N/A
Completed N=63
Survey Study for Velaglucerase Alfa (VPRIV) in Japan
Source: ClinicalTrials.gov NCT03625882 ↗Enrolled (actual)
63
Serious AEs
45.0%
Results posted
Jan 2025
Primary outcomePrimary: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAE) Following Initiation of Treatment With Velaglucerase Alfa — 41; 27 Participants
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAE) Following Initiation of Treatment With Velaglucerase Alfa |
41; 27 | — |
| SECONDARY Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Hemoglobin Concentration |
11; 4; 20 | — |
| SECONDARY Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Platelet Counts |
9; 6; 4 | — |
| SECONDARY Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Liver Volumes |
0; 1; 6 | — |
| SECONDARY Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Spleen Volumes |
3; 2; 3 | — |
| SECONDARY Lumbar Spine Bone Mineral Density (BMD) T-scores and Femur Neck BMD T-scores at Baseline and the Last Data Collection |
-4.30; -0.30; -2.90; -0.60 | — |
| SECONDARY Lumbar Spine BMD Z-scores and Femur Neck BMD Z-scores at Baseline and the Last Data Collection |
-2.20; -1.15; -0.60; -0.50 | — |
| SECONDARY Lumbar Spine BMD and Femur Neck BMD at Baseline and the Last Data Collection |
0.7215; 0.3550; 0.6335; 0.3400 | — |
| SECONDARY Number of Participants With Positive Anti-velaglucerase Alfa Antibody Test Results |
2 | — |
| SECONDARY Number of Participants With Adverse Drug Reaction and Serious Adverse Drug Reaction Following Initiation of Treatment With Velaglucerase Alfa |
14; 5 | — |
| SECONDARY Number of Participants With Adverse Reactions Categorized as Infusion Reactions Following Initiation of Treatment With Velaglucerase Alfa |
6 | — |
Eligibility Criteria
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
-
Data sourced from ClinicalTrials.gov (NCT03625882). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.