SGT-501 gene therapy for RYR2-related CPVT

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 1b, first-in-human, multicenter, open-label dose-finding program gives a single IV dose of SGT-501 and follows participants for safety. Adult cohorts are enrolled first with an optional pediatric cohort (ages 7 to <18) initiated after DSMB review. Eligible participants require a pathogenic or likely pathogenic RYR2 variant and a history of life-threatening ventricular arrhythmia while on stable beta-blocker and/or flecainide therapy. Participants undergo an active 1-year monitoring period followed by 4 years of long-term follow-up for safety and durability signals.

Why it matters

Who can join: the full rules

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 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.
* Other inclusion criteria to be applied as per protocol.

Exclusion Criteria:

* 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).
* Other exclusion criteria to be applied as per protocol.

Where it's running

• Boston Children's Hospital
  Boston, Massachusetts, United States Recruiting
• Mayo Clinic
  Rochester, Minnesota, United States Recruiting
• Cleveland Clinic
  Cleveland, Ohio, United States Recruiting
• St. Paul's Hospital
  Vancouver, British Columbia, Canada Recruiting
• University of Pennsylvania
  Philadelphia, Pennsylvania, United States Not yet recruiting

Study contacts

• Study coordinator: Solid Bio Clinical Trials
• Email: clinicaltrials@solidbio.com
• Phone: 617-337-4680

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.