NCT07721025 · Lexeo Therapeutics
Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
(SUNRISE-FA 2)
What this study is about
The purpose of Study LX2006-03, a conducted at multiple hospitals, Phase 2, where both patients and doctors know the treatment given, randomly assigned, controlled study, is to evaluate the effectiveness and safety of LX2006 gene therapy in participants with Friedreich ataxia (FA) cardiomyopathy (CM).
View original scientific description
The purpose of Study LX2006-03, a multicenter, Phase 2, open-label, randomized, controlled study, is to evaluate the efficacy and safety of LX2006 gene therapy in participants with Friedreich ataxia (FA) cardiomyopathy (CM).
Interventions
GENETIC
LX2006
Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)
OTHER
Usual Care
Cohort 1: Participants ≥16 years of age with FA-CM Participants will receive usual care for 26 weeks before receiving treatment with LX2006 (single crossover).
Primary outcome measures
Percent change from baseline in left ventricular mass index by cardiac MRI
Time frame: At Week 26
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Male or female, age at least 6 years at the time of signing the informed consent (and assent, if applicable).
- Diagnosis of FA, based on clinical phenotype and genotype (GAA expansion on the frataxin gene)
- Onset of FA on or before 25 years of age
- Confirmed left ventricular hypertrophy and abnormal left ventricular mass index
- Left ventricular ejection fraction at least 30%
- Anti-AAVrh.10 total antibody titer less than the protocol-specified maximum level
Exclusion criteria
- Presence of other forms of cardiomyopathy that contribute to heart failure
- Current use of inotrope infusion or presence of a ventricular assist device
- Contraindication to cardiac MRI
- Prior organ transplant
- Previous gene transfer or cell therapy
- Poorly controlled diabetes (hemoglobin A1c ≥8%)
- Active hematologic or solid organ cancer Other inclusion/exclusion criteria to be applied as per protocol.
Where
- Tampa, Florida
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jul 22, 2026 · Source of record for eligibility and locations