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NCT05843552 · ClinicalTrials.gov registry record
Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease
A clinical trial of Gaucher Disease, sponsored by University of Minnesota.
- Recruiting
- Registry status
- 30
- Enrollment target
- 1
- Study location
NCT05843552: Recruiting study of Gaucher Disease, sponsored by University of Minnesota.
NCT05843552 is a study of Gaucher Disease that is actively recruiting participants, run by University of Minnesota. The registered enrollment target is 30 participants, below the 1,056-participant average among 13 other Gaucher Disease trials with a reported enrollment target (97% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05843552, a study of Gaucher Disease, is actively recruiting participants, sponsored by University of Minnesota.
- RECRUITING
- Registry status
- 30 participants
- Enrollment target
- 1
- Study location
Study Summary
This is an observational study intended to generate preliminary data to understand how lysosomal dysfunction can affect the biogenesis of extracellular vesicles, its content and function. The primary objective of the proposed project is to decipher how extracellular vesicle (EV) biogenesis and its role in intercellular communication can be impaired as a consequence of defects in lysosomal function. Collectively these defects in EV biogenesis and function can contribute to the neuroinflammation observed in lysosomal storage diseases. Since EVs can cross the blood-brain barrier, their characterization may be valuable in identifying novel biomarkers. In the presence of a GBA1 mutation, the decrease in GCase activity will lower overall lysosome function and increase the secretion of EVs. Further, there will be differences in EV size, its cargo including lipids, RNA and proteins and their aggregates. In comparison to healthy controls, EVs isolated from patients with Gaucher disease (GD) and GBA1 carriers is hypothesized to show significant differences in terms of its characteristics and content, which can contribute to our understanding of the link between lysosomes and neurological disease.
Primary Outcome
Examine EV quantities isolated from plasma samples collected from patients with GD and carriers and compare to healthy individuals.
Conditions Studied
Interventions
- OTHER no intervention
Study Locations (1)
Minnesota
- University of Minnesota - Minneapolis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 30 participants |
| Start Date | 2023-04-30 |
| Est. Completion | 2026-03-31 |
What NCT05843552 shows while recruiting
NCT05843552 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 30 participants, a relatively small participant target, below the 1,056-participant average among 13 other Gaucher Disease trials with a reported enrollment target (97% lower).
The record links to 1 condition, with Gaucher Disease appearing as the primary indexed condition, and to 1 intervention - of which no intervention is the first listed.
NCT05843552 reports a single indexed study location in Minnesota.
Frequently Asked Questions
What is clinical trial NCT05843552 about?
NCT05843552 is a clinical study titled "Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease". This is an observational study intended to generate preliminary data to understand how lysosomal dysfunction can affect the biogenesis of extracellular vesicles, its content and function. The primary objective of the proposed project is to decipher how extracellular vesicle (EV) biogenesis and its r...
What is the current status of trial NCT05843552?
This trial is currently recruiting. The enrollment target is 30 participants. The study started on 2023-04-30. Estimated completion is 2026-03-31.
What conditions does trial NCT05843552 study?
This clinical trial studies the following conditions: Gaucher Disease.
What interventions are being tested in trial NCT05843552?
The interventions under investigation include: no intervention (OTHER).
Who is sponsoring clinical trial NCT05843552?
This trial is sponsored by University of Minnesota, which has 966 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05843552 being conducted?
This trial has 1 study location across Minnesota. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Gaucher Disease
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT05843552's enrollment target sits among peer trials
30 8th of 13 higher than 5 of 13 other Gaucher Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Gaucher Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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