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A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
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
Who can join
Ages 7 and older · All sexes
Full eligibility criteria
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.
About the study
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.
What is being tested
- SGT-501 (drug)
Sponsor: Solid Biosciences Inc. · Participants: 18 · Started: Feb 23, 2026
Contact the study team
- Solid Bio Clinical Trials · Phone: 617-337-4680
Official record on ClinicalTrials.gov — NCT07148089
Locations in the U.S.
| Massachusetts | Boston Children's Hospital, Boston |
| Minnesota | Mayo Clinic, Rochester |
| Ohio | Cleveland Clinic, Cleveland |
| Pennsylvania | University of Pennsylvania, Philadelphia (Not yet recruiting) |
From ClinicalTrials.gov, data retrieved Oct 2, 2026. Each study sets its own eligibility; the study team decides who can join.