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

Terminated

• Conditions: Gaucher Disease

• Registration Number: NCT04094181
• Lead Sponsor: Shire

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2019-09-17
• Study First Submitted QC: 2019-09-17
• Study First Posted: 2019-09-18
• Study Start: 2019-09-19
• Primary Completion: 2021-11-17
• Study Completion: 2021-11-17
• Status Verified: 2024-01
• Last Update Submitted: 2024-01-31
• Last Update Posted: 2024-02-02

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 2

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Number of Participants with Adverse Events (AEs) Following the Transition From Other ERTs/SRTs to VPRIV	Baseline up to 12 months	An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. Number of participants transitioning from other ERTs/SRTs to VPRIV with AEs will be reported.

Secondary Outcome Measures

Name	Time	Method
Change From Baseline in Use of Glucosylspingosine (Lyso-Gb1) Biomarker	Baseline, Month 12	Change in use of Lyso-Gb1 by participants following the transition from other ERTs/SRTs to velaglucerase alfa (VPRIV) will be assessed.
Change From Baseline in Gaucher Disease Questionnaire Patient Reported Outcomes (PRO) Score at Month 12	Baseline, Month 12	Gaucher disease questionnaire (PRO) scores will be calculated for each adult participant. The questionnaire asks how much the participant is affected by the Gaucher disease. A total summated score is calculated and ranges from 0 to 100 with higher scores indicating more severe impairment and worse quality of life.

Trial Locations

Locations (1)

University of Alberta/Medical Genetics Clinic; 🇨🇦 Edmonton, Alberta, Canada