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NCT04718779 · ClinicalTrials.gov registry record · Phase 4
A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy
A Phase 4 study, sponsored by Takeda.
- Completed
- Registry status
- Phase 4
- Development phase
- 4
- Enrollment target
NCT04718779 is a Phase 4 study that has completed, run by Takeda. The registered enrollment target is 4 participants.
The verdict
NCT04718779, a Phase 4 study, has completed, sponsored by Takeda.
- COMPLETED
- Registry status
- Phase 4
- Development phase
- 4 participants
- Enrollment target
Study Summary
The study will provide information on outcomes in people with type 1 Gaucher disease when they are treated with velaglucarase alfa (also called VPRIV), under standard care. Standard care means the participant will be treated according to the clinic's standard practice. The study sponsor will not be involved in how participants are treated with VPRIV, will provide instructions on how the clinic will record what happens during the study. VPRIV is a type of enzyme replacement therapy (also known as ERT). Before starting the study, participants must either have switched from substrate reduction therapies (SRT) to VPRIV or switched from other enzyme replacement therapies to SRT then finally to VPRIV. During this time, medical data will be collected from the participants' medical records. During the study, participants will be treated with VPRIV according to their clinic's standard practice. VPRIV is given by a slow injection into the vein, also known as an infusion. This will happen in the clinic or at home. The study will record if blood levels of specific substances remain stable or improve during the switch to treatment with VPRIV. Some of these substances will show if organs such as the liver or spleen are working well. Others are blood cells that help blood to clot, known as platelets. Another is a substance in a red blood cell used to carry oxygen around the body, known as hemoglobin. Participants will use a digital tool so they can be more involved in decision making in their treatment. The digital tool is a mobile phone app, in which each participant can log their daily activities, their general health and wellbeing, and other key information. Medical data will also be collected from the participants' charts during this time. Health problems of the participants will be recorded during the study to check if there were any side effects from VPRIV treatment. Participants will be in this study for up to 12 months.
Interventions
- OTHER No Intervention
- OTHER Digital Engagement Application (GD App)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 4 participants |
| Start Date | 2021-04-22 |
| Est. Completion | 2023-02-16 |
| Phase | Phase 4 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04718779
The ClinicalTrials.gov registry entry for NCT04718779 describes a study currently listed as completed, categorized as Phase 4. The registered enrollment target is 4 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Takeda, which has 408 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 2 interventions - of which No Intervention is the first listed.
NCT04718779 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT04718779 about?
NCT04718779 is a clinical study titled "A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy". The study will provide information on outcomes in people with type 1 Gaucher disease when they are treated with velaglucarase alfa (also called VPRIV), under standard care. Standard care means the participant will be treated according to the clinic's standard practice. The study sponsor will not be ...
What is the current status of trial NCT04718779?
This trial is currently completed. It is a Phase 4 study. The enrollment target is 4 participants. The study started on 2021-04-22. Estimated completion is 2023-02-16.
What interventions are being tested in trial NCT04718779?
The interventions under investigation include: No Intervention (OTHER), Digital Engagement Application (GD App) (OTHER).
Who is sponsoring clinical trial NCT04718779?
This trial is sponsored by Takeda, which has 408 total clinical trials registered on ClinicalTrials.gov.
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