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GABA and GSH in FRDA
Sponsor: Children's Hospital of Philadelphia
Summary
The goal of this study is to obtain gamma-aminobutyric acid (GABA) and glutathione (GSH) assessment derived from magnetic resonance spectroscopy (MRS), to be used as a potential biomarker in patients with Friedreich Ataxia (FRDA) prior to (Aim 1), and after taking Omaveloxolone (Aim 2). Analysis will consist of: A. Comparison of values in controls with those of FRDA patients (Aim 1) B. Longitudinal comparison of values in FRDA patients repeated after Omaveloxolone administration at 3 time points (minimum of 6 months) (Aim 2) FRDA participants will be asked to complete an MRS scan at 3 timepoints in order to observe GABA and GSH activity.
Official title: Magnetic Resonance Spectroscopy (MRS) Estimates of Glutathione (GSH) and GABA as Biomarkers of Pathophysiology in FRDA
Key Details
Gender
All
Age Range
8 Years - 16 Years
Study Type
OBSERVATIONAL
Enrollment
60
Start Date
2024-05-08
Completion Date
2030-06
Last Updated
2026-06-09
Healthy Volunteers
No
Conditions
Interventions
MRI/ MRS (Magnetic Resonance Imaging /Magnetic Resonance Spectroscopy)
Subjects will undergo an MRI scan wherein the investigator will use a published, but recently developed, MRS protocol (HERMES) for simultaneous assessment of GABA and glutathione (GSH) in a single scan using a 3T MR scanner
Locations (1)
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States