VPRIV Drug Use-Result Survey (Japan)
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- Enrollment
- 63
- Locations
- 35
- Primary Endpoint
- Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAE) Following Initiation of Treatment With Velaglucerase Alfa
Study Overview
Brief Summary
The objective of this post-marketing survey study is to collect data to determine the safety and efficacy of velaglucerase alfa (VPRIV) in participants with Gaucher disease who are new to therapy or have been switched from another therapeutic agent for Gaucher disease.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Male or female participants with a confirmed diagnosis of Gaucher disease
- •Participants who are either naïve to treatment or participants that have been treated with another therapeutic agent for Gaucher disease
- •Participants who start VPRIV treatment or transition from VPRIV clinical studies during the enrollment period
Exclusion Criteria
- Not provided
Arms & Interventions
Gaucher Disease Participants Treated With VPRIV
Participants with Gaucher disease will be enrolled in this survey, who are in VPRIV treatment-naïve therapy or have been switched from another therapeutic agent for Gaucher disease.
Outcomes
Primary Outcomes
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAE) Following Initiation of Treatment With Velaglucerase Alfa
Time Frame: Baseline up to end of the study (8 years)
An AE is any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An SAE is any event that results in: death; life-threatening; requires inpatient hospitalisation or results in prolongation of existing hospitalisation; persistent or significant disability/incapacity; a congenital anomaly/birth defect or a medically important event.
Secondary Outcomes
- Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Hemoglobin Concentration(Baseline, Every 12 weeks up to 8 years)
- Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Platelet Counts(Baseline, Every 12 weeks up to 8 years)
- Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Liver Volumes(Baseline, Every 24 weeks up to 8 years)
- Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Spleen Volumes(Baseline, Every 24 weeks up to 8 years)
- Lumbar Spine Bone Mineral Density (BMD) T-scores and Femur Neck BMD T-scores at Baseline and the Last Data Collection(Baseline, at the time of last data collection (up to 8 years))
- Lumbar Spine BMD Z-scores and Femur Neck BMD Z-scores at Baseline and the Last Data Collection(Baseline, at the time of last data collection (up to 8 years))
- Lumbar Spine BMD and Femur Neck BMD at Baseline and the Last Data Collection(Baseline, at the time of last data collection (up to 8 years))
- Number of Participants With Positive Anti-velaglucerase Alfa Antibody Test Results(Baseline up to end of the study (8 years))
- Number of Participants With Adverse Drug Reaction and Serious Adverse Drug Reaction Following Initiation of Treatment With Velaglucerase Alfa(Baseline up to end of the study (8 years))
- Number of Participants With Adverse Reactions Categorized as Infusion Reactions Following Initiation of Treatment With Velaglucerase Alfa(Baseline up to end of the study (8 years))