Gaucher Disease Outcome Survey (GOS)

Gaucher Disease Clinical Trial

Official title:

Gaucher Disease Outcome Survey (GOS)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03291223
• Other study ID #: GOS
• Status: Recruiting
• Start date: July 27, 2010
• Est. completion date: January 3, 2025

Study information

• Verified date: June 2024
• Source: Takeda
• Contact: Shire Contact
• Phone: +1 866 842 5335
• Email: ClinicalTransparency[@]shire.com
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 1257
• Est. completion date: January 3, 2025
• Est. primary completion date: January 3, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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)

Related Conditions & MeSH terms

• Gaucher Disease

Locations

Country	Name	City	State
United States	Central Contact	Lexington	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Shire

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)	Treatment-emergent adverse events (TEAEs) are defined as adverse events (AEs) that either commenced or worsened following the first dose of VPRIV.	Baseline to one year for up to 20 years
Primary	Number of Participants With Infusion-related Reactions (IRRs)	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.	Baseline to one year for up to 20 years
Primary	Increase of Hemoglobin Concentration	Hemoglobin concentration will be assessed.	Baseline to one year for up to 20 years
Primary	Increase of Platelet Count	Platelet count will be assessed.	Baseline to one year for up to 20 years
Primary	Decrease in Liver Volume	Liver volume will be assessed by abdominal imaging.	Baseline to one year for up to 20 years
Primary	Decrease in Spleen Volume	Spleen volume will be assessed by abdominal imaging.	Baseline to one year for up to 20 years
Primary	Increase in Bone Mineral Density (BMD)	Bone mineral density will be assessed.	Baseline to one year for up to 20 years

External Links

•   To obtain more information on the study, click here/on this link