NCT05445323Active, not recruitingPhase 1/2
Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia
A Phase 1/2 Study of the Safety and Efficacy of LX2006 Gene Therapy in Participants With Cardiomyopathy Associated With Friedreich's Ataxia
Source: ClinicalTrials.gov · Last updated 23 Dec 2025
Brief summary (registry, in English)
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.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 8 (actual)
- First submitted
- 23 Jun 2022
- Start date
- 24 Aug 2022
- Primary completion date
- 30 Sept 2029
- Lead sponsor
- Lexeo Therapeutics
- Diseases
- Friedreich ataxia
- NCT number
- NCT05445323
What you commit to
- Study type: Interventional · Phase 1/2
- Participants enrolled: 8
- From 24 Aug 2022 to the primary completion date, 30 Sept 2029
- 3 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Participants who are receiving systemic corticosteroids or other immunosuppressive medications
Other illness
- Uncontrolled diabetes
- Active infection of any type, including hepatitis virus (A, B or C) or human immunodeficiency virus (HIV-1 and HIV-2)
- Contraindication to cardiac MRI
- Contraindications to cardiac biopsies
- History of significant coronary artery disease or any structural heart or vascular disease other than FA cardiomyopathy
- Presence of clinically significant, hemodynamically unstable arrhythmias, requiring physician intervention
- Uncontrolled psychiatric disease
- Other Inclusion/Exclusion criteria to be applied as per protocol.
Lab values
- Protocol specified ranges for left ventricular ejection fraction (LVEF) as measured by cardiac ECHO
Other
- Abnormal liver function
- Presence of clinically significant abnormalities as determined by the investigator, other than ECG abnormalities related to FA
Who the study is looking for
- Confirmed genetic diagnosis of FA, with onset being before 25 years of age
- Protocol specified ranges for antibodies
- Protocol specified measures of FA cardiomyopathy
Age in the registry: 18 years to 50 years · Sex: All · Does not accept healthy volunteers
Sites (3)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Ataxia Center and HD Center of Excellence, University of California Coordinator of this site? Confirm its status | Los Angeles | United States | Unknown status |
| Mayo Clinic Coordinator of this site? Confirm its status | Rochester | United States | Unknown status |
| University of South Florida Coordinator of this site? Confirm its status | Tampa | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.