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
| Verified date | March 2024 |
| Source | Takeda |
| Contact | Takeda Contact |
| Phone | +1-877-825-3327 |
| medinfoUS[@]takeda.com | |
| Is FDA regulated | No |
| Health authority | |
| Study type | Observational |
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.
| Status | Not yet recruiting |
| Enrollment | 60 |
| Est. completion date | September 30, 2026 |
| Est. primary completion date | September 30, 2026 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 2 Years and older |
| Eligibility | Inclusion criteria: 1. Gaucher disease participants who receive VPRIV® for home infusion and their caregivers. 2. Home infusion nurses administering VPRIV® to participants with Gaucher disease at home. Exclusion criteria: 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). |
| Country | Name | City | State |
|---|---|---|---|
| n/a | |||
| Lead Sponsor | Collaborator |
|---|---|
| Takeda |
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Other | Usefulness of the EM for Participants and Caregivers | EM includes home infusion guide, infusion diary, and safety and emergency plan. Usefulness will be assessed using the 12-item participant and caregiver questionnaire, and 15-item home infusion nurse questionnaire. | Day 1 | |
| Other | Usefulness of the EM for Home Infusion Nurses | EM includes home infusion guide, infusion diary, and safety and emergency plan. Usefulness will be assessed using the 12-item participant and caregiver questionnaire, and 15-item home infusion nurse questionnaire. | Day 1 | |
| Primary | Percentage of Participants and Caregivers Aware of Educational Material (EM) | EM includes home infusion guide, infusion diary, and safety and emergency plan. | Day 1 | |
| Primary | Percentage of Participants and Caregivers who Understand the EM | EM includes home infusion guide, infusion diary, and safety and emergency plan. | Day 1 | |
| Primary | Percentage of Participants and Caregivers who Utilize the EM | EM includes home infusion guide, infusion diary, and safety and emergency plan. | Day 1 | |
| Primary | Percentage of Home Infusion Nurses Aware of the EM | EM includes home infusion guide, infusion diary, and safety and emergency plan. | Day 1 | |
| Primary | Percentage of Home Infusion Nurses who Understand the EM | EM includes home infusion guide, infusion diary, and safety and emergency plan. | Day 1 | |
| Primary | Percentage of Home Infusion Nurses who Utilize the EM | EM includes home infusion guide, infusion diary, and safety and emergency plan. | Day 1 |
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Withdrawn |
NCT04189601 -
Complement Activation in the Lysosomal Storage Disorders
|
||
| 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 |
NCT04430881 -
A National Study in Patients With Unexplained Splenomegaly
|
||
| 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
|