A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
Recruiting
Phase 1
Interventional Study
Catecholaminergic Polymorphic Ventricular Tachycardia
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 7 and older
- Sex
- Any
- Trial phase
- Phase 1
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 18 (estimated)
- Sponsor
- Solid Biosciences Inc. · Industry
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About This Trial
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 partic…
Trial Locations
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Eligibility Criteria
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 lab…
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