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

  • End date
    May 31, 2023
  • participants needed
  • sponsor
Updated on 22 August 2021
replacement therapy
type 1 gaucher disease
velaglucerase alfa
substrate reduction therapy


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.

Condition Gaucher Disease, gaucher's disease
Clinical Study IdentifierNCT04094181
Last Modified on22 August 2021


Yes No Not Sure

Inclusion Criteria

Participant with GD1 currently being treated with an ERT/SRT other than VPRIV for at least 6 months before baseline enrolment; or participant previously treated with another ERT/ SRT for at least 6 months prior to transitioning to VPRIV
Participant or legally authorized representative has provided written informed consent

Exclusion Criteria

In the opinion of the investigator, participant is at high risk of non-compliance
In the opinion of the investigator, participant is unsuitable in any other way to participate in this study
Participant is pregnant
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What happens next?
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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