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

Shire

Status: Terminated

Conditions: Gaucher Disease

Study type: Observational

Funder types: Industry

Identifiers

NCT04094181

SHP669-405

EUPAS42338 (Registry Identifier)

Details and patient eligibility

About

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.

Enrollment: 2 patients

Sex: All

Volunteers: No Healthy Volunteers

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.

Trial design

2 participants in 1 patient group

VPRIV Participants

Description:

Participants who has been transitioned from ERTs/SRTs to VPRIV, the data will be collected retrospectively from time of transition until the point at which participant begins in this study and then will be followed up prospectively for 12 months.