A Phase 2, Multicenter, Open-label, Randomized, Controlled Study of LX2006 Gene Therapy in Participants With Friedreich Ataxia Cardiomyopathy (SUNRISE-FA 2)
Status: Recruiting
Location: See location...
Intervention Type: Other, Genetic
Study Type: Interventional
Study Phase: Phase 2
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).
Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Healthy Volunteers: f
View:
• 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
Locations
United States
Florida
University of South Florida
RECRUITING
Tampa
Contact Information
Primary
Lexeo Clinical Trials
clinicaltrials@lexeotx.com
212-547-9879
Time Frame
Start Date: 2026-06-25
Estimated Completion Date: 2032-06
Participants
Target number of participants: 26
Treatments
Experimental: LX2006
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.
Other: Usual Care
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).
Related Therapeutic Areas
Sponsors
Leads: Lexeo Therapeutics