Actively Recruiting
A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
Led by Solid Biosciences Inc. · Updated on 2026-04-27
18
Participants Needed
5
Research Sites
270 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official 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 with documented ventricular tachycardia or ventricular ectopy consistent with CPVT
- Central laboratory confirmation of pathogenic or likely pathogenic RYR2 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 before screening
- Prior exercise stress test showing ventricular arrhythmia score of 2 or higher
- For the first 2 participants in each cohort, a functioning ICD device required
- Up to date with meningococcal vaccination or willing to receive vaccine
- Other inclusion criteria per protocol
You will not qualify if you...
- Abnormal liver function with GGT > 1.5 times upper limit of normal or total bilirubin above normal
- Abnormal kidney function with estimated glomerular filtration rate less than 60 mL/min/1.73 m2
- Significant blood clotting abnormalities including high INR or APTT or low platelet count
- Presence of other cardiomyopathy or inherited arrhythmia from non-RYR2 mutations
- Previous treatment with gene transfer drugs
- Use of investigational drugs within 90 days or 5 half-lives prior to screening
- Contraindication or refusal of required immunosuppression
- Body mass index of 30 kg/m2 or higher
- Other exclusion criteria per protocol
AI-Screening
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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
CONTACT
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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