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
Solid Biosciences Inc.
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 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.
Eligibility
- Age range
- 7+ years
- Sex
- All
- Healthy volunteers
- No
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, arrhy…
Interventions
- DrugSGT-501
IV for infusion
Locations (5)
- Boston Children's HospitalBoston, Massachusetts
- Mayo ClinicRochester, Minnesota
- Cleveland ClinicCleveland, Ohio
- University of PennsylvaniaPhiladelphia, Pennsylvania
- St. Paul's HospitalVancouver, British Columbia