Gaucher Disease Outcome Survey (GOS)
ConditionGaucher Disease
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorShire
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
None
Trial 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.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
1,257 Participants
are grouped into 1 trial groupGroup A: GOS Participants
Trial outcomes
Primary outcomes
1
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.
Time frame
Baseline to one year for up to 20 years2
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.
Time frame
Baseline to one year for up to 20 years3
Increase of Hemoglobin Concentration
Hemoglobin concentration will be assessed.
Time frame
Baseline to one year for up to 20 years4
Increase of Platelet Count
Platelet count will be assessed.
Time frame
Baseline to one year for up to 20 years Source ClinicalTrials.gov