A Study to Assess Real-world Patient Characteristics and Clinical Course for Symptomatic Patients With PKP2-ACM
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18-65
SponsorLexeo Therapeutics
About this trial
An observational study to assess real-world patient characteristics and clinical course of disease in participants with PKP2-ACM.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Adults with a clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC)
Documentation of a pathogenic or likely pathogenic truncating variant in PKP2
Frequent premature ventricular contractions (PVCs)
Patients must have an ICD placed prior to enrollment
Disqualifiers
Evidence of variant(s) in addition to PKP2 that meet standard criteria to be considered pathogenic or likely pathogenic for an arrhythmogenic cardiomyopathy.
A history of other cardiac abnormalities as specified in the protocol.
New York Heart Association symptoms of heart failure of Class IV at the time of consent.
A history of prior gene transfer therapy.
Trial population
Patients aged 18-65 years at time of consent with confirmed diagnosis of ACM and documentation of PKP2 variant.
Trial design
Design model
Case-only
Time perspective
Other
Treatments tested in this trial
Not listed
Trial groups
No trial groups listed
Trial outcomes
Primary outcomes
1
Premature ventricular contractions (PVC)
Change from baseline
Time frame
12 monthsSecondary outcomes
1
Ventricular arrhythmias and associated clinical events
Change from baseline by interrogation of implanted cardioverter defibrillator and remote cardiac monitoring
Time frame
12 months2
ECG parameters
Change from baseline
Time frame
12 months3
Cardiac structure and function
Change from baseline via cardiac MRI and echocardiogram
Time frame
12 months4
Patient reported outcomes
Patients' Global Impression of Change (PGIC) scale, measuring participant perception of change from baseline.
Time frame
12 monthsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov