MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3)

Part of paid clinical trials in Minneapolis, Minnesota.

Sponsor
University of Minnesota
Study ID
NCT05586243
Status
Recruiting
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Conditions

  • Gaucher Disease, Type 3

Eligibility Criteria

Sex
ALL
Age
18 Years - 80 Years
Healthy Volunteers
Not accepted

Interventions

  • No intervention — OTHER
    This is an observational study looking at brain chemicals related to oxidative stress and inflammation using magnetic resonance spectroscopy (MRS).

Study Details

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.

Key Dates

Start date
Jan 1, 2023
Status verified
Jan 2026
Primary completion
Jul 31, 2026
Completion
Jul 31, 2026

Study Design

Enrollment
5 participants (estimated)

Primary Outcome Measure

Neurometabolite profile [ Time Frame: baseline ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
University of MinnesotaMinneapolisMinnesota55455
Reena V. Kartha, Ph.D
[email protected]
612-626-2436
Reena Kartha, Ph.D (PRINCIPAL_INVESTIGATOR)
Gulin Oz, Ph.D (SUB_INVESTIGATOR)
Kyle Rudser, Ph.D (SUB_INVESTIGATOR)
Jaehyeok Roh, Pharm.D (SUB_INVESTIGATOR)

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