Gaucher Disease Outcome Survey (GOS)

• ClinicalTrials.gov Identifier: NCT03291223
• Recruitment Status: Recruiting
• First Posted: September 25, 2017
• Last Update Posted: December 21, 2020
• Sponsor: Shire
• Information provided by (Responsible Party): Takeda ( Shire )

Study Description

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.

Study Design

• Study Type: Observational [Patient Registry]
• Estimated Enrollment: 1257 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Target Follow-Up Duration: 6 Months
• Official Title: Gaucher Disease Outcome Survey (GOS)
• Actual Study Start Date: July 27, 2010
• Estimated Primary Completion Date: December 15, 2021
• Estimated Study Completion Date: December 15, 2021

Groups and Cohorts

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

Outcome Measures

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.

Eligibility Criteria

• 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.

Criteria

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)

Contacts and Locations

Contacts

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

Locations

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

Shire

Investigators

• Study Director: Shire Study Physician; Shire

More Information

Additional Information:

• Responsible Party: Shire
• ClinicalTrials.gov Identifier: NCT03291223
• Other Study ID Numbers: GOS
• First Posted: September 25, 2017
• Last Update Posted: December 21, 2020
• Last Verified: December 2020

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Gaucher Disease; Sphingolipidoses; 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; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Lipid Metabolism Disorders