Who may be eligible for the GABA and GSH in FRDA trial?

Inclusion Criteria: * Age ≥ 8 years; \ 55 in intron 1 of Frataxin (FXN) and/or GAA repeat length \> 55 in intron 1 of FXN in one allele and another type of mutation that is inferred to cause loss of function in the second FXN allele as documented in the medical record * Friedreich's Ataxia Rating Scale (FARS) Functional staging score of ≤ 5\^ and total modified Friedreich's Ataxia Rating Scale (mFARS) score of ≤ 65 on enrolment Exclusion Criteria: * Age \ 16 years * Acute or ongoing medical or other conditions that is deemed to interfere with the conduct and assessments of the study * Other psychiatric or neurologic conditions apart from FRDA that, in the opinion of the Site Investigator, would interfere with the conduct and assessments of the study * MR contraindications (e.g., pacemaker or other metallic surgical implants) * Presence of metallic dental braces * Currently pregnant participants * Confined to wheelchair or bed with total dependency for all activities of daily living. Total disability. * Unable to understand English instruction

ClinicalTrials.gov record NCT07635030

GABA and GSH in FRDA

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 7, 2026

Magnetic Resonance Spectroscopy (MRS) Estimates of Glutathione (GSH) and GABA as Biomarkers of Pathophysiology in FRDA

About this page. This page shows the study's official ClinicalTrials.gov record ↗, with added help to make it easier to understand. We explain medical terms, highlight key details in plain language, and suggest questions you may want to ask the study team.

The record is periodically copied from ClinicalTrials.gov and may lag behind changes. AI summaries are checked by software, not a clinician.Read moreShow less

The official record is written by the study sponsor and published on ClinicalTrials.gov. It may not always reflect the latest changes. Before making any decisions, please confirm important information with the study team and your own healthcare team.

Where you see "AI summary", AI was used to create a plain-language explanation and software checked it against the source. It has not been reviewed by a clinician, so it may contain mistakes or leave out important details.

StatusEnrolling by invitationThe study enrols only people the study team invites, for example patients already at a particular clinic. It is not open to the public.

Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.

Age8 years old to 16 years old

SexAll

LocationUnited States

AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This observational, case-control study uses magnetic resonance spectroscopy (MRS) to measure brain GABA and glutathione (GSH) in children with Friedreich's ataxia and in neurotypical controls. FRDA participants (ages 8 up to but not including 16, and described as naive to Omaveloxolone) will be asked to complete MRS scans at three time points. Values from FRDA participants will be compared with controls and examined before and after Omaveloxolone administration as described in the record.

What it is studying. The study tests whether MRS-derived measures of GABA and GSH (and other MRS metabolites such as N-acetyl-aspartate and myo-inositol) differ between FRDA and neurotypical controls, and whether those measures change in FRDA before and after Omaveloxolone.

What the researchers hope to learn. To obtain MRS-based GABA and GSH measurements as potential biomarkers for FRDA; to compare those measures with neurotypical controls; and to assess changes in other MRS metabolites (including N-acetyl-aspartate and myo-inositol) in FRDA prior to and after Omaveloxolone.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Other: MRI/ MRS (Magnetic Resonance Imaging /Magnetic Resonance Spectroscopy)

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

Age ≥ 8 years; <16 years
Written informed consent provided
Balletic Guanine-adenine-adenine (GAA) trinucleotide repeat length > 55 in intron 1 of Frataxin (FXN) and/or GAA repeat length > 55 in intron 1 of FXN in one allele and another type of mutation that is inferred to cause loss of function in the second FXN allele as documented in the medical record
Friedreich's Ataxia Rating Scale (FARS) Functional staging score of ≤ 5\^ and total modified Friedreich's Ataxia Rating Scale (mFARS) score of ≤ 65 on enrolment

Exclusion criteria

Age < 8 years > 16 years
Acute or ongoing medical or other conditions that is deemed to interfere with the conduct and assessments of the study
Other psychiatric or neurologic conditions apart from FRDA that, in the opinion of the Site Investigator, would interfere with the conduct and assessments of the study
MR contraindications (e.g., pacemaker or other metallic surgical implants)
Presence of metallic dental braces
Currently pregnant participants
Confined to wheelchair or bed with total dependency for all activities of daily living. Total disability.
Unable to understand English instruction

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants will undergo non‑invasive MRI with magnetic resonance spectroscopy (MRS) using a HERMES protocol on a 3T scanner to measure GABA and glutathione (GSH). Children with FRDA are asked to have three MRS scans over the study (with at least a 6‑month span between timepoints); the registry does not specify the control scan schedule. The record lists a target study duration of 3 years. The registry does not describe other procedures or sampling beyond the MRI/MRS scans.

How the study is structured. Observational, prospective case-control study. Children with FRDA and neurotypical control children undergo MRI with a specialized MRS protocol (HERMES) on a 3T scanner to measure GABA and GSH. FRDA participants have scans at three time points (minimum spacing described as six months in the record) to allow comparison before and after Omaveloxolone; control participants provide comparative MRS data.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
The registry says participants are enrolled by invitation. Who decides who is invited, and how?
The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
Which of the eligibility criteria would need to be checked with tests before I could join?

More on Friedreich ataxia

Clinical trials26 trials listed for Friedreich ataxiaEvery trial the registry lists for Friedreich ataxia, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Friedreich ataxia researchLatest monthly update: September 2026. Includes: Heart change stages. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start: May 8, 2024
Primary completion: June 2028 (estimated)
Study completion: June 2030 (estimated)
Target enrollment: 60 participants (estimated)
Last updated on registry: Jun 9, 2026
Data verified: Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor: Children's Hospital of Philadelphia

Participating sites (1)

The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104 · United States

Terms used on this page · explanations by MedicalUpdates.ai

biomarker: Something measurable in the body, such as a blood test result or an imaging finding, that indicates how a condition or treatment is behaving.
Written informed consent: The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
enrolment: The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
contraindications: A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
Inclusion Criteria: The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria: The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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