Arrhythmogenic Right Ventricular CardiomyopathyRIDGENCT06311708
Non-interventional Study of Seroprevalence of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) and the Progression of Disease in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
An observational study of Arrhythmogenic Right Ventricular Cardiomyopathy, run by Tenaya Therapeutics at 21 sites in 6 countries.
Trial summary
This is a multicenter, non-interventional study to observe the natural progression of the disease and to study the prevalence of pre-existing antibodies to AAV9 used for gene therapy in a population of patients with PKP2 gene-associated ARVC. Read more about this study
- Who is it for?
- Ages 14 to 65 years
- Conditions the study is for
- Arrhythmogenic Right Ventricular Cardiomyopathy
- How many people
- 200 participants
- Where
- 21 sitesin 6 countries
- Study dates
- 31 Jan 2023to 11 Jul 2030
- 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 14 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients who are 14-65 years of age at the time of consent, and with confirmed mutations in PKP2 gene with a diagnosis of ARVC.
Who may be able to join
- Ages 14-65 years, inclusive, at the time of consent
- Pathogenic or likely pathogenic PKP2 gene mutation
- Diagnosed with ARVC and meet 2010 Modified Task Force Criteria for ARVC as affected.
- Functioning ICD
Who cannot join
- Currently receiving systemic immunosuppressive therapy, cytotoxic chemotherapy, immunoglobulin therapy or monoclonal antibody therapy
- History of clinically significant liver disease, hepatitis B virus, hepatitis C virus, human immunodeficiency virus, or tuberculosis infection
- Previously dosed with any investigational or approved gene therapy product at any time
- Concurrent participation in another interventional clinical trial unless approved by the Sponsor. Participation in a noninterventional study may be allowed at the investigator's discretion.
- History of cardiac transplant.
Trial locations and contacts
21 sites in 6 countries. Contact the study team or a site near you to ask about taking part.
Niharika Kamat, MS contact
21 sites in 6 countries
United States8 sites, 8 recruiting
- Recruiting
University of Colorado, Denver
Aurora, Colorado, 80045
Matthew Taylor, MD, PhD
- Recruiting
John Hopkins University School of Medicine
Baltimore, Maryland, 21287
Hugh G Calkins, MD
- Recruiting
Brigham and Women's Hospital
Boston, Massachusetts, 02115
Neal Lakdawala, MD
- Recruiting
Medical University of South Carolina
Charleston, South Carolina, 29425
Daniel Judge, MD
- Recruiting
Cleveland Clinic
Cleveland, Ohio, 44195
Wai Hong Wilson Tang, MD
- Recruiting
New York University
New York, New York, 10016
Larry Chinitz, MD
- Recruiting
Mayo Clinic
Rochester, Minnesota, 55905
John Giudicessi, MD PhD
- Recruiting
University of California San Francisco
San Francisco, California, 94143
Vasanth Vedantham, MD, PhD
France4 sites, 4 recruiting
- Recruiting
Hopital Louis Pradel
Bron
Philippe Chevalier, MD, PhD
- Recruiting
Nantes University Hospital
Nantes, 44000
Vincent Probst, MD
- Recruiting
Pitié-Salpêtrière Hospital
Paris, 75013
Estelle Gandjbakhch, MD, PhD
- Recruiting
Hôpital Haut-Lévêque
Pessac, 33604
Frederic Sacher, MD, PhD
United Kingdom4 sites, 4 recruiting
- Recruiting
The Queen Elizabeth Hospital
Glasgow, G514TF
Caroline Coats, MD
- Recruiting
Barts & The London Health NHS Trust
London, E1 1BB
Perry Elliott, MD
- Recruiting
St. George's University Hospitals NHS Foundation Trust
London, SW17 0QT
Elijah Behr, MD
- Recruiting
Royal Brompton & Harefield NHS Foundation Trust
London, SW3 6NP
Antonio Pantazis, MD
Germany2 sites, 2 recruiting
- Recruiting
University Hospital Muenster
Münster, 48149
Eric Schulze-Bahr, MD
- Recruiting
Wuerzburg University Hospital
Würzburg, 97080
Brenda Gerull, MD
Italy2 sites, 2 recruiting
- Recruiting
Centro Cardiologico Monzino
Milan, 20138
Claudio Tondo, MD
- Recruiting
Istituti Clinici Scientifici Maugeri SpA
Pavia
Silvia Priori, MD, PhD
Sweden1 site, 1 recruiting
- Recruiting
Skåne University Hospital
Malmö, 214 28
Pyotr G Platonov, MD
About this study
Official title: Seroprevalence Study of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
This is a multicenter, non-interventional study to observe the natural progression of the disease and to study the prevalence of pre-existing antibodies to AAV9 used for gene therapy in a population of patients with PKP2 gene-associated ARVC. Participation from all patients is encouraged regardless of interest in or eligibility for gene therapy.
Read the detailed description
Patients will receive standard of care treatments and assessments under the care of their healthcare provider. Biologic samples will be collected annually to measure cardiac and other related biomarkers. Clinical and observational data will be collected prospectively for up to 5 years from the date of enrollment, or until the patient withdraws consent/assent, undergoes heart transplantation, or dies.
If consent is provided, there may be a one-time sample collection to evaluate genetics for research purposes. Quality of Life (QoL) questionnaires will be used to assess a patient's wellbeing and quality of life. If not included as part of a patient's standard of care, diagnostic Holter (or equivalent) monitoring will be required annually. No investigational product will be administered. Participation from all patients is encouraged regardless of interest in or eligibility for gene therapy.
- Keywords
- PKP2 Mutation Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC), Arrhythmogenic Cardiomyopathy (ACM), PKP2-associated ARVC, PKP2-ARVC, PKP2-ACM, Adeno Associated Virus (AAV), Adeno-Associated Virus Serotype 9 (AAV9), Gene Therapy, Gene Transfer, Genetic cardiomyopathy, Heart Failure
- Medical subject headings
- Arrhythmogenic Right Ventricular Dysplasia, Heart Failure
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Retrospective and Prospective | Patients who meet the eligibility criteria are observed and data collected both prospectively and retrospectively. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Investigate the seroprevalence of pre-existing antibodies to AAV9 in patients with PKP2-associated ARVC
Measured: 5 years
Secondary outcomes (4)
To characterize the burden of illness in patients with pathogenic or likely pathogenic PKP2 mutations
Measured: 5 years
To characterize arrhythmic risk in patients with pathogenic or likely pathogenic PKP2 mutations
Measured: 5 years
To evaluate functional status and Quality of Life (QoL) in patients with pathogenic or likely pathogenic PKP2 mutations
Measured: 5 years
To evaluate heart function as assessed by imaging in patients with pathogenic or likely pathogenic PKP2 mutations
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Mar 2024
- Study start31 Jan 2023
- Primary completion29 Mar 2030 (estimated)
- Study completion11 Jul 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Arrhythmogenic Right Ventricular 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
- Other
- Time perspective
- Other
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tenaya Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- University of California, San Francisco, Brigham and Women's Hospital, New York University, University of Colorado, Denver, Johns Hopkins University, The Cleveland Clinic, Mayo Clinic, Medical University of South Carolina, Hopital Louis Pradel, Istituti Clinici Scientifici Maugeri SpA, The Queen Elizabeth Hospital, St George's University Hospitals NHS Foundation Trust, Royal Brompton & Harefield NHS Foundation Trust, Barts & The London NHS Trust, Skane University Hospital, Centro Cardiologico Monzino, Hôpital Haut-Lévêque, Nantes University Hospital, Pitié-Salpêtrière Hospital, Wuerzburg University Hospital, University Hospital Muenster
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06311708
- Sponsor's protocol ID
- TN-401-0011
- Acronym
- RIDGE
- Status last verified
- Oct 2024
- First submitted
- 8 Mar 2024
- Last updated
- 1 Nov 2024
- Results posted
- 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 1 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Seroprevalence Study of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
- ClinicalTrials.gov ID
- NCT06311708
- Acronym / sponsor ID
- RIDGE / TN-401-0011
- Keywords
- PKP2 Mutation Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC), Arrhythmogenic Cardiomyopathy (ACM), PKP2-associated ARVC, PKP2-ARVC, PKP2-ACM, Adeno Associated Virus (AAV), Adeno-Associated Virus Serotype 9 (AAV9), Gene Therapy
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Tenaya TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- University of California, San Francisco, Brigham and Women's Hospital, New York University, University of Colorado, Denver, Johns Hopkins University, The Cleveland Clinic, Mayo Clinic, Medical University of South Carolina, Hopital Louis Pradel, Istituti Clinici Scientifici Maugeri SpA, The Queen Elizabeth Hospital, St George's University Hospitals NHS Foundation Trust, Royal Brompton & Harefield NHS Foundation Trust, Barts & The London NHS Trust, Skane University Hospital, Centro Cardiologico Monzino, Hôpital Haut-Lévêque, Nantes University Hospital, Pitié-Salpêtrière Hospital, Wuerzburg University Hospital, University Hospital Muenster
- Study start
- 31 Jan 2023 (actual)
- Primary completion
- 29 Mar 2030 (estimated)
- Study completion
- 11 Jul 2030 (estimated)
- First posted
- 15 Mar 2024 (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.
This is a multicenter, non-interventional study to observe the natural progression of the disease and to study the prevalence of pre-existing antibodies to AAV9 used for gene therapy in a population of patients with PKP2 gene-associated ARVC. Participation from all patients is encouraged regardless of interest in or eligibility for gene therapy.
Detailed description
Patients will receive standard of care treatments and assessments under the care of their healthcare provider. Biologic samples will be collected annually to measure cardiac and other related biomarkers. Clinical and observational data will be collected prospectively for up to 5 years from the date of enrollment, or until the patient withdraws consent/assent, undergoes heart transplantation, or dies.
If consent is provided, there may be a one-time sample collection to evaluate genetics for research purposes. Quality of Life (QoL) questionnaires will be used to assess a patient's wellbeing and quality of life. If not included as part of a patient's standard of care, diagnostic Holter (or equivalent) monitoring will be required annually. No investigational product will be administered. Participation from all patients is encouraged regardless of interest in or eligibility for gene therapy.
Study design
- Observational model
- Other
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Retrospective and Prospective | – | –Patients who meet the eligibility criteria are observed and data collected both prospectively and retrospectively. |
Who may qualify
- Ages
- 14 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients who are 14-65 years of age at the time of consent, and with confirmed mutations in PKP2 gene with a diagnosis of ARVC.
Key inclusion criteria (4 listed)
- Ages 14-65 years, inclusive, at the time of consent
- Pathogenic or likely pathogenic PKP2 gene mutation
- Diagnosed with ARVC and meet 2010 Modified Task Force Criteria for ARVC as affected.
- Functioning ICD
Key exclusion criteria (5 listed)
- Currently receiving systemic immunosuppressive therapy, cytotoxic chemotherapy, immunoglobulin therapy or monoclonal antibody therapy
- History of clinically significant liver disease, hepatitis B virus, hepatitis C virus, human immunodeficiency virus, or tuberculosis infection
- Previously dosed with any investigational or approved gene therapy product at any time
- Concurrent participation in another interventional clinical trial unless approved by the Sponsor. Participation in a noninterventional study may be allowed at the investigator's discretion.
- History of cardiac transplant.
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
Investigate the seroprevalence of pre-existing antibodies to AAV9 in patients with PKP2-associated ARVC
Time frame: 5 years
4 secondary outcomes
To characterize the burden of illness in patients with pathogenic or likely pathogenic PKP2 mutations
Time frame: 5 years
To characterize arrhythmic risk in patients with pathogenic or likely pathogenic PKP2 mutations
Time frame: 5 years
To evaluate functional status and Quality of Life (QoL) in patients with pathogenic or likely pathogenic PKP2 mutations
Time frame: 5 years
To evaluate heart function as assessed by imaging in patients with pathogenic or likely pathogenic PKP2 mutations
Time frame: 5 years
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 1 Nov 2024.
- 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.
Niharika Kamat, MS contact
21 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Arrhythmogenic Right Ventricular Cardiomyopathy, run by Tenaya Therapeutics at 21 sites in 6 countries.
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 4 inclusion and 5 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 1 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 21 sites in United States, France, United Kingdom, Germany, Italy and 1 more country. 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 Matthew Pollman, MD (650-209-8092, mpollman@tenayathera.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Investigate the seroprevalence of pre-existing antibodies to AAV9 in patients with PKP2-associated ARVC (time frame: 5 years). 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 1 Nov 2024.