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

NCT04094181 | Terminated DMC

• 2 other identifiers: SHP669-405EUPAS42338
• Study Type: observational
• Target: 2
• Locations: 1 country
• Sites: 1

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.

Conditions

Gaucher Disease

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• Change From Baseline in Use of Glucosylspingosine (Lyso-Gb1) Biomarker

  Baseline, Month 12

• Change From Baseline in Gaucher Disease Questionnaire Patient Reported Outcomes (PRO) Score at Month 12

  Baseline, Month 12

Study Arms (1)

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.

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Shire: lead

Study Sites (1)

University of Alberta/Medical Genetics Clinic

Edmonton, Alberta, T6G 2H7, Canada

Location

Related Links

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

MeSH Terms

Conditions

Gaucher Disease

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Officials

• Study Director

  Shire

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: OTHER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 17, 2019
• First Posted: September 18, 2019
• Study Start: September 19, 2019
• Primary Completion: November 17, 2021
• Study Completion: November 17, 2021
• Last Updated: February 2, 2024

Record last verified: 2024-01

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1)