Friedreich AtaxiaPhase 2SUNRISE-FA 2NCT07721025
Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
A Phase 2 study testing LX2006 in people with Friedreich Ataxia and Cardiomyopathy, Secondary, run by Lexeo Therapeutics at 1 site in United States.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Friedreich Ataxia, Cardiomyopathy, Secondary
- What is being tested?
- LX2006
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 1 siteUnited States
- Study dates
- 25 Jun 2026to Jun 2032
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
University of South Florida
Tampa, Florida, 33612
Lucretia Campbell, 813-974-5633, lcampbel@usf.edu
About this study
Official title: A Phase 2, Multicenter, Open-label, Randomized, Controlled Study of LX2006 Gene Therapy in Participants With Friedreich Ataxia Cardiomyopathy (SUNRISE-FA 2)
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).
- Conditions
- Friedreich Ataxia, Cardiomyopathy, Secondary
- Being tested
- LX2006, Usual Care
- Keywords
- Friedreich Ataxia Cardiomyopathy, FA-CM, Friedreich Ataxia, Friedreich's Ataxia, FA, FRDA, Cardiomyopathy, Genetic Cardiomyopathy, Mitochondrial Cardiomyopathy, Cardiac Disease, Left Ventricular Hypertrophy, Hypertrophic Cardiomyopathy (Non-Obstructive), HCM, Gene Therapy, FXN Gene, FXN, Frataxin Gene, LX2006, SUNRISE-FA 2, SUNRISE-FA
- Medical subject headings
- Friedreich Ataxia, Cardiomyopathies, Heart Diseases, Hypertrophy, Left Ventricular, Cardiomyopathy, Hypertrophic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LX2006Genetic
Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)
Usual CareOther
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).
| Study group | Role | What they receive |
|---|---|---|
| LX2006 | Experimental | 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. Receives: Genetic: LX2006 |
| Usual Care | Other | 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). Receives: Other: Usual Care |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cohort 1: Percent change from baseline in left ventricular mass index by cardiac MRI
Measured: At Week 26
Cohort 2: Incidence and severity of treatment-emergent adverse events
Measured: Through Month 60
Cohort 2: Change in clinical routine safety laboratory tests as assessed by standardized clinical laboratory reference ranges
Measured: Through Month 60
Cohort 2: Change in clinical vital signs (body temperature [°C])
Measured: Through Month 60
Cohort 2: Change in clinical vital signs (systolic and diastolic blood pressure [mmHg])
Measured: Through Month 60
Cohort 2: Change in clinical vital signs (heart rate [beats per minute])
Measured: Through Month 60
Cohort 2: Change in clinical vital signs (respiratory rate [breaths per minute])
Measured: Through Month 60
Cohort 2: Change in clinical vital signs (oxygen saturation [%])
Measured: 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])
Measured: Through Month 60
Secondary outcomes (15)
Cohorts 1 and 2: Change from baseline in high-sensitivity troponin I
Measured: At Week 26
Cohort 1: Change from baseline in maximal wall thickness by cardiac MRI
Measured: 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
Measured: 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
Measured: 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
Measured: At Week 26, at Week 52, and through Month 60
Cohort 1: Incidence and severity of treatment-emergent adverse events
Measured: Through Month 60
Cohort 1: Change in clinical routine safety laboratory tests as assessed by standardized clinical laboratory reference ranges
Measured: Through Month 60
Cohort 1: Change in clinical vital signs (body temperature [°C])
Measured: Through Month 60
Cohort 1: Change in clinical vital signs (systolic and diastolic blood pressure [mmHg])
Measured: Through Month 60
Cohort 1: Change in clinical vital signs (heart rate [beats per minute])
Measured: Through Month 60
Cohort 1: Change in clinical vital signs (respiratory rate [breaths per minute])
Measured: Through Month 60
Cohort 1: Change in clinical vital signs (oxygen saturation [%])
Measured: 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]
Measured: 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)
Measured: 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)
Measured: At Week 26
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2026
- Study start25 Jun 2026
- Primary completionJun 2032 (estimated)
- Study completionJun 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Friedreich AtaxiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 26 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Lexeo Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Lexeo Clinical Trials, Study director, Lexeo Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07721025
- Sponsor's protocol ID
- LX2006-03
- Other IDs
- 2025-524970-40-00, 2025
- Acronym
- SUNRISE-FA 2
- Status last verified
- Aug 2026
- First submitted
- 24 Jun 2026
- Last updated
- 2 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Multicenter, Open-label, Randomized, Controlled Study of LX2006 Gene Therapy in Participants With Friedreich Ataxia Cardiomyopathy (SUNRISE-FA 2)
- ClinicalTrials.gov ID
- NCT07721025
- Acronym / sponsor ID
- SUNRISE-FA 2 / LX2006-03
- Conditions
- Friedreich Ataxia, Cardiomyopathy, Secondary
- Keywords
- Friedreich Ataxia Cardiomyopathy, FA-CM, Friedreich Ataxia, Friedreich's Ataxia, FA, FRDA, Cardiomyopathy, Genetic Cardiomyopathy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 26 participants (estimated)
- Lead sponsor
- Lexeo TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 25 Jun 2026 (actual)
- Primary completion
- Jun 2032 (estimated)
- Study completion
- Jun 2032 (estimated)
- First posted
- 22 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| LX2006 | Experimental | Genetic: LX2006Cohort 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. |
| Usual Care | Other | Other: Usual CareCohort 1: Participants ≥16 years of age with FA-CM Participants will receive usual care for 26 weeks before receiving treatment with LX2006 (single crossover). |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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%
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LX2006Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN) | Genetic | LX2006 |
| Usual CareCohort 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 |
Primary outcome
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
15 secondary outcomes
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
Time frame: At Month 60
Cohort 1: Change from baseline in modified Friedreich's Ataxia Rating Scale
Time frame: At Week 26, at Week 52, and through Month 60
Cohort 1: Change from baseline in Kansas City Cardiomyopathy Questionnaire
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
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Sep 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Lexeo Clinical Trials study director
Lexeo Therapeutics, Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing LX2006 in people with Friedreich Ataxia and Cardiomyopathy, Secondary, run by Lexeo Therapeutics at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 2 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Lexeo Clinical Trials (212-547-9879, clinicaltrials@lexeotx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cohort 1: Percent change from baseline in left ventricular mass index by cardiac MRI (time frame: At Week 26). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Sep 2026.