Actively Recruiting
A Phase 1b, Open-Label, Multicenter Study to Investigate Safety and Tolerability of a Single Intravenous Dose of SGT-501 in Patients With Catecholaminergic Polymorphic Ventricular Tachycardia
Led by Solid Biosciences Inc. · Updated on 2026-06-02
18
Participants Needed
5
Research Sites
204 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and tolerability of a single intravenous dose of SGT-501 gene therapy in participants with catecholaminergic polymorphic ventricular tachycardia (CPVT), a rare heart condition characterized by abnormal heart rhythms triggered by exercise or stress. This Phase 1b study focuses on adults initially and includes children aged 7 to under 18 years following safety reviews by a monitoring board. The study is sponsored by Solid Biosciences Inc. and aims to gather important safety data in this first-in-human trial. Participants will receive one intravenous infusion of SGT-501 at varying doses depending on their cohort: Cohort 1 and 2 include adults aged 18 and older, while Cohort 3 includes children aged 7 to under 18 years. The dosing for children will be at or below the levels assessed in adults. The study is open-label and dose-finding, with optional dose exploration in Cohort 2. Participants will be monitored closely during the active treatment period of one year. Throughout the study, participants will be followed for a total of five years, including four years of long-term follow-up after treatment. Researchers will assess safety by tracking treatment-emergent adverse events up to 360 days post-dose. They will also evaluate changes in ventricular arrhythmia scores using exercise stress tests. Participants will undergo clinical assessments, laboratory tests, and monitoring to evaluate heart rhythm and overall health during the study and follow-up periods.
CONDITIONS
Brief Title
A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Clinical diagnosis of CPVT based on history of specific ventricular tachycardia or ectopy with exercise
- Presence of a pathogenic or likely pathogenic RYR2 gene variant
- History of life-threatening ventricular arrhythmic event such as survived sudden cardiac arrest, arrhythmic syncope, or sustained ventricular tachycardia
- Stable dose of beta-blocker and/or flecainide for at least 1 month prior to screening
- Ventricular arrhythmia score of 2 or higher on prior exercise stress test
- For first 2 participants in each cohort, a functioning implantable cardioverter defibrillator (ICD) is required; may be waived for younger cohort after safety review
- Up to date with meningococcal vaccination or willing to receive it
- Other inclusion criteria as per protocol
You will not qualify if you...
- Abnormal liver function indicated by elevated GGT or bilirubin levels
- Reduced kidney function with estimated glomerular filtration rate below 60 mL/min/1.73 m2
- Significant clotting abnormalities including elevated INR or low platelets
- Other cardiomyopathy or inherited arrhythmia mutations besides RYR2
- Previous treatment with approved or investigational gene therapy
- Exposure to another investigational drug within 90 days or 5 half-lives
- Contraindications or unwillingness to follow immunosuppression regimen
- Body mass index of 30 kg/m2 or higher
- Other exclusion criteria as per protocol
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single day treatment with follow-up through Day 360
Participants receive a single intravenous (IV) infusion of the gene therapy SGT-501.
1 infusion visit and multiple follow-up visits through Day 360
Trial Site Locations
Total: 5 locations
1
Boston Children's Hospital
Boston, Massachusetts, United States, 02459
Actively Recruiting
2
Mayo Clinic
Rochester, Minnesota, United States, 55905
Actively Recruiting
3
Cleveland Clinic
Cleveland, Ohio, United States, 44195
Actively Recruiting
4
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Not Yet Recruiting
5
St. Paul's Hospital
Vancouver, British Columbia, Canada, BC V6Z 1Y6
Actively Recruiting
Research Team
S
Solid Bio Clinical Trials
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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