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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up change from baseline to end of year 5 post dose

Awards & highlights

Study 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.Check my eligibility

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.See study design

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

Select...

I was diagnosed with Fanconi anemia before I turned 25.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ change from baseline to end of year 5 post dose

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~change from baseline to end of year 5 post dose

This trial's timeline: 3 weeks for screening, Varies for treatment, and change from baseline to end of year 5 post dose for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Treatment-emergent adverse events (TEAEs) and Treatment-emergent serious events (TESAEs)

Secondary outcome measures

Change from baseline in LVEF

Change from baseline in LVMi

Change from baseline in cardiac fibrosis as measured by cardiac MRI

+2 more

Trial Design

1Treatment groups

Experimental Treatment

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

0 / 1Eligibility criteria met

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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there still opportunities to join this trial?

"Affirmative. According to information hosted on clinicaltrials.gov, this medical study is actively seeking participants after being first posted on August 24th 2022 and recently updated November 28th 2022. 10 individuals must be sought from 4 locations in order for the trial to commence."

Answered by AI

What type of individual is most suitable to partake in this research?

"This medical trial is looking for 10 qualified subjects aged between 18 and 40 with a confirmed diagnosis of Friedreich Ataxia. To be eligible, participants must fit the following criteria: have no contraindications to cardiac biopsies; meet protocol specified ranges for cardiopulmonary exercise testing (CPET) arm ergometry; present no evidence of active infection or hepatitis virus (A, B or C); possess normal liver and kidney function; show protocol-specified ranges on left ventricular ejection fraction (LVEF), left ventricular hypertrophy (LVH) as measured by mass index (LVMi),"

Answered by AI

Is this clinical trial open to participants aged 70 or older?

"This clinical trial allows for the enrolment of participants aged 18 and above, but no older than 40."

Answered by AI

To what extent is the current population enrolled in this trial?

"Confirmed. Per the information on clinicaltrials.gov, this medical investigation is actively recruiting patients; it was initially advertised on August 24th 2022 and updated lastly on November 28th of that same year. The trial requires 10 volunteers to be recruited from 4 different hospital sites."

Answered by AI

Are there any American healthcare facilities that are currently conducting this investigation?

"At the current moment, this trial is enrolling from 4 different sites in Rochester, Iowa City, Tampa and other locales. If you plan to join the study, it's suggested that you pick a nearby venue for convenience purposes."

Answered by AI