Recruiting

Observational Study

Sponsor:

Shire

Code:

NCT03291223

Conditions

Gaucher Disease

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Study Details

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.

Conditions

Gaucher Disease

Study ID

NCT03291223

Start date

Dec 29, 2010

Status verified date

May, 2026

Completion date

Sep 30, 2026

Anticipated

Primary completion date

Sep 30, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

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 Design

Enrollment

1257 participants

Anticipated

Interventions and Outcome Measures

Arms

GOS Participants

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

Primary outcome measure

  • 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 ]
  • Number of Participants With Infusion-related Reactions (IRRs) [ Time Frame: Baseline to one year for up to 20 years ]
  • Increase of Hemoglobin Concentration [ Time Frame: Baseline to one year for up to 20 years ]
  • Increase of Platelet Count [ Time Frame: Baseline to one year for up to 20 years ]
  • Decrease in Liver Volume [ Time Frame: Baseline to one year for up to 20 years ]
  • Decrease in Spleen Volume [ Time Frame: Baseline to one year for up to 20 years ]
  • Increase in Bone Mineral Density (BMD) [ Time Frame: Baseline to one year for up to 20 years ]

Central Contacts and Locations

Central contacts

Shire Contact

+1 866 842 5335ClinicalTransparency@shire.com

Locations

Central Contact

Recruiting

Lexington, Massachusetts, United States, 02421

Contacts

Shire Central Contact

ClinicalTransparency@shire.com

Principal Investigator:

Central Contact

More Information

Sponsor

Shire

Last update posted

May 14, 2026

Last verified

May, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Shire on 2026-05-14.