Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia
NCT05445323 · View on ClinicalTrials.gov ↗
Study Summary
This is a Phase 1/2, open-label, dose-ascending, multicenter study of the safety and efficacy of LX2006 for participants who have Friedreich's Ataxia with evidence of cardiomyopathy. The study will evaluate up to three doses of single administration of LX2006 (AAVrh.10hFXN), an adeno-associated virus (AAV) gene therapy designed to intravenously deliver the human frataxin (hFXN) gene to cardiac cells over a 52-week period. Long-term safety and efficacy will be evaluated for an additional 4-years for a total of 5-years post LX2006 treatment.
Conditions Studied
Interventions
- GENETIC Low dose LX2006
- GENETIC Mid Dose LX2006
- GENETIC High Dose LX2006
Study Locations (3)
California
- Ataxia Center and HD Center of Excellence, University of California — Los Angeles
Florida
- University of South Florida — Tampa
Minnesota
- Mayo Clinic — Rochester
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 8 participants |
| Start Date | 2022-08-24 |
| Est. Completion | 2029-09 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05445323
The ClinicalTrials.gov registry entry for NCT05445323 describes a study currently listed as active not recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 8 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Lexeo Therapeutics, which has 5 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 2 conditions, with Friedreich Ataxia appearing as the primary indexed condition, and to 3 interventions — of which Low dose LX2006 is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05445323 reports 3 study locations spanning 3 distinct geographic areas — top geographies include California, Florida, Minnesota. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT05445323 about?
NCT05445323 is a clinical study titled "Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia". This is a Phase 1/2, open-label, dose-ascending, multicenter study of the safety and efficacy of LX2006 for participants who have Friedreich's Ataxia with evidence of cardiomyopathy. The study will evaluate up to three doses of single administration of LX2006 (AAVrh.10hFXN), an adeno-associated viru...
What is the current status of trial NCT05445323?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 8 participants. The study started on 2022-08-24. Estimated completion is 2029-09.
What conditions does trial NCT05445323 study?
This clinical trial studies the following conditions: Friedreich Ataxia, Cardiomyopathy, Secondary. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05445323?
The interventions under investigation include: Low dose LX2006 (GENETIC), Mid Dose LX2006 (GENETIC), High Dose LX2006 (GENETIC). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05445323?
This trial is sponsored by Lexeo Therapeutics, which has 5 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT05445323 being conducted?
This trial has 3 study locations across California, Florida, Minnesota. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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