NCT07721025RecruitingPhase 2
Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
A Phase 2, Multicenter, Open-label, Randomized, Controlled Study of LX2006 Gene Therapy in Participants With Friedreich Ataxia Cardiomyopathy (SUNRISE-FA 2)
Source: ClinicalTrials.gov · Last updated 2 Sept 2026
Brief summary (registry, in English)
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).
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 26 (estimated)
- First submitted
- 24 Jun 2026
- Start date
- 25 Jun 2026
- Primary completion date
- Jun 2032
- Lead sponsor
- Lexeo Therapeutics
- NCT number
- NCT07721025
- EU CT number
- 2025-524970-40-00
What you commit to
- Study type: Interventional · Phase 2
- Planned participants: 26
- From 25 Jun 2026 to the primary completion date, Jun 2032
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Prior organ transplant
- Previous gene transfer or cell therapy
Other illness
- Presence of other forms of cardiomyopathy that contribute to heart failure
- Contraindication to cardiac MRI
- Active hematologic or solid organ cancer
- Other inclusion/exclusion criteria to be applied as per protocol.
Lab values
- Poorly controlled diabetes (hemoglobin A1c ≥8%)
Other
- Current use of inotrope infusion or presence of a ventricular assist device
Who the study is looking for
- 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
Age in the registry: 6 years to - · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| University of South Florida Coordinator of this site? Confirm its status | Tampa | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.