Observational Study of Extracellular Vesicles in Gaucher Disease
This is an observational study looking at Gaucher disease. Researchers want to understand how problems with lysosomes (small parts of cells that break down waste) might affect tiny particles called extracellular vesicles (EVs). They are studying the quantity and size of these EVs in people with Gaucher disease, carriers of the Gaucher gene, and healthy individuals. The goal is to see if changes in EVs could help identify new ways to understand and track Gaucher disease, especially since EVs can cross into the brain. They will measure EV quantity at the start and after 3 months, and EV size at the start. You can join if you are between 18 and 80 years old, have a confirmed Gaucher diagnosis, are a confirmed carrier, or are a healthy control without the GBA1 gene mutation. The study is currently recruiting about 30 participants.
- Study design
- This is an observational study with no intervention, meaning participants will not receive any new treatments. It plans to enroll about 30 participants.
- What's involved
- EV quantity will be measured at baseline and again at 3 months. EV size will be measured at baseline.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for at least 3 months, based on the endpoint measurements.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease
At a glance
Conditions
NCT05843552
Where you'd take part
This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
University of Minnesota
Minneapolis, Minnesotastudy coordinator listed
Recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Study leadership
- Reena Kartha, PhD, MS · PRINCIPAL_INVESTIGATOR · University of Minnesota
- Subbaya Subramanian, PhD, MS · PRINCIPAL_INVESTIGATOR · University of Minnesota
Who to contact
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Inclusion
Exclusion
What this trial measures
- EVs quantitybaseline
Examine EV quantities isolated from plasma samples collected from patients with GD and carriers and compare to healthy individuals.
- EVs quantity3months
Examine EV quantities isolated from plasma samples collected from patients with GD and carriers and compare to healthy individuals.
- EVs sizebaseline
Examine EV sizes isolated from plasma samples collected from patients with GD and carriers and compare to healthy individuals.
- EVs size3months
Examine EV sizes isolated from plasma samples collected from patients with GD and carriers and compare to healthy individuals.
- EVs contentbaseline
Examine contents in vesicles isolated from plasma samples collected from patients with GD and carriers and compare to healthy individuals.
- EVs content3months
Examine contents in vesicles isolated from plasma samples collected from patients with GD and carriers and compare to healthy individuals.