8 Participants Needed

Gene Therapy for Cardiomyopathy in Friedreich's Ataxia

Recruiting at 3 trial locations
LC
Overseen ByLEXEO Clinical Trials
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This is a Phase 1/2, open-label, dose-ascending, multicenter study of the safety and efficacy of LX2006 for participants who have Friedreich's Ataxia with evidence of cardiomyopathy. The study will evaluate up to three doses of single administration of LX2006 (AAVrh.10hFXN), an adeno-associated virus (AAV) gene therapy designed to intravenously deliver the human frataxin (hFXN) gene to cardiac cells over a 52-week period. Long-term safety and efficacy will be evaluated for an additional 4-years for a total of 5-years post LX2006 treatment.

Do I need to stop my current medications for the trial?

The trial excludes participants who are taking systemic corticosteroids or other immunosuppressive medications, so you may need to stop these if you are currently taking them. The protocol does not specify about other medications.

Is the gene therapy treatment LX2006 generally safe in humans?

Research on similar gene therapies using adeno-associated virus (AAV) vectors, like those for Duchenne muscular dystrophy, shows they are generally safe and well-tolerated in humans, with no significant immune response observed in clinical trials.12345

How does the treatment LX2006 differ from other treatments for Friedreich's Ataxia cardiomyopathy?

LX2006 is a gene therapy that uses an adeno-associated virus (AAV) to deliver a normal copy of the frataxin gene directly to heart cells, aiming to correct the underlying genetic cause of cardiomyopathy in Friedreich's Ataxia. This approach is unique because it targets the root cause of the disease at the genetic level, unlike other treatments that may only address symptoms.678910

What data supports the effectiveness of the treatment LX2006 for cardiomyopathy in Friedreich's Ataxia?

Research on similar gene therapies using adeno-associated viruses (AAV) for Duchenne muscular dystrophy (DMD) shows promise in improving heart function and reducing cardiac issues. These studies suggest that AAV-based gene therapy can be effective in treating heart-related conditions, which may indicate potential benefits for cardiomyopathy in Friedreich's Ataxia.1231112

Who Is on the Research Team?

LC

LEXEO Clinical Trials

Principal Investigator

Lexeo Therapeutics

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

Your levels of specific antibodies need to be within certain ranges as outlined in the study protocol.
You have specific heart problems described in the study protocol.
I was diagnosed with Fanconi anemia before I turned 25.

Exclusion Criteria

You have a mental health condition that is not being well managed.
My diabetes is not under control.
I have heart disease not related to FA cardiomyopathy.
See 9 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single administration of LX2006 gene therapy, with dose escalation across cohorts

52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 years

What Are the Treatments Tested in This Trial?

Interventions

  • LX2006
Trial Overview 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.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: Cohort 1/ Cohort 2/ Cohort 3Experimental Treatment3 Interventions

Find a Clinic Near You

Who Is Running the Clinical Trial?

Lexeo Therapeutics

Lead Sponsor

Trials
5
Recruited
110+

Published Research Related to This Trial

The micro-dystrophin gene transfer using rAAVrh74.MHCK7 was found to be well tolerated in a phase 1/2a trial with four young patients, showing only mild to moderate adverse events and no serious complications over one year.
All patients demonstrated significant expression of micro-dystrophin in muscle fibers and improvements in functional measures, such as North Star Ambulatory Assessment scores and reduced creatine kinase levels, indicating potential benefits beyond standard care for Duchenne muscular dystrophy.
Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial.Mendell, JR., Sahenk, Z., Lehman, K., et al.[2021]
The clinical trial demonstrated that the chimeric AAV2.5 vector is safe and well tolerated in boys with Duchenne muscular dystrophy (DMD), with no cellular immune response detected against the AAV2.5 capsid.
AAV2.5 showed effective gene transfer, with recombinant AAV genomes found in all patients, indicating its potential for efficient muscle transduction and paving the way for future gene therapy applications.
Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector.Bowles, DE., McPhee, SW., Li, C., et al.[2022]
In a study of 48 pediatric patients with Friedreich's ataxia, 85% showed elevated left ventricular mass, indicating a common subclinical hypertrophic cardiomyopathy despite normal heart function as measured by ejection fraction.
Concentric hypertrophy was found in 44% of subjects and was specifically linked to significant diastolic and systolic dysfunction, highlighting the need for cardiac monitoring in these patients.
The Subclinical Cardiomyopathy of Friedreich's Ataxia in a Pediatric Population.Plehn, JF., Hasbani, K., Ernst, I., et al.[2019]

Citations

Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial. [2021]
AAV micro-dystrophin gene therapy alleviates stress-induced cardiac death but not myocardial fibrosis in >21-m-old mdx mice, an end-stage model of Duchenne muscular dystrophy cardiomyopathy. [2021]
[Study on the recombinant adeno-associated virus vector carrying LacZ gene expression in the skeletal muscle]. [2007]
AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy. [2022]
Long-term preservation of cardiac structure and function after adeno-associated virus serotype 9-mediated microdystrophin gene transfer in mdx mice. [2012]
Cardiac expression of a mini-dystrophin that normalizes skeletal muscle force only partially restores heart function in aged Mdx mice. [2021]
Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector. [2022]
The Subclinical Cardiomyopathy of Friedreich's Ataxia in a Pediatric Population. [2019]
Stress-Induced Mouse Model of the Cardiac Manifestations of Friedreich's Ataxia Corrected by AAV-mediated Gene Therapy. [2023]
Excision of the expanded GAA repeats corrects cardiomyopathy phenotypes of iPSC-derived Friedreich's ataxia cardiomyocytes. [2023]
11.United Statespubmed.ncbi.nlm.nih.gov
Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia. [2023]
Correction of half the cardiomyocytes fully rescue Friedreich ataxia mitochondrial cardiomyopathy through cell-autonomous mechanisms. [2023]
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