- ICH GCP
- US Clinical Trials Registry
- Clinical Trial 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.
Study Overview
Status
Conditions
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.
Study Type
Enrollment (Estimated)
Contacts and Locations
Study Contact
- Name: Shire Contact
- Phone Number: +1 866 842 5335
- Email: ClinicalTransparency@shire.com
Study Locations
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Massachusetts
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Lexington, Massachusetts, United States, 02421
- Recruiting
- Central Contact
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Contact:
- Shire Central Contact
- Email: ClinicalTransparency@shire.com
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Principal Investigator:
- Central Contact
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
Sampling Method
Study Population
Description
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)
Study Plan
How is the study designed?
Design Details
Cohorts and Interventions
Group / Cohort |
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GOS Participants
GOS is a disease specific registry open to all Gaucher patients irrespective of treatment status or type of treatment
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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
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Treatment-emergent adverse events (TEAEs) are defined as adverse events (AEs) that either commenced or worsened following the first dose of VPRIV.
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Baseline to one year for up to 20 years
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Number of Participants With Infusion-related Reactions (IRRs)
Time Frame: Baseline to one year for up to 20 years
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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.
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Baseline to one year for up to 20 years
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Increase of Hemoglobin Concentration
Time Frame: Baseline to one year for up to 20 years
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Hemoglobin concentration will be assessed.
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Baseline to one year for up to 20 years
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Increase of Platelet Count
Time Frame: Baseline to one year for up to 20 years
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Platelet count will be assessed.
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Baseline to one year for up to 20 years
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Decrease in Liver Volume
Time Frame: Baseline to one year for up to 20 years
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Liver volume will be assessed by abdominal imaging.
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Baseline to one year for up to 20 years
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Decrease in Spleen Volume
Time Frame: Baseline to one year for up to 20 years
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Spleen volume will be assessed by abdominal imaging.
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Baseline to one year for up to 20 years
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Increase in Bone Mineral Density (BMD)
Time Frame: Baseline to one year for up to 20 years
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Bone mineral density will be assessed.
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Baseline to one year for up to 20 years
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Collaborators and Investigators
Sponsor
Investigators
- Study Director: Shire Study Physician, Shire
Publications and helpful links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Genetic Diseases, Inborn
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Lipid Metabolism Disorders
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Sphingolipidoses
- Lysosomal Storage Diseases, Nervous System
- Lipidoses
- Lipid Metabolism, Inborn Errors
- Gaucher Disease
Other Study ID Numbers
- GOS
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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