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Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

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: NCT06109181
Recruitment Status : Recruiting
First Posted : October 31, 2023
Last Update Posted : April 3, 2024
Sponsor:
Information provided by (Responsible Party):
Lexeo Therapeutics

Study Description
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Brief Summary:
This is a Phase 1/2, first-in-human, open-label, intravenous, dose-escalating, multicenter trial that is designed to assess the safety and tolerability of LX2020 in adult patients with PKP2-ACM

Condition or disease Intervention/treatment Phase
Arrhythmogenic Cardiomyopathy PKP2-ACM PKP2-ARVC Genetic: LX2020 Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 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 LX2020 Gene Therapy in Patients With Arrhythmogenic Cardiomyopathy Due to a Plakophilin-2 Pathogenic Variant
Actual Study Start Date : February 29, 2024
Estimated Primary Completion Date : February 2026
Estimated Study Completion Date : February 2027

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: LX2020
Single ascending dose of LX2020, with a starting dose of 2.0 x10^13 gc/kg, in multiple cohorts
 Genetic: LX2020
LX2020 is an adeno-associated viral vector encoding the human Plakophilin-2 (PKP2) gene (AAVrh.10hPKP2)


Outcome Measures
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Primary Outcome Measures :
  1. Percentage of subjects who experienced at least 1 treatment emergent adverse event (TEAE) and/or 1 treatment emergent serious adverse event (TESAE). [ Time Frame: 12 months ]
    Evaluation of Safety and Tolerability of LX2020


Other Outcome Measures:
  1. Selected Exploratory [ Time Frame: 12 months ]
    Change in the frequency of ventricular arrhythmias from baseline.


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Selected Inclusion Criteria:

  • Adults with a clinical diagnosis of ACM meeting the 2010 revised Task Force Criteria (TFC)
  • Genetic testing documenting a pathogenic or likely pathogenic variant in PKP2
  • Frequent premature ventricular complexes (PVCs)
  • Implantable cardioverter-defibrillator (ICD) implantation ≥ 12 weeks prior to consent
  • Left ventricular ejection fraction ≥ 50%

Selected Exclusion Criteria:

  • Evidence of variant(s) in addition to PKP2 that meets the standard criteria to be considered pathogenic or likely pathogenic for ACM
  • Other cardiac abnormalities as specified in the protocol
  • New York Heart Association Functional Class III and IV at the time of consent
  • History of prior gene transfer therapy
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): NCT06109181


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

Locations
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United States, Michigan
University of Michigan Recruiting
Ann Arbor, Michigan, United States, 48109
Contact: Eric Smith, MD    734-764-4500    ersm@med.umich.edu   
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: NCT06109181    
Other Study ID Numbers: LX2020-01
First Posted: October 31, 2023    Key Record Dates
Last Update Posted: April 3, 2024
Last Verified: April 2024

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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:
Gene Therapy
Arrhythmogenic Cardiomyopathy
ACM
Cardiomyopathy
ARVC
Arrhythmogenic Right Ventricular
Arrhythmogenic Right Ventricular Dysplasia
Genetic cardiomyopathy
 PKP2 Gene
Plakophilin-2
LX2020
HEROIC-PKP2
Ventricular Arrhythmia
PVCs
Sudden Cardiac Death
Cardiac Arrest
Additional relevant MeSH terms:
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Cardiomyopathies
Heart Diseases
Cardiovascular Diseases