GABA and GSH in FRDA

Part of paid clinical trials in Philadelphia, Pennsylvania.

Sponsor
Children's Hospital of Philadelphia
Study ID
NCT07635030
Status
Enrolling By Invitation

Conditions

  • FRDA
  • Friedreich's Ataxia

Eligibility Criteria

Sex
ALL
Age
8 Years - 16 Years
Healthy Volunteers
Not accepted

Interventions

  • MRI/ MRS (Magnetic Resonance Imaging /Magnetic Resonance Spectroscopy) — OTHER
    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

Study Details

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.

Key Dates

Start date
May 8, 2024
Status verified
Jun 2026
Primary completion
Jun 30, 2028
Completion
Jun 30, 2030

Study Design

Enrollment
60 participants (estimated)

Arms

  • Arm: Control
    Neurotypical (NT) children aged 8 \<16 years old
  • Arm: Children with Friedreich's Ataxia (FRDA)
    Children with Friedreich's Ataxia (FRDA) aged 8 \<16 years old

Primary Outcome Measure

GABA Assessment [ Time Frame: 3 years ]

Locations (1)

FacilityCityStateZIPSite coordinators
The Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-

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By research site
The Children's Hospital of Philadelphia· Philadelphia, PA

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