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SGT-501 Gene Therapy for Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)

This study is testing a gene therapy called SGT-501 for people with Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT), a heart condition that can cause dangerous heart rhythms. Researchers want to see how safe SGT-501 is when given as a single intravenous (IV) infusion. The study will enroll 18 participants, including adults and children aged 7 and older, who have a confirmed diagnosis of CPVT and a specific genetic change (RYR2 variant). The main goal is to track any side effects participants might experience for about a year after receiving the treatment. The study is currently recruiting participants.

Study design
This is a Phase 1b, open-label, dose-finding study, meaning all participants will receive SGT-501 and researchers will know which dose is given. It plans to enroll 18 participants.
What's involved
Participants will receive a single intravenous infusion of SGT-501 and will be monitored for 5 years after treatment, including an active treatment period of 1 year.
Compensation
Not stated in the trial record.
Follow-up
Participants will be monitored for 5 years after receiving SGT-501, with an active treatment period of 1 year and a long-term follow-up period of 4 years.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07148089

A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)

Recruiting
PHASE1Ages 7+InterventionalTreatment
Solid Biosciences Inc.
~18 participants
Updated 2026-08-10 on ClinicalTrials.gov
What's tested:SGT-501

At a glance

Recruiting sites
4 of 5 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of Participants with Treatment-Emergent Adverse Event (TEAEs)s through Day 360
Measured over First dose through Day 360

Conditions

Catecholaminergic Polymorphic Ventricular Tachycardia

Explore related trials

Catecholaminergic Polymorphic Ventricular Tachycardia by ECG FindingRYR2 Gene

Where it's being run

5 sites across 5 states
Massachusetts1
Minnesota1
Ohio1
Pennsylvania1
British Columbia1

Who to contact

Solid Bio Clinical Trials
[email protected]
617-337-4680
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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 laboratory determination of a RYR2 variant that is pathogenic or likely pathogenic for CPVT.
Documented history of life-threatening ventricular arrhythmic event defined as: survived sudden cardiac arrest, sudden cardiac arrest with appropriate implantable cardioverter defibrillator (ICD) shock, arrhythmic syncope, or sustained ventricular tachycardia (30 seconds or more) with or without ICD shock.
On stable dose (defined as no change in dose by more than 50% for at least 1 month prior to Screening) of standard-of-care therapy defined as a beta-blocker and/or flecainide.
Documented prior history of EST demonstrating a ventricular arrythmia score (VAS) score of ≥ 2.
For the first 2 participants in each cohort only: a properly functioning ICD device in place. Following review of data from Cohorts 1 and 2, the Data Safety and Monitoring Board (DSMB) will determine if this criterion is required for participants in Cohort 3.
Must be up to date with meningococcal vaccination per national guidelines or willing to receive meningococcal vaccine to achieve this.

Exclusion

Abnormal liver function: gamma-glutamyl transferase (GGT) \> 1.5 × upper limit of normal \[ULN\] or total bilirubin \> ULN).
Abnormal renal function defined by estimated glomerular filtration rate \< 60 milliliter /minute (mL/min)/1.73-square meter (m\^2) using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) Formula.
Clinically significant abnormalities of coagulation including international normalized ratio or activated partial thromboplastin time \> 1.2 × ULN or platelets \< 150,000 cells/cubic millimeter (mm\^3).
Potential concomitant cardiomyopathy or inherited arrhythmia as evidenced by pathogenic or likely pathogenic mutation other than RYR2 obtained on cardiac panel during Screening.
Current or prior treatment with an approved or investigational gene transfer drug.
Exposure to another investigational drug within 90 days prior to Screening or 5 half-lives since last administration, whichever is longer.
Contraindication or unwillingness to receive required immunosuppression regimen.
Body mass index ≥ 30 kilograms per square meter (kg/m\^2).

What this trial measures

  • Number of Participants with Treatment-Emergent Adverse Event (TEAEs)s through Day 360First dose through Day 360