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NCT04649866 · ClinicalTrials.gov registry record · NA
Coronary Artery Disease in Patients With Friedreich's Ataxia
A NA study, sponsored by University of Pittsburgh.
- Completed
- Registry status
- NA
- Development phase
- 7
- Enrollment target
NCT04649866: Completed NA study, sponsored by University of Pittsburgh.
NCT04649866 is a NA study that has completed, run by University of Pittsburgh. The registered enrollment target is 7 participants, below the 1,404-participant average among 58,714 other NA trials with a reported enrollment target (100% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04649866, a NA study, has completed, sponsored by University of Pittsburgh.
- COMPLETED
- Registry status
- NA
- Development phase
- 7 participants
- Enrollment target
Study Summary
Friedreich's ataxia is a debilitating, inherited disease cause by mutations in a protein called frataxin (FXN). FXN is one of several proteins that controls the production of iron-sulfur clusters, molecules that are essential for energy production in our cells as well as repair of our genetic code embedded in DNA molecules. Friedreich's ataxia (FRDA) and deficiency of FXN results in a nerve disease affecting coordination and a condition called hypertrophic cardiomyopathy (HCM), marked by an abnormal thickening of the heart. Patients with HCM can then develop pulmonary hypertension (PH), a deadly condition of the blood vessels of the lung. While most of the research in FRDA has focused on nerves and heart muscle, alterations in blood vessels of the heart and lung may worsen disease in FRDA. But, the role of FXN in these blood vessels has never been defined. Investigators pilot data suggest that Frataxin (FXN ) deficiency can control senescence and downstream function in various types of Endothelial cells (ECs), investigators hypothesize that Friedreich's Ataxia (FRDA) patients may demonstrate endothelial cells EC abnormalities throughout the vasculature potentially before overt cardiomyopathy develops.
Primary Outcome
We will quantify vasomotor tone in regard to level of vasoconstriction and vasodilation at rest and in response to pharmacologic stimulation
Interventions
- DIAGNOSTIC_TEST plethysmography
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 7 participants |
| Start Date | 2021-02-15 |
| Est. Completion | 2023-09-01 |
| Phase | NA |
What the finished NCT04649866 record still lists
NCT04649866 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 7 participants, a relatively small participant target, below the 1,404-participant average among 58,714 other NA trials with a reported enrollment target (100% lower).
The record links to 0 conditions, and to 1 intervention - of which plethysmography is the first listed.
NCT04649866 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04649866 about?
NCT04649866 is a clinical study titled "Coronary Artery Disease in Patients With Friedreich's Ataxia". Friedreich's ataxia is a debilitating, inherited disease cause by mutations in a protein called frataxin (FXN). FXN is one of several proteins that controls the production of iron-sulfur clusters, molecules that are essential for energy production in our cells as well as repair of our genetic code e...
What is the current status of trial NCT04649866?
This trial is currently completed. It is a NA study. The enrollment target is 7 participants. The study started on 2021-02-15. Estimated completion is 2023-09-01.
What interventions are being tested in trial NCT04649866?
The interventions under investigation include: plethysmography (DIAGNOSTIC_TEST).
Who is sponsoring clinical trial NCT04649866?
This trial is sponsored by University of Pittsburgh, which has 1,238 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04649866's enrollment target sits among peer trials
7 1982nd of 2000 higher than 18 of 2,000 other NA trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other NA 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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