Arrhythmogenic CardiomyopathySNAPSHOT- PKP2NCT06976606
A Study to Assess Real-world Patient Characteristics and Clinical Course for Symptomatic Patients With PKP2-ACM
An observational study of Arrhythmogenic Cardiomyopathy and PKP2-ACM, run by Lexeo Therapeutics at 6 sites in United States.
Trial summary
An observational study to assess real-world patient characteristics and clinical course of disease in participants with PKP2-ACM. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Arrhythmogenic Cardiomyopathy, PKP2-ACM, PKP2-ARVC
- How many people
- 40 participants
- Where
- 6 sitesUnited States
- Study dates
- 23 Jan 2024to Sep 2027
- How long will I take part?
- 1 Year
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients aged 18-65 years at time of consent with confirmed diagnosis of ACM and documentation of PKP2 variant.
Who may be able to join
- 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
- Left ventricular ejection fraction (LVEF) ≥ 50% for Part A participants. Left ventricular ejection fraction (LVEF) ≥40% for Part B participants.
Who cannot join
- 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 locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
6 sites in 1 country
United States6 sites, 6 recruiting
- Recruiting
University of Michigan
Ann Arbor, Michigan, 48109
Muhammad Hussain, 734-765-8217, muhammhu@med.umich.edu
- Recruiting
Johns Hopkins University
Baltimore, Maryland, 21287
Crystal Tichnell, 410-502-7161, ctichne1@jhmi.edu
- Recruiting
Northshore University Healthsystem Research Institute
Columbia, Maryland, 21044
bprindeville@northshore.org
- Recruiting
Henry Ford Hospital
Detroit, Michigan, 48202
wcabral1@hfhs.org
- Recruiting
Leland Stanford Junior University
Redwood City, California, 94063-3126
Priya Nair, 512-217-3812, ashas@stanford.edu
- Recruiting
University of Rochester
Rochester, New York, 14642
lori_caufield@urmc.rochester.edu
About this study
Official title: An Observational Study to Assess Real-world Patient Characteristics, Clinical Course, and Treatment Patterns for Symptomatic Patients With Arrhythmogenic Cardiomyopathy (ACM) Due To a PlaKoPhilin-2 Pathogenic Variant (PKP2)
An observational study to assess real-world patient characteristics and clinical course of disease in participants with PKP2-ACM.
Read the detailed description
SNAPSHOT-PKP2 is an observational, multicenter study that consists of 2 parts, a retrospective EMR review (up to 2 years) and a prospective observational study (1 year), to evaluate the clinical burden of illness of patients with PKP2-ACM, and to prospectively evaluate changes in key cardiac parameters and patient-reported outcome measures (PROMs) associated with PKP2-ACM progression.
- Conditions
- Arrhythmogenic Cardiomyopathy, PKP2-ACM, PKP2-ARVC
- Keywords
- Arrhythmogenic Cardiomyopathy, ACM, Cardiomyopathy, ARVC, Arrhythmogenic Right Ventricular, Arrhythmogenic Right Ventricular Dysplasia, Genetic cardiomyopathy, Gene Therapy, PKP2 Gene, Plakophilin-2, LX2020, SNAPSHOT-PKP2, Ventricular Arrhythmia, PVCs, Sudden Cardiac Death, Cardiac Arrest
- Medical subject headings
- Cardiomyopathies, Arrhythmogenic Right Ventricular Dysplasia, Death, Sudden, Cardiac, Heart Arrest
What is being measured
The primary outcome is the main question the study is designed to answer.
Premature ventricular contractions (PVC)
Change from baseline
Measured: 12 months
Secondary outcomes (7)
Ventricular arrhythmias and associated clinical events
Change from baseline by interrogation of implanted cardioverter defibrillator and remote cardiac monitoring
Measured: 12 months
ECG parameters
Change from baseline
Measured: 12 months
Cardiac structure and function
Change from baseline via cardiac MRI and echocardiogram
Measured: 12 months
Patient reported outcomes
Patients' Global Impression of Change (PGIC) scale, measuring participant perception of change from baseline.
Measured: 12 months
Patient reported outcomes
Patients' Global Impression of Severity (PGIS) scale, measuring patient perception of disease progression.
Measured: 12 months
Cardiac Biomarkers
Change from baseline
Measured: 12 months
Health resource utilization
Number of ED, urgent care and outpatient visits, and hospitalizations
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 May 2025
- Study start23 Jan 2024
- Primary completionJul 2027 (estimated)
- Study completionSep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Arrhythmogenic CardiomyopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Other
- Enrolment
- 40 participants (estimated)
- Target duration
- 1 Year
Sponsor and investigators
- Lead sponsor
- Lexeo Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Lexeo Clinical Trials, Study director, Lexeo Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06976606
- Sponsor's protocol ID
- LX2020-03
- Acronym
- SNAPSHOT- PKP2
- Status last verified
- May 2025
- First submitted
- 28 Mar 2024
- Last updated
- 16 May 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Observational Study to Assess Real-world Patient Characteristics, Clinical Course, and Treatment Patterns for Symptomatic Patients With Arrhythmogenic Cardiomyopathy (ACM) Due To a PlaKoPhilin-2 Pathogenic Variant (PKP2)
- ClinicalTrials.gov ID
- NCT06976606
- Acronym / sponsor ID
- SNAPSHOT- PKP2 / LX2020-03
- Conditions
- Arrhythmogenic Cardiomyopathy, PKP2-ACM, PKP2-ARVC
- Keywords
- Arrhythmogenic Cardiomyopathy, ACM, Cardiomyopathy, ARVC, Arrhythmogenic Right Ventricular, Arrhythmogenic Right Ventricular Dysplasia, Genetic cardiomyopathy, Gene Therapy
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Lexeo TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 23 Jan 2024 (actual)
- Primary completion
- Jul 2027 (estimated)
- Study completion
- Sep 2027 (estimated)
- First posted
- 16 May 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
An observational study to assess real-world patient characteristics and clinical course of disease in participants with PKP2-ACM.
Detailed description
SNAPSHOT-PKP2 is an observational, multicenter study that consists of 2 parts, a retrospective EMR review (up to 2 years) and a prospective observational study (1 year), to evaluate the clinical burden of illness of patients with PKP2-ACM, and to prospectively evaluate changes in key cardiac parameters and patient-reported outcome measures (PROMs) associated with PKP2-ACM progression.
Study design
- Observational model
- Case only
- Time perspective
- Other
- Target follow-up
- 1 Year
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients aged 18-65 years at time of consent with confirmed diagnosis of ACM and documentation of PKP2 variant.
Key inclusion criteria (5 listed)
- 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
- Left ventricular ejection fraction (LVEF) ≥ 50% for Part A participants. Left ventricular ejection fraction (LVEF) ≥40% for Part B participants.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Premature ventricular contractions (PVC)
Time frame: 12 months
Change from baseline
7 secondary outcomes
Ventricular arrhythmias and associated clinical events
Time frame: 12 months
ECG parameters
Time frame: 12 months
Cardiac structure and function
Time frame: 12 months
Patient reported outcomes
Time frame: 12 months
Patient reported outcomes
Time frame: 12 months
Cardiac Biomarkers
Time frame: 12 months
Health resource utilization
Time frame: 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 May 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Lexeo Clinical Trials study director
Lexeo Therapeutics
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Arrhythmogenic Cardiomyopathy and PKP2-ACM, run by Lexeo Therapeutics at 6 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Person*: Lexeo Clinical Trials (212-547-9879, clinicaltrials@lexeotx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Premature ventricular contractions (PVC) (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 May 2025.