RecruitingPhase 1NCT07148089

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


Sponsor

Solid Biosciences Inc.

Enrollment

18 participants

Start Date

Feb 23, 2026

Study Type

INTERVENTIONAL

Conditions

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

Min Age: 7 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called SGT-501 for people with catecholaminergic polymorphic ventricular tachycardia. The study is currently recruiting participants at 5 locations. People eligible for this study include aged 7 Years and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGSGT-501

IV for infusion


Locations(5)

Boston Children's Hospital

Boston, Massachusetts, United States

Mayo Clinic

Rochester, Minnesota, United States

Cleveland Clinic

Cleveland, Ohio, United States

University of Pennsylvania

Philadelphia, Pennsylvania, United States

St. Paul's Hospital

Vancouver, British Columbia, Canada

View Full Details on ClinicalTrials.gov

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NCT07148089


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