NCT05669729

Brief Summary

The main purpose of this survey is to determine participants', caregivers', and nurses' understanding and use of educational materials (EM) on VPRIV home treatment. EM includes an infusion diary and guide and an emergency plan related to VPRIV infusion given at home for Gaucher disease. The survey is conducted in European countries. Data will be collected directly from participants, caregivers, and nurses in form of a questionnaire, electronic or paper.

Trial Health

65
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
60

participants targeted

Target at P25-P50 for all trials

Timeline
12mo left

Started Sep 2026

Shorter than P25 for all trials

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

December 20, 2022

Completed
14 days until next milestone

First Posted

Study publicly available on registry

January 3, 2023

Completed
3.7 years until next milestone

Study Start

First participant enrolled

September 30, 2026

Expected
9 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 30, 2027

3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

September 30, 2027

Last Updated

April 18, 2025

Status Verified

April 1, 2025

Enrollment Period

9 months

First QC Date

December 20, 2022

Last Update Submit

April 17, 2025

Conditions

Keywords

Drug Therapy

Outcome Measures

Primary Outcomes (6)

  • Percentage of Participants and Caregivers Aware of Educational Material (EM)

    EM includes home infusion guide, infusion diary, and safety and emergency plan.

    Day 1

  • Percentage of Participants and Caregivers who Understand the EM

    EM includes home infusion guide, infusion diary, and safety and emergency plan.

    Day 1

  • Percentage of Participants and Caregivers who Utilize the EM

    EM includes home infusion guide, infusion diary, and safety and emergency plan.

    Day 1

  • Percentage of Home Infusion Nurses Aware of the EM

    EM includes home infusion guide, infusion diary, and safety and emergency plan.

    Day 1

  • Percentage of Home Infusion Nurses who Understand the EM

    EM includes home infusion guide, infusion diary, and safety and emergency plan.

    Day 1

  • Percentage of Home Infusion Nurses who Utilize the EM

    EM includes home infusion guide, infusion diary, and safety and emergency plan.

    Day 1

Other Outcomes (2)

  • Usefulness of the EM for Participants and Caregivers

    Day 1

  • Usefulness of the EM for Home Infusion Nurses

    Day 1

Study Arms (1)

VPRIV® Home Infusion

Participants diagnosed with Gaucher disease will receive VPRIV® home infusion, administered by their caregivers and home infusion nurses will participate in the survey and complete the non-nominative web-based questionnaires on Day 1.

Eligibility Criteria

Age2 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

This study will recruit Gaucher disease participants receiving VPRIV® home infusion, their caregivers and home infusion nurses who administer VPRIV® infusion at home in European countries.

You may qualify if:

  • Gaucher disease participants who receive VPRIV® for home infusion and their caregivers.
  • Home infusion nurses administering VPRIV® to participants with Gaucher disease at home.

You may not qualify if:

  • Potential respondents will be excluded if they have conflicts of interest with the survey (e.g., if they are employed by regulatory bodies, or pharmaceutical industry).

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Related Links

MeSH Terms

Conditions

Gaucher Disease

Condition Hierarchy (Ancestors)

SphingolipidosesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLipidosesLipid Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesLipid Metabolism Disorders

Study Officials

  • Study Director

    Takeda

    STUDY DIRECTOR

Central Study Contacts

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
CROSS SECTIONAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 20, 2022

First Posted

January 3, 2023

Study Start (Estimated)

September 30, 2026

Primary Completion (Estimated)

June 30, 2027

Study Completion (Estimated)

September 30, 2027

Last Updated

April 18, 2025

Record last verified: 2025-04

Data Sharing

IPD Sharing
Will share

Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

Shared Documents
STUDY PROTOCOL, SAP, ICF, CSR
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.
More information