Status:
RECRUITING
A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
Lead Sponsor:
Solid Biosciences Inc.
Conditions:
Catecholaminergic Polymorphic Ventricular Tachycardia
Eligibility:
All Genders
7+ years
Phase:
PHASE1
Brief Summary
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 ve...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
January 5 2026
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 1 2031
Estimated Enrollment :
18 Patients enrolled
Trial Details
Trial ID
NCT07148089
Start Date
January 5 2026
End Date
May 1 2031
Last Update
December 29 2025
Active Locations (5)
Enter a location and click search to find clinical trials sorted by distance.
1
Boston Children's Hospital
Boston, Massachusetts, United States, 02459
2
Mayo Clinic
Rochester, Minnesota, United States, 55905
3
Cleveland Clinic
Cleveland, Ohio, United States, 44195
4
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104