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
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