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Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05445323
Recruitment Status : Recruiting
First Posted : July 6, 2022
Last Update Posted : February 8, 2024
Sponsor:
Information provided by (Responsible Party):
Lexeo Therapeutics

Study Description
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Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Friedreich Ataxia Cardiomyopathy, Secondary Genetic: Low dose LX2006 Genetic: Mid Dose LX2006 Genetic: High Dose LX2006 Phase 1 Phase 2

Detailed Description:

Friedreich's ataxia (FA) is a rare, autosomal recessive disease caused by a mutation in the autosomal frataxin (FXN) gene. Progressive cardiomyopathy with cardiac hypertrophy and fibrosis is observed in most individuals with FA. The disease is more severe in those with earlier onset. Presently, there is no therapy that alters the progression of cardiomyopathy in FA, which is responsible for 59% of FA-related deaths.

The primary objective of this dose escalation study is to assess the safety and tolerability of three ascending doses of LX2006 in patients with FA-associated cardiomyopathy. LX2006 is designed to restore hFXN levels in order to improve mitochondrial function. Assessments of cardiac function, biomarkers and other preliminary efficacy endpoints are also included in this study.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 9 participants
Allocation: N/A
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1/2 Study of the Safety and Efficacy of LX2006 Gene Therapy in Participants With Cardiomyopathy Associated With Friedreich's Ataxia
Actual Study Start Date : August 24, 2022
Estimated Primary Completion Date : September 2029
Estimated Study Completion Date : September 2029

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Cohort 1/ Cohort 2/ Cohort 3 Genetic: Low dose LX2006
Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)

Genetic: Mid Dose LX2006
Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)

Genetic: High Dose LX2006
Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN)


Outcome Measures
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Primary Outcome Measures :
  1. Treatment-emergent adverse events (TEAEs) and Treatment-emergent serious events (TESAEs) [ Time Frame: Change from baseline to end of year 5 post dose ]

Secondary Outcome Measures :
  1. Change from baseline in LVMi [ Time Frame: Change from baseline to end of year 5 post dose ]
  2. Change from baseline in LVEF [ Time Frame: Change from baseline to end of year 5 post dose ]
  3. Change from baseline in cardiac fibrosis as measured by cardiac MRI [ Time Frame: Change from baseline to end of year 5 post dose ]
  4. Change from baseline in measures of cardiopulmonary exercise tolerance [ Time Frame: Change from baseline to end of year 5 post dose ]
  5. Presence and severity of cardiac arrythmias [ Time Frame: Change from baseline to end of year 5 post dose ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 50 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Confirmed genetic diagnosis of FA, with onset being before 25 years of age
  • Protocol specified ranges for antibodies
  • Protocol specified measures of FA cardiomyopathy

Exclusion Criteria:

  • Protocol specified ranges for left ventricular ejection fraction (LVEF) as measured by cardiac ECHO
  • Uncontrolled diabetes
  • Abnormal liver function
  • Active infection of any type, including hepatitis virus (A, B or C) or human immunodeficiency virus (HIV-1 and HIV-2)
  • Contraindication to cardiac MRI
  • Contraindications to cardiac biopsies
  • Participants who are receiving systemic corticosteroids or other immunosuppressive medications
  • History of significant coronary artery disease or any structural heart or vascular disease other than FA cardiomyopathy
  • Presence of clinically significant, hemodynamically unstable arrhythmias, requiring physician intervention
  • Presence of clinically significant abnormalities as determined by the investigator, other than ECG abnormalities related to FA
  • Uncontrolled psychiatric disease

Other Inclusion/Exclusion criteria to be applied as per protocol.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05445323


Contacts
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Contact: LEXEO Clinical Trials 212-547-9879 clinicaltrials@lexeotx.com

Locations
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United States, California
Ataxia Center and HD Center of Excellence, University of California Recruiting
Los Angeles, California, United States, 90095
Contact: Aaron Fisher    310-206-8153    adfisher@mednet.ucla.edu   
United States, Florida
University of South Florida Recruiting
Tampa, Florida, United States, 33612
Contact: Lucretia Campbell    813-974-5633    lcampbel@usf.edu   
United States, Iowa
University of Iowa Recruiting
Iowa City, Iowa, United States, 52242
Contact: Ciara Gibbs    319-384-9618    ciara-gibbs@uiowa.edu   
United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
Contact: Michaela Kolarova    507-266-7969    kolarova.michaela@mayo.edu   
Contact: Clinical Genomics Research Team       rstcgresearch@mayo.edu   
United States, Pennsylvania
Hospital of the University of Pennsylvania Not yet recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: David Lynch, MD, PhD         
Canada, Quebec
Centre hospitalier de l Universite de Montreal (CHUM) Recruiting
Montréal, Quebec, Canada, H2X 0C1
Contact: Antoine Duquette, MD    514-890-8000 ext 30737    uit.eligibilite.chum@ssss.gouv.qc.ca   
Sponsors and Collaborators
Lexeo Therapeutics
Investigators
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Study Director: LEXEO Clinical Trials Lexeo Therapeutics
More Information
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Responsible Party: Lexeo Therapeutics
ClinicalTrials.gov Identifier: NCT05445323    
Other Study ID Numbers: LX2006-01
First Posted: July 6, 2022    Key Record Dates
Last Update Posted: February 8, 2024
Last Verified: February 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Lexeo Therapeutics:
Friedreich's Ataxia
Cardiomyopathy
FA
Gene therapy
 FXN Gene
Frataxin Gene
LX2006
Additional relevant MeSH terms:
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Ataxia
Cerebellar Ataxia
Friedreich Ataxia
Cardiomyopathies
Heart Diseases
Cardiovascular Diseases
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Cerebellar Diseases
 Brain Diseases
Central Nervous System Diseases
Spinocerebellar Degenerations
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases