ClinicalTrials.gov record NCT07721025

Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 7, 2026

A Phase 2, Multicenter, Open-label, Randomized, Controlled Study of LX2006 Gene Therapy in Participants With Friedreich Ataxia Cardiomyopathy (SUNRISE-FA 2)

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 2A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
Age6 years old and older
SexAll
LocationUnited States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 2, randomized, open-label, single-masked study evaluates the safety and efficacy of LX2006 gene therapy in people with Friedreich ataxia who have cardiomyopathy. In the older cohort (≥16 years) participants are randomized to receive LX2006 immediately or receive usual care for 26 weeks then cross over to LX2006; a younger cohort (6 to <16 years) will receive LX2006 after initial safety is assessed and is not randomized. Participants are followed with cardiac imaging, biomarker and clinical assessments for ongoing study measures.

What it is studying. The study evaluates whether LX2006 (an AAVrh.10 vector carrying the FXN gene) affects heart structure and function in Friedreich ataxia cardiomyopathy and assesses its safety. The primary measure is percent change from baseline in left ventricular mass index by cardiac MRI at Week 26; other measures include cardiac biomarkers, imaging, clinical cardiac events, neurological rating scales, quality-of-life questionnaires, and safety measures through Month 60.

What the researchers hope to learn. To evaluate the efficacy and safety of LX2006 gene therapy in people with Friedreich ataxia cardiomyopathy. The study aims to measure changes in cardiac structure by MRI, cardiac biomarkers, neurological function, quality of life, clinical cardiovascular events, and safety over time.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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 being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Genetic
  • LX2006
Other
  • Usual Care

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants will receive the study gene therapy LX2006 (the trial includes a group that receives usual care for 26 weeks then crosses over to receive LX2006). Visits include cardiac imaging (cardiac MRI), blood tests including troponin and routine safety labs, heart monitoring (ECG, vital signs), neurological assessments (modified Friedreich's Ataxia Rating Scale), and questionnaires about heart-related symptoms and global impressions. Follow-up and safety monitoring continue through Month 60. The record does not state the exact route or number of doses of LX2006.

How the study is structured. Interventional Phase 2 trial, randomized with a crossover design and single masking. Older participants (≥16 years) are randomized to LX2006 or to 26 weeks of usual care followed by LX2006 (single crossover); younger participants (6 to <16 years) receive LX2006 after safety is evaluated in the older group. Follow-up includes repeated imaging, blood tests, clinical assessments, and questionnaires through long-term follow-up.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Friedreich ataxia

Clinical trials26 trials listed for Friedreich ataxiaEvery trial the registry lists for Friedreich ataxia, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Friedreich ataxia researchLatest monthly update: September 2026. Includes: Heart change stages. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Jun 25, 2026
Primary completion
June 2032 (estimated)
Study completion
June 2032 (estimated)
Target enrollment
26 participants (estimated)
Last updated on registry
Sep 2, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Lexeo Therapeutics

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

open-label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
randomized
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
assent
A child's or dependent adult's agreement to take part, given alongside the formal consent of a parent or legal representative.
Contraindication
A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
MRI
A scan that uses magnets and radio waves, not X-rays, to make detailed pictures of the inside of the body. Some implants and metal objects mean a person cannot have one.
exclusion criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this website; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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