Gene therapy trial aims to tame deadly heart rhythms

NCT ID NCT07148089

Recruiting now Disease control Sponsor: Solid Biosciences Inc. Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Aug 11, 2026 · Updated 5 times

Summary

This early-stage trial tests a single dose of SGT-501, a gene therapy given by IV, in 18 people with a rare inherited heart condition called CPVT that can cause dangerous fast heartbeats during exercise or stress. The main goal is to check safety and find the right dose in adults and children aged 7 and older. Participants will be followed for 5 years to monitor side effects and any changes in heart rhythm during exercise tests.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: SGT-501 (gene therapy given as a single intravenous infusion)
What this could lead to: If successful, this could point toward a one-time gene therapy to control dangerous heart rhythms in people with CPVT, potentially reducing reliance on lifelong medication.
What could go wrong: This is a very early (Phase 1b) and small (18 participants) safety study. It is the first time SGT-501 is tested in humans, so safety and effectiveness are unknown. There is a risk of side effects or no benefit.

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Conditions

The condition(s) this trial relates to.

catecholaminergic polymorphic ventricular tachycardia Polymorphic Catecholaminergic Ventricular Tachycardia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CATECHOLAMINERGIC POLYMORPHIC VENTRICULAR TACHYCARDIA

Contacts and locations

Locations

Boston Children's Hospital
RECRUITING

Boston, Massachusetts, 02459, United States
Cleveland Clinic
RECRUITING

Cleveland, Ohio, 44195, United States
Mayo Clinic
RECRUITING

Rochester, Minnesota, 55905, United States
St. Paul's Hospital
RECRUITING

Vancouver, British Columbia, BC V6Z 1Y6, Canada
University of Pennsylvania
NOT_YET_RECRUITING

Philadelphia, Pennsylvania, 19104, United States

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