A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT) (ARTEMIS)

A Phase 1b, Multicenter, Open-Label, Dose Finding Study to Investigate the Safety and Tolerability of a Single Intravenous Dose of SGT-501 in Patients With Catecholaminergic Polymorphic Ventricular Tachycardia

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 participants with catecholaminergic polymorphic ventricular tachycardia (CPVT). The first-in-human (FIH) safety study will focus on obtaining safety data in adult participants. Cohort 1 and Cohort 2 (optional for dose exploration) will include participants ≥ 18 years of age. Cohort 3 will include participants ≥ 7 to < 18 years of age and will be initiated following data and safety monitoring board (DSMB) recommendations. Participants will be monitored for 5 years post-administration of SGT-501 including the active treatment period (1 year) and long-term follow-up (LTFU) (4 years) period.

Study Overview

• Status: Recruiting
• Conditions: Catecholaminergic Polymorphic Ventricular Tachycardia
• Intervention / Treatment: Drug: SGT-501

• Study Type: Interventional
• Enrollment (Estimated): 18
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Solid Bio Clinical Trials; Phone Number: 617-337-4680; Email: clinicaltrials@solidbio.com

Study Locations

• Canada
  • British Columbia
    • Vancouver, British Columbia, Canada, BC V6Z 1Y6
      • Recruiting
      • St. Paul's Hospital
      • Contact: Brianna Davies; Phone Number: 64252 604-682-2344; Email: brianna.davies@phc.ca
      • Principal Investigator: Thomas Roston, MD
• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02459
      • Recruiting
      • Boston Children's Hospital
      • Contact: Elizabeth DeWitt, MD; Email: gene.therapy@childrens.harvard.edu
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Recruiting
      • Mayo Clinic
      • Contact: Kolawole Olarinoye; Phone Number: 507-293-1564; Email: Olarinoye.Kolawole@mayo.edu
      • Principal Investigator: John Giudicessi, MD
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Recruiting
      • Cleveland Clinic
      • Principal Investigator: Peter Aziz, MD
      • Contact: Paige Ewing; Phone Number: 216-445-8798; Email: ewingp@ccf.org
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Not yet recruiting
      • University of Pennsylvania

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

Type of Participant and Disease Characteristics:

• Clinical diagnosis of CPVT, based on documented history of polymorphic or bidirectional non-sustained ventricular tachycardia with exercise or ventricular ectopy in a pattern consistent with CPVT on EST.
• Central Screening laboratory determination of a RYR2 variant that is pathogenic or likely pathogenic for CPVT.
• Documented history of life-threatening ventricular arrhythmic event defined as: survived sudden cardiac arrest, sudden cardiac arrest with appropriate implantable cardioverter defibrillator (ICD) shock, arrhythmic syncope, or sustained ventricular tachycardia (30 seconds or more) with or without ICD shock.
• On stable dose (defined as no change in dose by more than 50% for at least 1 month prior to Screening) of standard-of-care therapy defined as a beta-blocker and/or flecainide.
• Documented prior history of EST demonstrating a ventricular arrythmia score (VAS) score of ≥ 2.
• For the first 2 participants in each cohort only: a properly functioning ICD device in place. Following review of data from Cohorts 1 and 2, the Data Safety and Monitoring Board (DSMB) will determine if this criterion is required for participants in Cohort 3.
• Must be up to date with meningococcal vaccination per national guidelines or willing to receive meningococcal vaccine to achieve this.
• Other inclusion criteria to be applied as per protocol.

Exclusion Criteria:

• Abnormal liver function: gamma-glutamyl transferase (GGT) > 1.5 × upper limit of normal [ULN] or total bilirubin > ULN).
• Abnormal renal function defined by estimated glomerular filtration rate < 60 milliliter /minute (mL/min)/1.73-square meter (m^2) using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) Formula.
• 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).
• Potential concomitant cardiomyopathy or inherited arrhythmia as evidenced by pathogenic or likely pathogenic mutation other than RYR2 obtained on cardiac panel during Screening.
• Current or prior treatment with an approved or investigational gene transfer drug.
• Exposure to another investigational drug within 90 days prior to Screening or 5 half-lives since last administration, whichever is longer.
• Contraindication or unwillingness to receive required immunosuppression regimen.
• Body mass index ≥ 30 kilograms per square meter (kg/m^2).
• Other exclusion criteria to be applied as per protocol.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1 (≥ 18 years of age); Participants will receive a single intravenous (IV) infusion (dose 1) of SGT-501.	Drug: SGT-501; IV for infusion
Experimental: Cohort 2 (≥ 18 years of age); Participants will receive a single IV infusion (dose 2) of SGT-501.	Drug: SGT-501; IV for infusion
Experimental: Cohort 3 (≥ 7 to < 18 years); Participants will receive a single IV infusion (level at or below dose(s) assessed in adults) of SGT-501.	Drug: SGT-501; IV for infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of Participants with Treatment-Emergent Adverse Event (TEAEs)s through Day 360	First dose through Day 360

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in ventricular arrhythmia score (VAS) on exercise stress test (EST) at Day 180	An EST will be used to evaluate ventricular arrhythmia scores. The EST will be performed using the standard Bruce treadmill protocol.	Baseline, Day 180

Collaborators and Investigators

• Sponsor: Solid Biosciences Inc.

Study record dates

Study Major Dates

• Study Start (Actual): February 23, 2026
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): May 1, 2031

Study Registration Dates

• First Submitted: August 22, 2025
• First Submitted That Met QC Criteria: August 22, 2025
• First Posted (Actual): August 29, 2025

Study Record Updates

• Last Update Posted (Actual): August 10, 2026
• Last Update Submitted That Met QC Criteria: August 7, 2026
• Last Verified: August 1, 2026

More Information

Terms related to this study

• Keywords: Gene Therapy; Cardiac; Catecholaminergic polymorphic ventricular tachycardia (CPVT); SGT-501; Ryanodine Receptor 2 (RYR2); adeno-associated virus serotype 8 (AAV8)
• Additional Relevant MeSH Terms: Cardiac Conduction System Disease; Cardiovascular Diseases; Pathologic Processes; Heart Diseases; Arrhythmias, Cardiac; Tachycardia, Ventricular; Tachycardia; Pathological Conditions, Signs and Symptoms; Polymorphic Catecholaminergic Ventricular Tachycardia
• Other Study ID Numbers: SGT-501-101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes