Gaucher Disease Clinical Trial
Official title:
A Survey Among Patients, Caregivers and Home Infusion Nurses Based in the European Union to Assess Their Awareness and Understanding of Educational Materials Supporting VPRIV® Infusion at Home
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.
The participants diagnosed with Gaucher disease receiving VPRIV® home infusion, their caregivers and home infusion nurses were observed in this study. This cross-sectional study will determine and assess the understanding of the educational material (EM), including infusion diary and emergency plan associated with VPRIV® home infusion in participants diagnosed with Gaucher disease, their caregivers and home infusion nurses. The study will recruit approximately 30 participants/caregivers and 30 home infusion nurses. The survey will be conducted through non-nominative web-based questionnaires that will be completed in approximately 10-15 minutes. All participants will be enrolled in a single observational group: • VPRIV® Home Infusion The trial will be conducted in home-based setting in Austria, Belgium, Finland, France, Germany, Italy, the Netherlands, and Spain. ;
Status | Clinical Trial | Phase | |
---|---|---|---|
Withdrawn |
NCT04189601 -
Complement Activation in the Lysosomal Storage Disorders
|
||
Completed |
NCT04430881 -
A National Study in Patients With Unexplained Splenomegaly
|
||
Completed |
NCT02536937 -
A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate
|
Phase 1 | |
Completed |
NCT02536911 -
A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate
|
Phase 1 | |
Completed |
NCT01411228 -
A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
|
Phase 3 | |
Terminated |
NCT04094181 -
A Study of VPRIV in Participants With Gaucher Disease Previously Treated With Other Enzyme Replacement Therapies or Substrate Reduction Therapies
|
||
Completed |
NCT00391625 -
Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)
|
Phase 1/Phase 2 | |
Completed |
NCT03625882 -
Survey Study for Velaglucerase Alfa (VPRIV) in Japan
|
||
Active, not recruiting |
NCT05526664 -
Omics Gaucher Study: Multiomic Approach
|
||
Completed |
NCT02536755 -
Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies
|
Phase 3 | |
Recruiting |
NCT01344096 -
Thrombocytopathy in Gaucher Disease Patients
|
N/A | |
Completed |
NCT01881633 -
A Study of the Tolerability, Safety, and Pharmacokinetics of ISU302 in Healthy Volunteers
|
Phase 1 | |
Recruiting |
NCT06116071 -
Biomarkers Related to Bone in Pediatric Gaucher Disease
|
||
Recruiting |
NCT01951989 -
Intra-monocyte Imiglucerase Kinetics in Gaucher Disease
|
Phase 2 | |
Completed |
NCT00258778 -
Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD)
|
Phase 1 | |
Recruiting |
NCT04388969 -
World Data on Ambroxol for Patients With GD and GBA Related PD
|
||
Recruiting |
NCT05992532 -
GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma
|
||
Terminated |
NCT04145037 -
Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease
|
Phase 1/Phase 2 | |
Completed |
NCT00302146 -
Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations
|
||
Active, not recruiting |
NCT02605603 -
SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease
|