Phase 1b Study of SGT-501 Gene Therapy for Treating Catecholaminergic Polymorphic Ventricular Tachycardia in Children and Adults

What this Trial Is About

Researchers are investigating the safety and tolerability of a single intravenous dose of SGT-501 gene therapy in people with catecholaminergic polymorphic ventricular tachycardia CPVT, a rare heart rhythm disorder. This Phase 1b study focuses on adults initially, with later inclusion of children aged 7 to under 18 years, following safety reviews. The study aims to collect early safety data and understand the effects of this gene therapy over time. Participants will receive one intravenous infusion of SGT-501 at different dose levels depending on their age cohort. Adults aged 18 and older are divided into two cohorts receiving different doses, while children aged 7 to under 18 years will receive a dose at or below those given to adults. The study includes an active treatment period of one year followed by a long-term follow-up of four years to monitor participants health and response after treatment. During the study, participants will be monitored closely for treatment-emergent adverse events up to one year after the infusion. Researchers will assess ventricular arrhythmia scores through exercise stress tests at baseline and six months. Participants will undergo safety evaluations, laboratory tests, and disease monitoring over five years in total, including the long-term follow-up period. This extended observation ensures careful evaluation of the gene therapys effects and safety.

A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)