LX2006 Gene Therapy for Friedreich Ataxia
(SUNRISE-FA 2 Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
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).
Who Is on the Research Team?
Lexeo Clinical Trials
Principal Investigator
Lexeo Therapeutics, Inc.
Are You a Good Fit for This Trial?
This trial is for people aged 6 and older diagnosed with Friedreich ataxia who developed symptoms by age 25, have a specific heart problem (left ventricular hypertrophy), and meet certain heart function requirements. People with other major heart diseases, prior organ transplants, uncontrolled diabetes, or previous gene therapy can't join.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Usual Care
Participants receive usual care for 26 weeks before receiving treatment with LX2006
Treatment
Participants receive LX2006 gene therapy
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- LX2006
Trial Overview
The study compares LX2006 gene therapy to usual care in patients with Friedreich ataxia-related cardiomyopathy. Participants are randomly assigned to receive either the new gene therapy or standard treatment. The goal is to see if LX2006 improves heart health and is safe.
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
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).
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.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Lexeo Therapeutics
Lead Sponsor
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