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NCT06496451 · ClinicalTrials.gov registry record
Measurement of Frataxin mRNA in Biofluids
A clinical trial of Friedreich Ataxia, sponsored by University of Florida.
- Completed
- Registry status
- 10
- Enrollment target
- 1
- Study location
NCT06496451: Completed study of Friedreich Ataxia, sponsored by University of Florida.
NCT06496451 is a study of Friedreich Ataxia that has completed, run by University of Florida. The registered enrollment target is 10 participants, below the 449-participant average among 14 other Friedreich Ataxia trials with a reported enrollment target (98% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06496451, a study of Friedreich Ataxia, has completed, sponsored by University of Florida.
- COMPLETED
- Registry status
- 10 participants
- Enrollment target
- 1
- Study location
Study Summary
The purpose of this research study is to determine a way to measure frataxin messenger RNA (mRNA) in fluids such as blood and cerebrospinal fluid (CSF) from patients with Friedreich's ataxia (FRDA). The gene mutation in FRDA leads to low levels of the mRNA and then low levels of the protein frataxin that leads to the disease. Treatments being developed for FRDA have the ability increase these levels including in brain where it is needed. Currently, there is no accepted way to measure frataxin protein or the messenger RNA (from which the protein is made) in the spinal fluid that surrounds the brain. In our study, the investigators aim to measure frataxin mRNA in both the blood and CSF. The investigators will use our ability to isolate structures called exosomes from these fluids. Exosomes are tiny, microscopic sacs that are known to contain many important biological molecules, and the investigators are able to detect frataxin mRNA in CSF from patients with other illnesses and from non-diseased participants. The investigators believe that parallel studies of exosomes in blood and CSF from patients with FRDA can tell us as to whether the frataxin mRNA in the CSF or blood of FRDA patients can serve as a measure of frataxin production in the brain. With one participation visit the investigators will be able to study the relationship of frataxin mRNA levels in the participant\'s CSF and blood with measures of disease severity. If successful, this will provide an important tool to monitor treatments for FRDA that aim to increase frataxin production.
Primary Outcome
The study plans to measure frataxin mRNA in exosomes from blood and spinal fluid as a biomarker in Friedreich ataxia
Conditions Studied
Interventions
- OTHER Spinal tap for CSF collection
- OTHER Venipuncture for blood sample
Study Locations (1)
Florida
- University of Florida - Gainesville
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2024-08-08 |
| Est. Completion | 2026-01-14 |
What the finished NCT06496451 record still lists
NCT06496451 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 449-participant average among 14 other Friedreich Ataxia trials with a reported enrollment target (98% lower).
The record links to 1 condition, with Friedreich Ataxia appearing as the primary indexed condition, and to 2 interventions - of which Spinal tap for CSF collection is the first listed.
NCT06496451 reports a single indexed study location in Florida.
Frequently Asked Questions
What is clinical trial NCT06496451 about?
NCT06496451 is a clinical study titled "Measurement of Frataxin mRNA in Biofluids". The purpose of this research study is to determine a way to measure frataxin messenger RNA (mRNA) in fluids such as blood and cerebrospinal fluid (CSF) from patients with Friedreich's ataxia (FRDA). The gene mutation in FRDA leads to low levels of the mRNA and then low levels of the protein frataxin...
What is the current status of trial NCT06496451?
This trial is currently completed. The enrollment target is 10 participants. The study started on 2024-08-08. Estimated completion is 2026-01-14.
What conditions does trial NCT06496451 study?
This clinical trial studies the following conditions: Friedreich Ataxia.
What interventions are being tested in trial NCT06496451?
The interventions under investigation include: Spinal tap for CSF collection (OTHER), Venipuncture for blood sample (OTHER).
Who is sponsoring clinical trial NCT06496451?
This trial is sponsored by University of Florida, which has 1,106 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06496451 being conducted?
This trial has 1 study location across Florida. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Friedreich Ataxia
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT06496451's enrollment target sits among peer trials
10 14th of 14 higher than 1 of 14 other Friedreich Ataxia trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Friedreich Ataxia trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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Clinical Course Of Disease In Participants With FA-CM
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