Solid Biosciences Gene Therapy Trial for CPVT

Now enrolling patients with catecholaminergic polymorphic ventricular tachycardia (CPVT).

Quick Facts 

📌 Trial Name: ARTEMIS Study

📌 Condition: CPVT caused by a pathogenic or likely pathogenic variant in RYR2

📌 Ages: Adults 18 years of age or older to begin with followed by those between the ages of 7 and 18 years old.

📌 Sites: United States and Canada (North America).

📌 Status: Now Enrolling

About This Trial

The ARTEMIS Study is the first in-human clinical trial to see if an investigational gene therapy drug, called SGT-501, is safe, tolerable, and works in humans. Everyone taking part in the ARTEMIS Study will be given the study drug along with drugs to suppress your body’s immune response and antibiotics to prevent infection. You will receive the drug via a single IV infusion (through a vein in your arm). There will be a short hospital stay after you receive SGT-501 to monitor your health.

The goals of the study are to understand:

• How safe the gene therapy is for humans
• Any potential side effects
• The best dose of the gene therapy 
• The effects of this gene therapy on the human body

Learn more about this trial by watching our recent webinar with Solid Biosciences

What is the gene therapy being investigated, and how does it work?

CPVT is a genetic condition that affects the heart’s rhythm. It is caused by changes in certain genes responsible for balancing the calcium levels in heart cells. There are different genes that can cause CPVT – including RYR2. Changes in this gene can lead to abnormal heart rhythms, particularly during physical activity or emotional stress. 

The purpose of this research study is to see if a new investigational gene therapy called SGT-501 is safe, tolerable, and works in humans. In this research study, SGT-501 is used to insert a copy of a functional gene into your cells. The goal of treatment with SGT-501 is to increase a protein that will improve calcium levels when there is a mutation in the RYR2 gene causing CPVT. The goal of this gene therapy is to potentially change your underlying CPVT rather than just managing your symptoms.

Learn more about gene therapy trials – including frequently asked questions, how gene therapy is different from other clinical trials, and questions you may have as you consider whether participation is right for you – here.

What are the basic eligibility criteria?

• Please note that the first two patients in this gene therapy trial are required to have ICDs as an extra safety precaution. At the moment, this study is only recruiting patients with ICDs. After the first two patients with ICDs are dosed, this study will open up to those without devices.
• Participants for this phase of the trial must be 18 years or older (1st and 2nd cohort) or between the ages of 7 and 18 years (3rd cohort).
• Clinical diagnosis of CPVT.
• Have been taking a beta-blocker (I.e., nadolol or propranolol) and/or flecainide for at least 1 month prior to screening.
• Are able to perform an Exercise Stress Test on a treadmill.
• Have experienced heart arrhythmia symptoms in the past or have experienced arrhythmias due to CPVT.
• Genetic testing documenting a pathogenic or likely pathogenic variant in RYR2. Not sure of your gene? SADS can help with genetic testing. Learn more ➡️

Full eligibility criteria will be evaluated by the clinical trial doctor to determine if an individual is the right candidate for this trial. 

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 follow-up care will be needed.

Can I stay on my current treatment?

Participants should be taking a beta-blocker (i.e., nadolol or propranolol) and/or flecainide for at least 1 month prior to screening and throughout the study. Please discuss continuation of current treatments or medications with the study doctor.

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

Participants in this study will need to make regular office visits during the study period. 

During the first 45 days, you will attend frequent study center visits to closely monitor your health and well-being. Visits will become less frequent during the remainder of the Active Treatment Period (1 year) and the Long-term Follow-up Period (4 years). Details can be discussed with the study doctor.

Current clinical trial site locations are listed below. Additional sites in the U.S. and Canada may open soon. If you are interested in hearing from SADS when other locations begin enrolling, please register below.

• Rochester, MN *Enrolling Patients 
• Boston, MA *Not Yet Recruiting 
• Vancouver, BC (Canada) – please note that this site is only taking referrals from Canadian citizens. *Not Yet Recruiting 
• Cleveland, OH *Not Yet Recruiting 
• Philadelphia, PA *Not Yet Recruiting 

Learn more about these sites on ClinicalTrials.gov here.

Will the costs of participating in the trial be covered by Solid Biosciences?

Yes! The investigational gene therapy and all study-related assessments will be provided at no cost, and you will be reimbursed for reasonable study-related expenses.

Form for Initial Interest in Enrollment 

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.

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 view.