NCT07148089

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

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

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 18 (estimated)
• Sponsor: Solid Biosciences Inc. · Industry
• Sex: All
• Age: 7 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Catecholaminergic Polymorphic Ventricular Tachycardia

Interventions

Type	Name	Description
DRUG	SGT-501	IV for infusion

Timeline

• Start date: 2026-02-23
• Primary completion: 2027-06-01
• Completion: 2031-05-01
• First posted: 2025-08-29
• Last updated: 2026-03-27

Locations

5 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study