A Phase 2 interventional study of LX2006 and Usual Care in Friedreich Ataxia and Cardiomyopathy, Secondary, sponsored by Lexeo Therapeutics. Recruiting at 1 site in United States. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2026-09-02.
Sponsored by Lexeo Therapeutics · Phase 2, Interventional, and Treatment
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).
110 studies on the registry are indexed under Friedreich Ataxia; 27 are open to participants now.
This study's planned enrollment of 26 is below the median of 30 across 75 interventional studies indexed under Friedreich Ataxia.
Browse Friedreich Ataxia studies →Lexeo Therapeutics is the lead sponsor of 8 studies on the registry; 4 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Other inclusion/exclusion criteria to be applied as per protocol.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HbA1c
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cohort 1: Participants ≥16 years of age with FA-CM Cohort 2: Participants ≥6 to \<16 years of age with FA-CM. As Cohort 2 will not be randomized, all participants will receive LX2006 upon enrollment. Participants in Cohort 2 will be enrolled after safety is assessed in a group of participants in Cohort 1.
Genetic: LX2006
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).
Other: Usual Care
Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)
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).
Cohort 1: Percent change from baseline in left ventricular mass index by cardiac MRI
Time frame: At Week 26
Cohort 2: Incidence and severity of treatment-emergent adverse events
Time frame: Through Month 60
Cohort 2: Change in clinical routine safety laboratory tests as assessed by standardized clinical laboratory reference ranges
Time frame: Through Month 60
Cohort 2: Change in clinical vital signs (body temperature [°C])
Time frame: Through Month 60
Cohort 2: Change in clinical vital signs (systolic and diastolic blood pressure [mmHg])
Time frame: Through Month 60
Cohort 2: Change in clinical vital signs (heart rate [beats per minute])
Time frame: Through Month 60
Cohort 2: Change in clinical vital signs (respiratory rate [breaths per minute])
Time frame: Through Month 60
Cohort 2: Change in clinical vital signs (oxygen saturation [%])
Time frame: Through Month 60
Cohort 2: Change in clinical 12-lead ECG findings (ECG machine automatically calculates the heart rate [beats per minute] and measures PR, QT, QT interval corrected using Fridericia's method intervals, and QRS complex [milliseconds])
Time frame: Through Month 60
Cohorts 1 and 2: Change from baseline in high-sensitivity troponin I
Time frame: At Week 26
Cohort 1: Change from baseline in maximal wall thickness by cardiac MRI
Time frame: At Week 26
Cohort 1: Number of the following cardiovascular events as collected from the participant by the study doctor during scheduled study visits
* Death due to any cause * Hospitalization for heart failure * Non-fatal stroke * Non-fatal heart attack * Non-fatal life-threatening rapid or irregular heartbeat * Heart transplant * Insertion of a left ventricular assist device (pump) in the heart
Time frame: At Month 60
Cohort 1: Change from baseline in modified Friedreich's Ataxia Rating Scale
A neurological assessment tool for Friedreich ataxia that is measured on a scale of 0 to 93 with high scores indicating greater physical impairment
Time frame: At Week 26, at Week 52, and through Month 60
Cohort 1: Change from baseline in Kansas City Cardiomyopathy Questionnaire
A cardiac assessment tool scored from 0 to 100, with higher scores indicating better health status and lower scores reflecting more severe heart failure symptoms and functional limitations
Time frame: At Week 26, at Week 52, and through Month 60
Cohort 1: Incidence and severity of treatment-emergent adverse events
Time frame: Through Month 60
Cohort 1: Change in clinical routine safety laboratory tests as assessed by standardized clinical laboratory reference ranges
Time frame: Through Month 60
Cohort 1: Change in clinical vital signs (body temperature [°C])
Time frame: Through Month 60
Cohort 1: Change in clinical vital signs (systolic and diastolic blood pressure [mmHg])
Time frame: Through Month 60
Cohort 1: Change in clinical vital signs (heart rate [beats per minute])
Time frame: Through Month 60
Cohort 1: Change in clinical vital signs (respiratory rate [breaths per minute])
Time frame: Through Month 60
Cohort 1: Change in clinical vital signs (oxygen saturation [%])
Time frame: Through Month 60
Cohort 1: Change in clinical 12-lead ECG findings
ECG machine automatically calculates the heart rate \[beats per minute\] and measures PR, QT, QT interval corrected using Fridericia's method intervals, and QRS complex \[milliseconds\]
Time frame: Through Month 60
Cohort 2: Percent change from baseline in left ventricular mass index by cardiac MRI (or by ECHO for participants aged ≥6 to <12 years who did not have the cardiac MRI at baseline)
Time frame: At Week 26
Cohort 2: Change from baseline in maximal wall thickness by cardiac MRI (or by ECHO for participants aged ≥6 to <12 years who did not have the cardiac MRI at baseline)
Time frame: At Week 26
Started Jun 2026 (actual). Primary completion is estimated for Jun 2032, 72 months after the start.
Of 12 phase 2 interventional studies under Friedreich Ataxia, half reached primary completion within 22 months of starting (the median), counting those still running; a quarter within 14 and three quarters within 34. This study's planned 72 months is above that.
How these figures are worked out, and which studies are left out of them.
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Lexeo Therapeutics