NCT07635030Enrolling by invitation
GABA and GSH in FRDA
Magnetic Resonance Spectroscopy (MRS) Estimates of Glutathione (GSH) and GABA as Biomarkers of Pathophysiology in FRDA
Source: ClinicalTrials.gov · Last updated 9 Jun 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Enrolling by invitation
- Study type
- Observational
- Enrollment
- 60 (estimated)
- First submitted
- 1 Jun 2026
- Start date
- 8 May 2024
- Primary completion date
- Jun 2028
- Lead sponsor
- Children's Hospital of Philadelphia
- Diseases
- Friedreich ataxia
- NCT number
- NCT07635030
What you commit to
- Study type: Observational
- Planned participants: 60
- From 8 May 2024 to the primary completion date, Jun 2028
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- MR contraindications (e.g., pacemaker or other metallic surgical implants)
Other illness
- 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
Age
- Age \< 8 years \> 16 years
Other
- 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
Who the study is looking for
- 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
Age in the registry: 8 years to 16 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| The Children's Hospital Of Philadelphia Coordinator of this site? Confirm its status | Philadelphia | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.