SGT-501 Gene Therapy for CPVT

(ARTEMIS Trial)

Not yet recruiting at 4 trial locations

Overseen By Solid Bio Clinical Trials

Age: Any Age

Sex: Any

Trial Phase: Phase 1

Sponsor: Solid Biosciences Inc.

Must be taking: Beta-blockers, Flecainide

Must not be taking: Gene transfer drugs

Disqualifiers: Liver, Renal, Coagulation, Cardiomyopathy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 1 year

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new gene therapy, SGT-501, to determine its safety and tolerability for individuals with catecholaminergic polymorphic ventricular tachycardia (CPVT), a heart condition that causes dangerous irregular heartbeats. Participants will receive a single intravenous dose of the therapy, with different doses tested in separate groups. This trial suits individuals with a clinical diagnosis of CPVT who have experienced life-threatening heart events, such as sudden cardiac arrest or serious arrhythmias. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.

Do I have to stop taking my current medications for the trial?

The trial requires participants to be on a stable dose of their current medications, specifically a beta-blocker and/or flecainide, for at least one month before screening. So, you will not need to stop taking these medications.

Is there any evidence suggesting that SGT-501 is likely to be safe for humans?

Research shows that SGT-501, a new gene therapy, is being tested for safety in people with CPVT, a rare heart condition. The FDA has given this treatment special designations, such as Fast Track, indicating its potential importance for treating serious conditions. However, this marks the first human trial of SGT-501.

The study is in its early stages, focusing primarily on assessing the treatment's safety. Participants will receive a single dose and undergo close monitoring for any side effects. This careful observation helps researchers understand how well individuals tolerate the treatment and allows for early detection of potential issues.

Due to this being the first human trial of SGT-501, information on its tolerance is limited. Participants will provide crucial safety data for future research. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for CPVT?

Researchers are excited about SGT-501 because it represents a novel gene therapy approach for treating Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT). Unlike traditional treatments like beta-blockers or calcium channel blockers, which manage symptoms, SGT-501 aims to address the root cause of CPVT by correcting the genetic mutation responsible for the condition. This innovative treatment is delivered via a single intravenous infusion, potentially offering a one-time solution that could significantly reduce the risk of life-threatening arrhythmias. By targeting the genetic basis of CPVT, SGT-501 promises a more comprehensive and lasting impact compared to conventional therapies.

What evidence suggests that SGT-501 might be an effective treatment for CPVT?

Research shows that SGT-501 is a promising new gene therapy for treating catecholaminergic polymorphic ventricular tachycardia (CPVT), a condition that causes dangerous heart rhythms due to calcium issues in heart cells. SGT-501 aims to correct this by delivering a healthy version of a specific gene to the heart cells. Early studies suggest that this therapy could help manage these irregular heartbeats. The FDA has recognized SGT-501 for its potential as a groundbreaking treatment. In this trial, participants will receive a single intravenous infusion of SGT-501, with different cohorts receiving varying doses. Since this is the first human trial, more research is needed to confirm its effectiveness. ^{2 3 4 5 6}

Are You a Good Fit for This Trial?

This trial is for adults and children (≥7 years old) with a heart rhythm disorder called CPVT. It's open to those who haven't had gene therapy before and are willing to be monitored for 5 years after treatment. People with other significant health issues or who've had certain treatments may not qualify.

Inclusion Criteria

I am vaccinated against meningitis as recommended, or I am willing to get vaccinated.

I have been diagnosed with CPVT based on specific heart rhythm patterns during exercise.

Documented prior history of EST demonstrating a ventricular arrhythmia score (VAS) score of ≥ 2

Exclusion Criteria

Clinically significant abnormalities of coagulation including international normalized ratio or activated partial thromboplastin time > 1.2 × ULN or platelets < 150,000 cells/cubic millimeter (mm^3)

Other exclusion criteria to be applied as per protocol

I have been treated with a gene therapy drug.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single intravenous dose of SGT-501

1 year

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 years

What Are the Treatments Tested in This Trial?

Interventions

• SGT-501

Trial Overview SGT-501, a new gene therapy, is being tested in people with CPVT. This first-in-human study will check its safety at different doses in adults first, then possibly in kids based on safety board advice. Participants get one IV dose and are watched over for a year actively, then four more years.

How Is the Trial Designed?

Group I: Cohort 3 (≥ 7 to < 18 years) Experimental Treatment 1 Intervention

Participants will receive a single IV infusion (level at or below dose(s) assessed in adults) of SGT-501.

Group II: Cohort 2 (≥ 18 years of age) Experimental Treatment 1 Intervention

Participants will receive a single IV infusion (dose 2) of SGT-501.

Group III: Cohort 1 (≥ 18 years of age) Experimental Treatment 1 Intervention

Participants will receive a single intravenous (IV) infusion (dose 1) of SGT-501.

Who Is Running the Clinical Trial?

Solid Biosciences Inc.

Lead Sponsor

Trials

4

Recruited

130+

Citations

1. investors.solidbio.com investors.solidbio.com/news-releases/news-release-details/solid-biosciences-receives-fda-fast-track-designation-sgt-501

Solid Biosciences Receives FDA Fast Track Designation ... SGT-501 has now received FDA Fast Track, Orphan Drug and Rare Pediatric Disease designations -. - Potential first-in-class therapy with FDA ...

2. cgtlive.com cgtlive.com/view/solid-biosciences-cpvt-gene-therapy-sgt-501-cleared-phase-1b-trial

Solid Biosciences' CPVT Gene Therapy SGT-501 Cleared ... SGT-501 is intended to address CPVT by providing a full-length, codon-optimized copy of the cardiac calsequestrin gene to the muscle cells of the heart.

3. firstwordpharma.com firstwordpharma.com/story/5988657

Solid Biosciences Reports Second Quarter 2025 Financial ... SGT-501 is a novel gene therapy intended to promote excess levels of the cardiac CASQ2 protein to address the underlying ryanodine receptor ( ...

4. investors.solidbio.com investors.solidbio.com/news-releases/news-release-details/solid-biosciences-announces-fda-ind-and-health-canada-cta

Overview & News - : - Press Release SGT-501 is a novel gene therapy for rare, life-threatening, genetic arrhythmogenic disease with no approved therapies.

5. withpower.com withpower.com/trial/phase-1-polymorphic-catecholaminergic-ventricular-tachycardia-9-2025-12c13

SGT-501 Gene Therapy for CPVT · Info for Participants Trial Overview SGT-501, a new gene therapy, is being tested in people with CPVT. This first-in-human study will check its safety at different doses in adults ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT07148089

NCT07148089 | A Study of SGT-501 Gene Therapy in ... This is a Phase 1b, Multicenter, Open-Label, Dose Finding Study to Investigate the Safety and Tolerability of a Single Intravenous Dose of SGT-501 in ...