New biomarker study aims to track Friedreich's ataxia treatments
NCT ID NCT06496451
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study looked at 10 adults with Friedreich's ataxia to find a way to measure frataxin mRNA in blood and spinal fluid. The goal was to create a tool that can tell if treatments are working to increase frataxin levels in the brain. Researchers used tiny particles called exosomes to detect the mRNA. This is a first step toward better monitoring of future therapies.
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Study facts
What this study's own registry entry says, in plain language.
- Participants
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10 people
The number who actually took part.
- Started
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Aug 2024
- Finished
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Jan 2026
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
People living with Friedreich ataxia (FRDA)
- Ages
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18 to 65 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Patients with symptomatic Friedreich ataxia (men and women) related to documented biallelic GAA expansion in the FXN gene * Age over 18 years * No contraindication to spinal tap including any coagulopathy or local infections * Competent to provide informed consent Exclusion Criteria: * Unable or unwilling to provide informed consent * Any uncontrolled medical illness that may increase the risk of spinal tap such as ongoing infection as determined by the investigators * FRDA related to an expansion in one allele and a conventional mutation on the other * Positive pregnancy screening
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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University of Florida
Gainesville, Florida, 32608, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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