Gaucher Disease Outcome Survey (GOS)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03291223|
Recruitment Status : Recruiting
First Posted : September 25, 2017
Last Update Posted : March 19, 2019
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|
|Study Type :||Observational [Patient Registry]|
|Estimated Enrollment :||1257 participants|
|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 1, 2020|
|Estimated Study Completion Date :||December 1, 2020|
GOS is a disease specific registry open to all Gaucher patients irrespective of treatment status or type of treatment
- 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.
- 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.
- Increase of Hemoglobin Concentration [ Time Frame: Baseline to one year for up to 20 years ]Hemoglobin concentration will be assessed.
- Increase of Platelet Count [ Time Frame: Baseline to one year for up to 20 years ]Platelet count will be assessed.
- Decrease in Liver Volume [ Time Frame: Baseline to one year for up to 20 years ]Liver volume will be assessed by abdominal imaging.
- Decrease in Spleen Volume [ Time Frame: Baseline to one year for up to 20 years ]Spleen volume will be assessed by abdominal imaging.
- Increase in Bone Mineral Density (BMD) [ Time Frame: Baseline to one year for up to 20 years ]Bone mineral density will be assessed.
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
|Contact: Shire Contact||+1 866 842 5335||ClinicalTransparency@shire.com|
|United States, Massachusetts|
|Lexington, Massachusetts, United States, 02421|
|Contact: Shire Central Contact ClinicalTransparency@shire.com|
|Principal Investigator: Central Contact|
|Study Director:||Shire Study Physician||Shire|