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

VPRIV® Non-Interventional Study in Patients Previously Treated With Other Enzyme Replacement Therapies (ERTs)/Substrate Reduction Therapies (SRTs)

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.

Study Overview

• Status: Terminated
• Conditions: Gaucher Disease

• Study Type: Observational
• Enrollment (Actual): 2

Contacts and Locations

Study Locations

• Canada
  • Alberta
    • Edmonton, Alberta, Canada, T6G 2H7
      • University of Alberta/Medical Genetics Clinic

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

Participants included in the study will have a diagnosis of Type 1 GD and be in enzyme/substrate replacement therapy other than VPRIV, for at least 6 months prior to enrolment in the study, or have been previously treated with an ERT/SRT other than VPRIV for a minimum of 6 months prior to transitioning to VPRIV.

Description

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 Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
VPRIV Participants; 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants with Adverse Events (AEs) Following the Transition From Other ERTs/SRTs to VPRIV	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.	Baseline up to 12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in Use of Glucosylspingosine (Lyso-Gb1) Biomarker	Change in use of Lyso-Gb1 by participants following the transition from other ERTs/SRTs to velaglucerase alfa (VPRIV) will be assessed.	Baseline, Month 12
Change From Baseline in Gaucher Disease Questionnaire Patient Reported Outcomes (PRO) Score at 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.	Baseline, Month 12

Collaborators and Investigators

• Sponsor: Shire
• Investigators: Study Director: Study Director, Shire

Publications and helpful links

Helpful Links

• To obtain more information on the study, click here/on this link

Study record dates

Study Major Dates

• Study Start (Actual): September 19, 2019
• Primary Completion (Actual): November 17, 2021
• Study Completion (Actual): November 17, 2021

Study Registration Dates

• First Submitted: September 17, 2019
• First Submitted That Met QC Criteria: September 17, 2019
• First Posted (Actual): September 18, 2019

Study Record Updates

• Last Update Posted (Actual): February 2, 2024
• Last Update Submitted That Met QC Criteria: January 31, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Lipidoses; Lipid Metabolism, Inborn Errors; Gaucher Disease
• Other Study ID Numbers: SHP669-405; EUPAS42338 (Registry Identifier: EUPAS)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants).
• IPD Sharing Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No