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ENROLLING BY INVITATION
NCT07635030

GABA and GSH in FRDA

Sponsor: Children's Hospital of Philadelphia

View on ClinicalTrials.gov

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

Interventions

OTHER

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