A Study of VPRIV in Participants With Gaucher Disease Previously Treated With Other Enzyme Replacement Therapies or Substrate Reduction Therapies

  • STATUS
    Not Recruiting
  • End date
    May 31, 2023
  • participants needed
    24
  • sponsor
    Shire
Updated on 23 May 2022
replacement therapy
type 1 gaucher disease
velaglucerase alfa
substrate reduction therapy
vpriv

Summary

The main aim of this study is to describe the safety profile of velaglucerase alfa (VPRIV) in participants with Gaucher disease type 1. Participants will be transitioning from other enzyme replacement therapies or substrate reduction therapies to VPRIV. Some participants may have already transitioned to treatment with VPRIV before this study started.

In this study, data on VPRIV will be collected from the medical records of participants who already transitioned to VPRIV before this study started. Other participants will join this study when they transition to VPRIV. All participants will be followed to allow for 12 months of observation from time of transition to VPRIV.

The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during the study.

Details
Condition Gaucher Disease
Clinical Study IdentifierNCT04094181
SponsorShire
Last Modified on23 May 2022

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