Gene therapy trial aims to tame deadly heart rhythms
NCT ID NCT07148089
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
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.
Disclaimer
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02459, United States
Contact Email: •••••@•••••
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Cleveland Clinic
RECRUITINGCleveland, Ohio, 44195, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Mayo Clinic
RECRUITINGRochester, Minnesota, 55905, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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St. Paul's Hospital
RECRUITINGVancouver, British Columbia, BC V6Z 1Y6, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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University of Pennsylvania
NOT_YET_RECRUITINGPhiladelphia, Pennsylvania, 19104, United States