start Date : July 27, 2010
Estimated Primary Completion Date : December 15, 2023
Estimated Study Completion Date : December 15, 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Gaucher Disease

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

Primary Outcome Measures :
  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.

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
GOS is a disease specific registry irrespective of treatment or treatment status, open to all patients of any age or sex with Gaucher disease of any type. Patients included may be those who are untreated, naive to therapy, individuals who are currently or have been previously treated with velaglucerase alfa (VPRIV), or individuals who have been receiving or are currently exposed to other treatments for Gaucher disease. There is no predefined sample size.

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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03291223

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Contact: Shire Contact +1 866 842 5335 ClinicalTransparency@shire.com

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United States, Massachusetts
Central Contact Recruiting
Lexington, Massachusetts, United States, 02421
Contact: Shire Central Contact       ClinicalTransparency@shire.com   
Principal Investigator: Central Contact         
Sponsors and Collaborators
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Study Director: Shire Study Physician Shire
Additional Information:
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Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT03291223    
Other Study ID Numbers: GOS
First Posted: September 25, 2017    Key Record Dates
Last Update Posted: December 28, 2021
Last Verified: December 2021

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Gaucher Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders