NCT05586243 MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3)
| NCT ID | NCT05586243 |
| Status | Recruiting |
| Phase | — |
| Sponsor | University of Minnesota |
| Condition | Gaucher Disease, Type 3 |
| Study Type | OBSERVATIONAL |
| Enrollment | 5 participants |
| Start Date | 2023-01-01 |
| Primary Completion | 2026-12-31 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 5 participants in total. It began in 2023-01-01 with a primary completion date of 2026-12-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
Recent studies have has shown that magnetic resonance spectroscopy (MRS) can provide validated neuronal markers in patients with Type 1 GD (GD1) who are on stable therapy. However, alterations in neurometabolites in adult patients with GD3, who have established neurological involvement, are not well understood. The goal of this study is to characterize neurometabolite profiles in adult patients with GD3 using MRS to identify novel biomarkers that can demonstrate treatment response. Additionally, a secondary aim is to evaluate relationships between neurometabolites and disease parameters, such as genotype, enzyme levels and Gaucher disease (GD) biomarkers.
Eligibility Criteria
Inclusion Criteria: * All participants must be 18 years or older. * All enrollees must understand and cooperate with requirements of the study in the opinion of the investigators and must be able to provide written informed consent. * Individuals with GD3 who are medically stable for participation in study in the opinion of the investigator. Exclusion Criteria: * Medically unstable conditions as determined by the investigators. * Concurrent disease; medical condition; or an extenuating circumstance that, in the opinion of the investigator, might compromise subject safety, study compliance, completion of the study, or the integrity of the data collected for the study. * Women who are pregnant or lactating or of child-bearing age that are not using acceptable forms of contraception. * Patients enrolled in another interventional study. * Patients who cannot or are unwilling to have blood drawn. * Inability to undergo Magnetic Resonance Imaging (MRI) scanning, including but not limited to unable to remain still in an MRI scanner for more than 30 minutes, claustrophobia, presence of paramagnetic substances or pacemakers in body, weight over 300 lbs. * Unable to adhere to study protocol for whatever reason.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT05586243 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, up to 80 Years, studying Gaucher Disease, Type 3. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT05586243 currently recruiting?
Yes, NCT05586243 is actively recruiting participants. Contact the research team at rvkartha@umn.edu for enrollment information.
Where is the NCT05586243 trial being conducted?
This trial is being conducted at Minneapolis, United States.
Who is sponsoring the NCT05586243 clinical trial?
NCT05586243 is sponsored by University of Minnesota. The trial plans to enroll 5 participants.