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Gene Therapy

Gene Therapy for Cardiomyopathy in Friedreich's Ataxia

Phase 1 & 2
Recruiting
Research Sponsored by Lexeo Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Confirmed genetic diagnosis of FA, with onset being before 25 years of age
Be between 18 and 65 years old
Must not have
Uncontrolled diabetes
History of significant coronary artery disease or any structural heart or vascular disease other than FA cardiomyopathy
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group

Summary

This trial will test a new gene therapy for Friedreich's Ataxia, a rare degenerative disease. The therapy will be delivered intravenously and will be evaluated for safety and efficacy over a 5 year period.

Who is the study for?
This trial is for individuals with Friedreich's Ataxia diagnosed before age 25 who have heart issues related to the condition. They must meet specific criteria for cardiomyopathy and antibody levels. Excluded are those with significant coronary disease, uncontrolled diabetes or psychiatric conditions, abnormal liver function, certain infections like hepatitis or HIV, unstable heart rhythms needing intervention, or on immunosuppressive drugs.
What is being tested?
The study tests LX2006 gene therapy at different doses in people with Friedreich's Ataxia-related cardiomyopathy. It involves a single administration of an AAV vector carrying the human frataxin gene to cardiac cells and monitors safety and effectiveness over one year, with a follow-up period extending to five years.
What are the potential side effects?
Potential side effects may include immune reactions due to viral vectors used in gene therapy (like fever or muscle pain), complications from cardiac procedures such as biopsies (bleeding or infection), and possible liver function changes.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I was diagnosed with Fanconi anemia before I turned 25.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My diabetes is not under control.
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I have heart disease not related to FA cardiomyopathy.
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My heart's pumping ability is within the required range.
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I cannot have heart tissue samples taken due to health risks.
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I do not have any active infections, including hepatitis or HIV.
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I am currently taking steroids or other medications that weaken my immune system.
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My liver isn't working properly.
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I have serious heart rhythm problems needing doctor's care.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Cohort 1/ Cohort 2/ Cohort 3Experimental Treatment3 Interventions

Find a Location

Who is running the clinical trial?

Lexeo TherapeuticsLead Sponsor
3 Previous Clinical Trials
40 Total Patients Enrolled
LEXEO Clinical TrialsStudy DirectorLexeo Therapeutics
1 Previous Clinical Trials
10 Total Patients Enrolled

Media Library

LX2006 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05445323 — Phase 1 & 2
Friedreich Ataxia Research Study Groups: Cohort 1/ Cohort 2/ Cohort 3
Friedreich Ataxia Clinical Trial 2023: LX2006 Highlights & Side Effects. Trial Name: NCT05445323 — Phase 1 & 2
LX2006 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05445323 — Phase 1 & 2
~6 spots leftby Sep 2029
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