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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

Tracking Information
First Submitted Date  ICMJE October 25, 2023
First Posted Date  ICMJE October 31, 2023
Last Update Posted Date April 3, 2024
Actual Study Start Date  ICMJE February 29, 2024
Estimated Primary Completion Date February 2026   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 30, 2023)		 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
Original Primary Outcome Measures  ICMJE
 (submitted: October 25, 2023)		 Safety and Tolerability of LX2020 [ Time Frame: 12 months ]
Percentage of subjects who experienced at least 1 treatment emergent adverse event (TEAE) and/or 1 treatment emergent serious adverse event (TESAE).
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures
 (submitted: October 25, 2023)		 Selected Exploratory [ Time Frame: 12 months ]
Change in the frequency of ventricular arrhythmias from baseline.
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title  ICMJE Gene Therapy for ACM Due to a PKP2 Pathogenic Variant
Official Title  ICMJE 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
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
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Arrhythmogenic Cardiomyopathy
  • PKP2-ACM
  • PKP2-ARVC
Intervention  ICMJE Genetic: LX2020
LX2020 is an adeno-associated viral vector encoding the human Plakophilin-2 (PKP2) gene (AAVrh.10hPKP2)
Study Arms  ICMJE Experimental: LX2020
Single ascending dose of LX2020, with a starting dose of 2.0 x10^13 gc/kg, in multiple cohorts
Intervention: Genetic: LX2020
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: October 25, 2023)		 10
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE February 2027
Estimated Primary Completion Date February 2026   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: LEXEO Clinical Trials 212-547-9879 clinicaltrials@lexeotx.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT06109181
Other Study ID Numbers  ICMJE LX2020-01
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Lexeo Therapeutics
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Lexeo Therapeutics
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: LEXEO Clinical Trials Lexeo Therapeutics
PRS Account Lexeo Therapeutics
Verification Date April 2024

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP