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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
18
IV for infusion
Boston Children's Hospital
Boston, Massachusetts, United States
NOT_YET_RECRUITINGMayo Clinic
Rochester, Minnesota, United States
RECRUITINGCleveland Clinic
Cleveland, Ohio, United States
NOT_YET_RECRUITINGUniversity of Pennsylvania
Philadelphia, Pennsylvania, United States
NOT_YET_RECRUITINGSt. Paul's Hospital
Vancouver, British Columbia, Canada
RECRUITINGNumber of Participants with Treatment-Emergent Adverse Event (TEAEs)s through Day 360
Time frame: First dose through Day 360
Change from baseline in ventricular arrhythmia score (VAS) on exercise stress test (EST) at Day 180
An EST will be used to evaluate ventricular arrhythmia scores. The EST will be performed using the standard Bruce treadmill protocol.
Time frame: Baseline, Day 180
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