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Gaucher Disease Outcome Survey (GOS)

IDENTIFIERS
Trial Protocol: GOS
Clinicaltrials.gov: NCT03291223
PURPOSE

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.

Recruiting
US

Trial at a glance

What medical condition is being studied?   Gaucher Disease
Are placebos part of the trial?   No
How many participants are being enrolled?   1257
When is the trial being conducted?    - 
How long is participation in the trial?   Patients will be followed in the registry for as long as the investigator or patient deems appropriate. Data from routine patient visits for the management of Gaucher disease will be entered into the registry database. Patients will be given the opportunity to complete health-related quality of life (HRQoL) questionnaires and the patient responses also will be entered into the registry database at follow-up visits. No treatment will be provided to the patient as a result of participating in this study. All treatments and/or any changes considered necessary for a patient’s welfare will be determined at the discretion of patient’s doctor. Management and care of patients are not affected by participation.

Key requirements

Sex   
All
Age   
All
Accepts Healthy Volunteers?   
No

Trial Details / Participation Requirements

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Trial Phase:   Phase 4
Trial Type:   Observational
Trial Arms/Groups or Cohorts:   
GOS Participants: GOS is a disease specific registry open to all Gaucher patients irrespective of treatment status or type of treatment
Primary Outcome Measure(s):   
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]
Entry Criteria:
Patients must have a documented diagnosis of Gaucher disease Cannot be currently enrolled in any ongoing blinded clinical trials

Trial Locations

Any
Recruiting
Any
RESULTS
Central Contact

73 Waltham St, Lexington, MA 02421, USA

Shire Central Contact
Recruiting
5,532 miles (8,903 km)  awayfrom
Новосибирск, Новосибирская область
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Trial Protocol ID: GOS
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