U
Menu
M
Donate

Rocket Pharmaceuticals Natural History Study for PKP2 ARVC (also known as ACM)

Now enrolling patients with PKP2-ACM.

Quick Facts 

📌 Studying: Natural history for patients who have ARVC (also known as ACM) with a PKP2 pathogenic (or likely pathogenic) genetic variant, managed with standard of care
📌 Condition: ARVC (also known as ACM) with a PKP2-variant and an existing ICD
📌 Ages: 12 years and older
📌 Sites: Currently enrolling in the United States
📌 Status: Now Enrolling

Learn More About Eligibility

About This Research Study

Rocket Pharmaceuticals is now enrolling people with ARVC (also known as ACM) with a PKP2 pathogenic (or likely pathogenic) genetic variant for a natural history study. The goal of this study is to describe the natural history and clinical events for these patients – including the signs and symptoms, key clinical events, and impact of the disease on quality of life.

Primary assessments include medical history, questionnaires, ICD data, examination of cardiac structure and performance including echocardiogram, and laboratory assessments including cardiac biomarkers and genetic testing. 

YouTube player

Learn more about this study by watching our recent webinar with Rocket Pharmaceuticals

What is a Natural History Study?

A natural history study is an observational (no new treatments tested) form of research that follows a group of people over time to understand how a specific disease or condition develops. The goal is to collect health information to learn about the disease’s progression and provide a foundation for developing new treatments and clinical trial designs.

Learn more about research studies – including frequently asked questions and questions you may have as you consider whether participation is right for you – here.

What are the basic eligibility criteria?

  • Age 12 years or older
  • Clinical diagnosis of arrhythmogenic cardiomyopathy (ACM)
  • Documentation of a pathogenic or likely pathogenic variant in PKP2
    • Not sure of your gene? We can help with genetic testing. Learn more 
  • Have an ICD (for at least 6 months)
  • Left ventricular ejection fraction of greater than or equal to 50%

Full eligibility criteria will be evaluated by the researcher to determine if an individual is the right candidate for this study

Start Eligibility Form

FAQs 

What if I change my mind about participating?

Once you enroll in the trial, you can leave the trial at any time for any reason. If you decide to leave the trial, please consult with your doctor to see what/if any, follow-up care will be needed.

Can I stay on my current treatment?

Yes. This is a non-interventional study, meaning that your treatments will not be changed, and you will not be put on any new treatments or have your current treatments modified.

Where are the trial sites and how often would I need to travel there?

Current clinical trial site locations are listed below, you may need to travel about 4-7 times to the trial site for the scheduled visit. Additional sites in the U.S. and Europe may open soon. If you are interested in hearing from SADS when other locations begin enrolling, please register below.

Duke University Hospital, Durham, North Carolina, USA * Enrolling Now 

Boston Children’s Hospital, Boston, Massachusetts, USA * Not Yet Recruiting

Amsterdam UMC, Amsterdam, Netherlands *Not Yet Recruiting

Learn more about these sites on ClinicalTrials.gov here.

Will the costs of participating in the trial be covered by Rocket Pharmaceuticals?

Yes! All study-related assessments will be provided at no cost, and you will be reimbursed for reasonable study-related expenses

Rocket Pharmaceuticals Natural History Study

After filling out the form below, you’ll have a chance to discuss this trial – and any questions you might have – directly with the staff at the SADS Foundation. Then a member of the SADS staff will connect you one-on-one with the site coordinator of your choice.

"*" indicates required fields

Name*
Location*
Do you have ARVC (also known as ACM)?*
Do you have a pathogenic or likely pathogenic PKP2 genetic variant?*
Do you have an ICD, and have you had the ICD for at least 6 months?*
Consent to Travel*
Consent*
I would like to get news and updates from SADS Foundation*

The information contained herein is being made available as a public service by the SADS Foundation (SADS). No posted information or material provided is intended to constitute medical or professional advice. SADS makes no representations or warranties, either express or implied, as to the accuracy of any posted information and assumes no responsibility for any errors or omissions contained therein. SADS makes no representations or warranties, either express or implied, that any products or services adhere to or satisfy any standards or requirements that may be applicable to such product or service. Furthermore, no warranty, express or implied, is created by providing information and does not in any way constitute an endorsement by SADS. No one shall be entitled to claim detrimental reliance on any views or information, or to claim any duty on the part of SADS to update posted information. In no event shall SADS be held liable to you or anyone else for any decision made or action taken in reliance on such information or views.