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NCT05445323 · ClinicalTrials.gov registry record · Phase 1
Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia
A Phase 1 study of Friedreich Ataxia and Cardiomyopathy, Secondary, sponsored by Lexeo Therapeutics.
- Active
- Registry status
- Phase 1
- Development phase
- 8
- Enrollment target
- 3
- Study locations
NCT05445323: Active Phase 1 study of Friedreich Ataxia and Cardiomyopathy, Secondary, sponsored by Lexeo Therapeutics.
NCT05445323 is a Phase 1 study of Friedreich Ataxia and Cardiomyopathy, Secondary that is active but no longer recruiting, run by Lexeo Therapeutics. The registered enrollment target is 8 participants, below the 449-participant average among 14 other Friedreich Ataxia trials with a reported enrollment target (98% lower). The trial reports 3 study locations across 3 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05445323, a Phase 1 study of Friedreich Ataxia and Cardiomyopathy, Secondary, is active but no longer recruiting, sponsored by Lexeo Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 8 participants
- Enrollment target
- 3
- Study locations
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 |
What the registry record for NCT05445323 still lists
NCT05445323 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 8 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 2 conditions, with Friedreich Ataxia appearing as the primary indexed condition, and to 3 interventions - of which Low dose LX2006 is the first listed.
NCT05445323 reports a single indexed study location in California, Florida, Minnesota.
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.
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).
Who is sponsoring clinical trial NCT05445323?
This trial is sponsored by Lexeo Therapeutics, which has 6 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
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Where NCT05445323's enrollment target sits among peer trials
8 15th of 14 the lowest 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.
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